<clinical_study>
  <!-- This xml conforms to an XML Schema at:
    https://clinicaltrials.gov/ct2/html/images/info/public.xsd -->
  <required_header>
    <download_date>ClinicalTrials.gov processed this data on September 20, 2023</download_date>
    <link_text>Link to the current ClinicalTrials.gov record.</link_text>
    <url>https://clinicaltrials.gov/ct2/show/NCT02993224</url>
  </required_header>
  <id_info>
    <org_study_id>CICL670FIC05</org_study_id>
    <secondary_id>2016-002282-61</secondary_id>
    <nct_id>NCT02993224</nct_id>
  </id_info>
  <brief_title>Open-label, Multicenter Study Assessing Preference for Deferasirox Film-coated Tablet Compared to Dispersible Tablet</brief_title>
  <acronym>Jupiter</acronym>
  <official_title>Open-label, Multicenter, Single Arm, Phase II Study Assessing Treatment Patient Preference for New Deferasirox Formulation (Film-coated Tablet) Compared to the Reference Deferasirox Dispersible Tablet Formulation</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Novartis Pharmaceuticals</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Novartis</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      Study to evaluate patient preference of deferasirox film-coated tablet (FCT) or deferasirox&#xD;
      dispersible tablet (DT) in patient with transfusion - dependent thalassemia or&#xD;
      non-transfusion -dependent thalassemia as measured by preference questionnaire at Week 48&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This was an open-label, multicenter, single arm, phase II study aimed at collecting data on&#xD;
      preference for iron chelation therapy in patients with transfusion-dependent thalassemia&#xD;
      (TDT) or non-transfusion-dependent thalassemia (NTDT) throughout a 48 week treatment period.&#xD;
      Participants who were either chelator-naïve, or who were previously treated with iron&#xD;
      chelators (excluding deferasirox) for at least 6 months continuously, were eligible to&#xD;
      participate in this study.&#xD;
&#xD;
      The study was divided into 2 phases:&#xD;
&#xD;
        1. Core Phase:&#xD;
&#xD;
             -  Screening phase which lasted for a maximum of 4 weeks to determine patient&#xD;
                eligibility followed by&#xD;
&#xD;
             -  Period 1: Participants were treated with deferasirox DT from Baseline visit Day 1&#xD;
                to Week 24&#xD;
&#xD;
             -  Period 2: Participants were treated with deferasirox FCT from Week 25 to Week 48:&#xD;
&#xD;
           At the discretion of the investigator, patients could switch from deferasirox DT to&#xD;
           deferasirox FCT at any time during Period 1 of the Core phase, and vice versa, from&#xD;
           deferasirox FCT to deferasirox DT at any time during Period 2 of the Core phase.&#xD;
           Re-switching treatments was not allowed within each period.&#xD;
&#xD;
        2. Extension Phase:&#xD;
&#xD;
      Participants could continue deferasirox FCT formulation as per the judgment of the&#xD;
      investigator, through an extension phase for a maximum of 48 weeks months from the last dose&#xD;
      of deferasirox FCT received at the end of period 2 in the Core Phase or until one of the end&#xD;
      of study criteria defined is met, whichever came first. Participation in the extension phase&#xD;
      was optional.&#xD;
&#xD;
      The end of study was defined as the earliest occurrence of one of the following:&#xD;
&#xD;
        -  The patient reached Week 96 in the Extension phase.&#xD;
&#xD;
        -  Deferasirox FCT was locally reimbursed for this indication (only applicable for the&#xD;
           Extension phase)&#xD;
&#xD;
        -  Another clinical study or post-trial access program became available that could continue&#xD;
           to provide deferasirox FCT in this patient population and all patients ongoing were&#xD;
           eligible to be transferred to that clinical study.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">July 27, 2017</start_date>
  <completion_date type="Actual">March 11, 2021</completion_date>
  <primary_completion_date type="Actual">January 29, 2020</primary_completion_date>
  <phase>Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>N/A</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <primary_purpose>Other</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Number of Participants Preferring Deferasirox FCT or DT at Week 48 Based on Preference Questionnaire (Item 2)</measure>
    <time_frame>Week 48</time_frame>
    <description>Number of participants preferring deferasirox FCT or DT as measured by preference questionnaire (item 2) at Week 48. The preference questionnaire was a 3-item questionnaire. At Week 48, the second item of the preference questionnaire asked the patients (or parents of young patients from 2 to 9 years old) which medicine did the patient &quot;like best&quot;: &quot;Tablet to dissolve in liquid&quot; (=deferasirox DT), &quot;Film coated tablet&quot; (=deferasirox FCT), &quot;Sprinkle powder on food&quot; (=deferasirox FCT) and &quot;I don't know&quot; (=none of the above). The number of participants who selected each response option for item 2 was assessed. The analysis was performed for participants who answered the item 2 of the preference questionnaire.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Number of Participants Preferring Deferasirox FCT, Deferasirox DT or Previous Iron Chelation Therapy at Week 28 Based on Preference Questionnaire (Item 2)</measure>
    <time_frame>Week 28</time_frame>
    <description>Number of participants preferring deferasirox FCT, deferasirox DT or previous iron chelation therapy as measured by preference questionnaire (item 2) at Week 28. The preference questionnaire was a 3-item questionnaire. At Week 28, the second item of this questionnaire asked the patients (or parents of young patients from 2 to 9 years old) which medicine did the patient &quot;like best&quot;: &quot;Tablet to dissolve in liquid&quot; (=deferasirox DT), &quot;Film coated tablet (taken once a day)&quot; (=deferasirox FCT), &quot;Sprinkle powder on food&quot; (=deferasirox FCT), &quot;Tablet (taken 3 times a day)&quot; (=previous iron chelation therapy), &quot;Injection&quot; (=previous iron chelation therapy) and &quot;I don't know&quot; (=none of the above). The number of participants who selected each response option for item 2 was assessed. This analysis was performed only for patients who had received iron chelation therapy prior to enrolling in the study and who answered the item 2 of the preference questionnaire.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Number of Participants Preferring Deferasirox DT or Previous Iron Chelation Therapy at Week 4 and Week 24 Based on Preference Questionnaire (Item 2)</measure>
    <time_frame>Week 4 and Week 24</time_frame>
    <description>Number of participants preferring deferasirox DT or previous iron chelation therapy as measured by preference questionnaire (item 2) at Week 4 and 24. The preference questionnaire was a 3-item questionnaire. At Week 4 and 24, the second item of the preference questionnaire asked the patients (or parents of young patients from 2 to 9 years old) which medicine did the patient &quot;like best&quot;: &quot;Tablet to dissolve in liquid&quot; (=deferasirox DT), &quot;Tablet (taken 3 times a day)&quot; (=previous iron chelation therapy), &quot;Injection&quot; (=previous iron chelation therapy) and &quot;I don't know&quot; (=none of the above). The number of participants who selected each response option for item 2 was assessed. This analysis was performed only for patients who had received iron chelation therapy prior to enrolling in the study and who answered item 2 of the preference questionnaire.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Number of Participants Selecting Each Reason for Treatment Preference as Assessed by the Preference Questionnaire at Week 28 and Week 48</measure>
    <time_frame>Week 28 and Week 48</time_frame>
    <description>The preference questionnaire was a 3 item questionnaire. The first item asked the patients (or parents of young patients from 2 to 9 years old) which medicine they were taking. The second item asked which of the medicines did the patient &quot;Like best&quot;. Finally, the third item asked the patient why he/she preferred the medicine they chose in the second item. The number of participants who selected each response option for item 3 was assessed. Participants could select multiple reasons for treatment preference at each timepoint.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Percentage of Consumed Tablet Counts During Deferasirox DT and Deferasirox FCT Treatment Periods</measure>
    <time_frame>Deferasirox DT: From Baseline up to Week 24. Deferasirox FCT: From Week 25 up to Week 48</time_frame>
    <description>The percentage of consumed tablet counts (compliance) was calculated for each treatment period in the core phase: deferasirox DT (period 1) and deferasirox FCT (period 2). Compliance was defined as the total tablet count consumed divided by total tablet count prescribed and multiplied by 100. Total tablet count consumed was calculated as total number of tablets dispensed minus total number of tablets lost/wasted or returned. Total tablet count prescribed was calculated as the number of tablets that the patient should have taken during this period. If a patient did not return the study drug, the compliance was not calculated.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change Over Time in Aftertaste Score of Palatability Questionnaire</measure>
    <time_frame>Week 4, 24, 28 and 48</time_frame>
    <description>The palatability questionnaire consisted of 4 items, three items measuring taste and one item measuring aftertaste. The aftertaste item scored on a 5-point response scale with the response option: Very good = 1, Good = 2, Neither good nor bad = 3, Bad = 4, Very bad = 5. This item offered an additional response option of &quot;no aftertaste&quot;. The aftertaste score was calculated among participants who had an aftertaste. Higher aftertaste scores indicated a worse aftertaste.&#xD;
For participants less than (&lt;) 10 years old, an observer palatability questionnaire was administered. Items and scoring algorithm remained the same as for participants greater than or equal to (≥) 10 years old.&#xD;
Change in aftertaste score over time was assessed</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change Over Time in Palatability Score of Palatability Questionnaire</measure>
    <time_frame>Week 4, 24, 28 and 48</time_frame>
    <description>The palatability questionnaire consisted of 4 items, three items measuring taste and one item measuring aftertaste. Among the taste items, first one measured taste on a 5-point response scale. The other two items measured what happened after taking the medicine and how the perceived amount of liquid taken with the medicine was. Responses to these 3 items were combined and converted into a single palatability score using a scoring matrix: each combination of responses on each of 3 items corresponded to a predefined palatability score. E.g. if a participant responded &quot;bad&quot; to item 1, &quot;vomited &lt;30min&quot; to item 2 and &quot;not enough&quot; to item 3, then the palatability score assigned was 0. This score ranged from 0 to 11; higher scores indicated better palatability. For participants &lt;10 years old, an observer palatability questionnaire was administered. Items and scoring algorithm were the same as for participants ≥10 years old. Change in palatability score over time was assessed</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline in Adherence Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire</measure>
    <time_frame>Baseline (week 2 or, if missing, week 3), week 24, 28 and 48</time_frame>
    <description>The mSICT patient reported outcome (PRO) consisted of 15 items that represented 3 domains: Adherence, Preference, and Concerns. The adherence domain score consisted of 6 adherence items, measured using a 5-point response scale. The adherence score was calculated by summing these 6 items, with scores ranging from 6 to 30. Higher scores indicated worse adherence.&#xD;
For participants &lt;10 years old, an observer version (ObsRO) was administered. The adherence score remained the same as for participants ≥10 years old.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline in Preference Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire</measure>
    <time_frame>Baseline (week 2 or, if missing, week 3), week 24, 28 and 48</time_frame>
    <description>The mSICT patient reported outcome (PRO) consisted of 15 items that represented 3 domains: Adherence, Preference, and Concerns.&#xD;
The preference/satisfaction domain score consisted of 2 preference/satisfaction items, measured using a 5-point response scale. The preference score was calculated by summing these 2 items, with scores ranging from 2 to 10. Higher scores indicated worse satisfaction.&#xD;
For participants &lt; 10 years old, an observer version (ObsRO) was administered. Preference score remained the same as for participants ≥ 10 years old.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline in Concerns Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire</measure>
    <time_frame>Baseline (week 2 or, if missing, week 3), week 24, 28 and 48</time_frame>
    <description>The mSICT patient reported outcome (PRO) consisted of 15 items that represented 3 domains: Adherence, Preference, and Concerns.&#xD;
The concerns domain score consisted of 3 items to address any concerns or worries with the medication. All 3 items were measured on a 5-point response scale. The concerns score was calculated by summing the 3 items, with scores ranging from 3 to 15. Higher scores indicated fewer concerns. For participants &lt; 10 years old, an observer version (ObsRO) was administered. Concerns score remained the same as for participants ≥ 10 years old.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline in Gastrointestinal (GI) Symptom Score Based on GI Questionnaire</measure>
    <time_frame>Baseline (week -1 or, if missing, week -2), week 24, 28 and 48</time_frame>
    <description>The GI symptom score was calculated from responses to 5 questions of the GI questionnaire, each with a possible score of 1 to 5, for an overall possible score range of 5 to 25, where a lower score represents a less severe GI symptom and a higher score represents a more severe GI symptom.&#xD;
An observer GI symptom questionnaire was administered to those patients who were &lt; 10 years old. The questionnaire was completed by the parents of the participants. All items and the scoring algorithm remained the same as for participants ≥ 10 years old.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline in Serum Ferritin Levels</measure>
    <time_frame>From Baseline (Day 1) up to 96 weeks</time_frame>
    <description>Absolute change from baseline over time in serum ferritin levels</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">148</enrollment>
  <condition>Transfusion-dependent Thalassemia</condition>
  <condition>Non-transfusion-dependent Thalassemia</condition>
  <arm_group>
    <arm_group_label>Deferasirox DT followed by deferasirox FCT</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Deferasirox dispersable tablet (DT)</intervention_name>
    <description>Deferasirox DT was provided as 125 mg, 250 mg, 500 mg dispersible tablets for oral use. The strengths provided in an individual country could differ and reflected the strengths available commercially in each country.&#xD;
For iron chelation naive participants, the starting dose on Baseline Day 1 was 20 mg/kg/day in TDT and 10 mg/kg/day in NTDT.&#xD;
For iron chelation (deferoxamine and/or deferiprone) pre-treated participants, the starting dose was equivalent to the dose of deferoxamine received (for participants pre-treated with deferoxamine) and based on their serum ferritin levels (for participants pre-treated with deferiprone).&#xD;
Participants took deferasirox DT once daily for 24 weeks (core phase). The required number of deferasirox DT tablets were to be dispersed with gentle stirring in a glass of water.</description>
    <arm_group_label>Deferasirox DT followed by deferasirox FCT</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Deferasirox film coated tablet (FCT)</intervention_name>
    <description>Deferasirox FCT was provided as 90 mg, 180 mg, 360 mg film coated tablets for oral use. The FCT starting dose on Week 25 was 14 mg/kg/day in TDT and 7 mg/kg/day in NTDT.&#xD;
Participants took deferasirox FCT once daily for 24 weeks during the core phase and up to 48 weeks during the extension phase. For patients with difficulties in swallowing deferasirox FCT, it was allowed to crush the film-coated tablets and administer the study drug by sprinkling the full dose on soft food (like yogurt or apple puree).</description>
    <arm_group_label>Deferasirox DT followed by deferasirox FCT</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Prior to any screening procedures were performed, written informed consent/assent must&#xD;
             be provided. For pediatric patients, consent was obtained from parent(s) or legal&#xD;
             patient's representative. Investigators also obtained assent of patients according to&#xD;
             local, regional or national guidelines.&#xD;
&#xD;
          2. Male and female patient aged ≥ 2 years&#xD;
&#xD;
          3. Deferasirox naïve patient or chelated naive patient or treated by other chelators for&#xD;
             at least 6 months, such as:&#xD;
&#xD;
               1. Deferiprone/ DFP&#xD;
&#xD;
               2. Deferoxamine /DFO&#xD;
&#xD;
               3. Combination (DFO + DFP)&#xD;
&#xD;
          4. Subject was willing to discontinue current iron chelation therapy at least 5 days&#xD;
             prior to study day 1 and for the duration of the study&#xD;
&#xD;
          5. Patients with transfusion-dependent thalassemia (independent of underlying condition)&#xD;
             with transfusional iron overload as shown by a serum ferritin level of &gt; 1000 ng/ml at&#xD;
             screening and if available, LIC &gt; 3 mg Fe/g dw within 6 months prior to screening&#xD;
&#xD;
          6. Patients with non-transfusion-dependent thalassemia with iron overload as shown by a&#xD;
             serum ferritin level of ≥ 800 ng/ml at screening and if available, LIC ≥ 5 mg Fe/g dw&#xD;
             within 6 months prior to screening&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Creatinine clearance below the contraindication limit in the locally approved&#xD;
             prescribing information.&#xD;
&#xD;
          2. Serum creatinine level &gt; 1.5 x ULN (upper limit of normal)&#xD;
&#xD;
          3. AST (SGOT) /ALT (SGPT) &gt; 5 x ULN, unless LIC confirmed as &gt;10 mg Fe/dw within 6 months&#xD;
             prior to screening visit.&#xD;
&#xD;
          4. Significant proteinuria as indicated by a urinary protein/creatinine ratio &gt; 0.5 mg/mg&#xD;
             in a non-first void urine sample.&#xD;
&#xD;
          5. Patients with significant impaired gastrointestinal (GI) function or GI disease that&#xD;
             might significantly alter the absorption of oral deferasirox (e.g. ulcerative&#xD;
             diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, or small&#xD;
             bowel resection).&#xD;
&#xD;
          6. Clinical or laboratory evidence of active Hepatitis B or Hepatitis C (HBsAg in the&#xD;
             absence of HBsAb OR HCV Ab positive with HCV RNA positive).&#xD;
&#xD;
          7. Patients with psychiatric or addictive disorders which prevent them from giving their&#xD;
             informed consent or undergoing any of the treatment options or patients unwilling or&#xD;
             unable to comply with the protocol&#xD;
&#xD;
          8. Patients with a known history of HIV seropositivity (Elisa or Western blot).&#xD;
&#xD;
          9. History of malignancy of any organ system, treated or untreated, within the past 5&#xD;
             years whether or not there is evidence of local recurrence or metastases, with the&#xD;
             exception of localized basal cell carcinoma of the skin.&#xD;
&#xD;
         10. Patients participating in another clinical trial or receiving an investigational drug.&#xD;
             Patients who have recently completed treatment with an investigational product must&#xD;
             have ceased this treatment for at least five times the half-life of the&#xD;
             investigational product.&#xD;
&#xD;
         11. History of hypersensitivity to any of the study drug or excipients.&#xD;
&#xD;
         12. Significant medical condition interfering with the ability to partake in this study&#xD;
             (e.g. systemic uncontrolled hypertension, unstable cardiac disease not controlled by&#xD;
             standard medical therapy, systemic disease (cardiovascular, renal, hepatic, etc.).&#xD;
&#xD;
         13. Women of child-bearing potential, defined as all women physiologically capable of&#xD;
             becoming pregnant, unless they were using effective methods of contraception during&#xD;
             dosing of study treatment&#xD;
&#xD;
         14. Women were considered post-menopausal and not of child bearing potential if they had&#xD;
             had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical profile&#xD;
             (e.g. age appropriate, history of vasomotor symptoms) or had had surgical bilateral&#xD;
             oophorectomy (with or without hysterectomy) or tubal ligation at least six weeks ago.&#xD;
             In the case of oophorectomy alone, only when the reproductive status of the woman had&#xD;
             been confirmed by follow up hormone level assessment is she considered not of child&#xD;
             bearing potential.&#xD;
&#xD;
         15. Sexually active males unless they used a condom during intercourse while taking drug&#xD;
             and for 28 days after stopping study medication and should not father a child in this&#xD;
             period. A condom was required to be used also by vasectomized men in order to prevent&#xD;
             delivery of the drug via seminal fluid.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>2 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <location>
    <facility>
      <name>Novartis Investigative Site</name>
      <address>
        <city>Alexandria</city>
        <zip>21131</zip>
        <country>Egypt</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Novartis Investigative Site</name>
      <address>
        <city>Cairo</city>
        <zip>11562</zip>
        <country>Egypt</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Novartis Investigative Site</name>
      <address>
        <city>Zagazig</city>
        <zip>44519</zip>
        <country>Egypt</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Novartis Investigative Site</name>
      <address>
        <city>Hazmiyeh</city>
        <state>Beirut</state>
        <zip>PO BOX 213</zip>
        <country>Lebanon</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Novartis Investigative Site</name>
      <address>
        <city>Rabat</city>
        <zip>10102</zip>
        <country>Morocco</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Novartis Investigative Site</name>
      <address>
        <city>Al Ahsa</city>
        <state>SAU</state>
        <country>Saudi Arabia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Novartis Investigative Site</name>
      <address>
        <city>Jeddah</city>
        <zip>21159</zip>
        <country>Saudi Arabia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Novartis Investigative Site</name>
      <address>
        <city>Jeddah</city>
        <zip>21589</zip>
        <country>Saudi Arabia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Novartis Investigative Site</name>
      <address>
        <city>Riyadh</city>
        <zip>11117</zip>
        <country>Saudi Arabia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Novartis Investigative Site</name>
      <address>
        <city>Bangkok noi</city>
        <state>Bangkok</state>
        <zip>10700</zip>
        <country>Thailand</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Novartis Investigative Site</name>
      <address>
        <city>Bangkoknoi</city>
        <state>Bangkok</state>
        <zip>10700</zip>
        <country>Thailand</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Novartis Investigative Site</name>
      <address>
        <city>Bangkok</city>
        <zip>10400</zip>
        <country>Thailand</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Novartis Investigative Site</name>
      <address>
        <city>Ankara</city>
        <zip>06100</zip>
        <country>Turkey</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Novartis Investigative Site</name>
      <address>
        <city>Antalya</city>
        <zip>07070</zip>
        <country>Turkey</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Novartis Investigative Site</name>
      <address>
        <city>Istanbul</city>
        <zip>34093</zip>
        <country>Turkey</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Novartis Investigative Site</name>
      <address>
        <city>Hanoi</city>
        <country>Vietnam</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Novartis Investigative Site</name>
      <address>
        <city>Ho Chi Minh City</city>
        <zip>DISTRICT 1</zip>
        <country>Vietnam</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Egypt</country>
    <country>Lebanon</country>
    <country>Morocco</country>
    <country>Saudi Arabia</country>
    <country>Thailand</country>
    <country>Turkey</country>
    <country>Vietnam</country>
  </location_countries>
  <verification_date>September 2021</verification_date>
  <study_first_submitted>November 8, 2016</study_first_submitted>
  <study_first_submitted_qc>December 12, 2016</study_first_submitted_qc>
  <study_first_posted type="Estimate">December 15, 2016</study_first_posted>
  <results_first_submitted>September 6, 2021</results_first_submitted>
  <results_first_submitted_qc>September 6, 2021</results_first_submitted_qc>
  <results_first_posted type="Actual">October 4, 2021</results_first_posted>
  <last_update_submitted>September 6, 2021</last_update_submitted>
  <last_update_submitted_qc>September 6, 2021</last_update_submitted_qc>
  <last_update_posted type="Actual">October 4, 2021</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>thalassemia</keyword>
  <keyword>TDT</keyword>
  <keyword>transfusion-dependent</keyword>
  <keyword>non-transfusion-dependent</keyword>
  <keyword>NTDT</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Thalassemia</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Deferasirox</mesh_term>
  </intervention_browse>
  <patient_data>
    <sharing_ipd>Yes</sharing_ipd>
    <ipd_description>Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.&#xD;
This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com</ipd_description>
  </patient_data>
  <provided_document_section>
    <provided_document>
      <document_type>Study Protocol</document_type>
      <document_has_protocol>Yes</document_has_protocol>
      <document_has_icf>No</document_has_icf>
      <document_has_sap>No</document_has_sap>
      <document_date>February 12, 2019</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/24/NCT02993224/Prot_000.pdf</document_url>
    </provided_document>
    <provided_document>
      <document_type>Statistical Analysis Plan</document_type>
      <document_has_protocol>No</document_has_protocol>
      <document_has_icf>No</document_has_icf>
      <document_has_sap>Yes</document_has_sap>
      <document_date>April 8, 2021</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/24/NCT02993224/SAP_001.pdf</document_url>
    </provided_document>
  </provided_document_section>

  <clinical_results>

    <participant_flow>
      <recruitment_details>Participants took part in 17 investigative sites in 7 countries</recruitment_details>
      <group_list>
        <group group_id="P1">
          <title>Deferasirox DT Followed by Deferasirox FCT</title>
          <description>Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT</description>
        </group>
      </group_list>
      <period_list>
        <period>
          <title>Core Phase - Period 1</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="148"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="140"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="8"/>
              </participants_list>
            </milestone>
          </milestone_list>
          <drop_withdraw_reason_list>
            <drop_withdraw_reason>
              <title>Adverse Event</title>
              <participants_list>
                <participants group_id="P1" count="4"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Withdrawal by Subject</title>
              <participants_list>
                <participants group_id="P1" count="1"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Lost to Follow-up</title>
              <participants_list>
                <participants group_id="P1" count="3"/>
              </participants_list>
            </drop_withdraw_reason>
          </drop_withdraw_reason_list>
        </period>
        <period>
          <title>Core Phase- Period 2</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="140"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="136"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="4"/>
              </participants_list>
            </milestone>
          </milestone_list>
          <drop_withdraw_reason_list>
            <drop_withdraw_reason>
              <title>Adverse Event</title>
              <participants_list>
                <participants group_id="P1" count="1"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Unwillingness To Comply With Protocol Procedures And Prescribed Study Treatment</title>
              <participants_list>
                <participants group_id="P1" count="2"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Allogenic stem cell transplantation</title>
              <participants_list>
                <participants group_id="P1" count="1"/>
              </participants_list>
            </drop_withdraw_reason>
          </drop_withdraw_reason_list>
        </period>
        <period>
          <title>Extension Phase</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="116"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="80"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="36"/>
              </participants_list>
            </milestone>
          </milestone_list>
          <drop_withdraw_reason_list>
            <drop_withdraw_reason>
              <title>Unwillingness To Comply With Protocol Procedures And Prescribed Study Treatment</title>
              <participants_list>
                <participants group_id="P1" count="1"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Adverse Event</title>
              <participants_list>
                <participants group_id="P1" count="3"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Withdrawal by Subject</title>
              <participants_list>
                <participants group_id="P1" count="3"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Lost to Follow-up</title>
              <participants_list>
                <participants group_id="P1" count="2"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Deferasirox FCT locally reimbursed</title>
              <participants_list>
                <participants group_id="P1" count="27"/>
              </participants_list>
            </drop_withdraw_reason>
          </drop_withdraw_reason_list>
        </period>
      </period_list>
    </participant_flow>

    <baseline>
      <group_list>
        <group group_id="B1">
          <title>Deferasirox DT Followed by Deferasirox FCT</title>
          <description>Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT</description>
        </group>
      </group_list>
      <analyzed_list>
        <analyzed>
          <units>Participants</units>
          <scope>Overall</scope>
          <count_list>
            <count group_id="B1" value="148"/>
          </count_list>
        </analyzed>
      </analyzed_list>
      <measure_list>
        <measure>
          <title>Age</title>
          <units>Years</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="15.32" spread="13.824"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Sex: Female, Male</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Female</title>
                  <measurement_list>
                    <measurement group_id="B1" value="82"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Male</title>
                  <measurement_list>
                    <measurement group_id="B1" value="66"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Race/Ethnicity, Customized</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Indian (Indian subcontinent)</title>
                  <measurement_list>
                    <measurement group_id="B1" value="5"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Other</title>
                  <measurement_list>
                    <measurement group_id="B1" value="143"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </measure_list>
    </baseline>

    <outcome_list>
      <outcome>
        <type>Primary</type>
        <title>Number of Participants Preferring Deferasirox FCT or DT at Week 48 Based on Preference Questionnaire (Item 2)</title>
        <description>Number of participants preferring deferasirox FCT or DT as measured by preference questionnaire (item 2) at Week 48. The preference questionnaire was a 3-item questionnaire. At Week 48, the second item of the preference questionnaire asked the patients (or parents of young patients from 2 to 9 years old) which medicine did the patient &quot;like best&quot;: &quot;Tablet to dissolve in liquid&quot; (=deferasirox DT), &quot;Film coated tablet&quot; (=deferasirox FCT), &quot;Sprinkle powder on food&quot; (=deferasirox FCT) and &quot;I don't know&quot; (=none of the above). The number of participants who selected each response option for item 2 was assessed. The analysis was performed for participants who answered the item 2 of the preference questionnaire.</description>
        <time_frame>Week 48</time_frame>
        <population>Full Analysis Set (FAS): Participants to whom study treatment was assigned and who received at least one dose of each study treatment (DT and FCT). Only participants with available data for this outcome measure were included in this analysis.</population>
        <group_list>
          <group group_id="O1">
            <title>Deferasirox DT Followed by Deferasirox FCT</title>
            <description>Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT</description>
          </group>
        </group_list>
        <measure>
          <title>Number of Participants Preferring Deferasirox FCT or DT at Week 48 Based on Preference Questionnaire (Item 2)</title>
          <description>Number of participants preferring deferasirox FCT or DT as measured by preference questionnaire (item 2) at Week 48. The preference questionnaire was a 3-item questionnaire. At Week 48, the second item of the preference questionnaire asked the patients (or parents of young patients from 2 to 9 years old) which medicine did the patient &quot;like best&quot;: &quot;Tablet to dissolve in liquid&quot; (=deferasirox DT), &quot;Film coated tablet&quot; (=deferasirox FCT), &quot;Sprinkle powder on food&quot; (=deferasirox FCT) and &quot;I don't know&quot; (=none of the above). The number of participants who selected each response option for item 2 was assessed. The analysis was performed for participants who answered the item 2 of the preference questionnaire.</description>
          <population>Full Analysis Set (FAS): Participants to whom study treatment was assigned and who received at least one dose of each study treatment (DT and FCT). Only participants with available data for this outcome measure were included in this analysis.</population>
          <units>Participants</units>
          <param>Count of Participants</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="134"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Preference for deferasirox DT</title>
                  <measurement_list>
                    <measurement group_id="O1" value="10"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Preference for deferasirox FCT</title>
                  <measurement_list>
                    <measurement group_id="O1" value="121"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Preference for none of the above</title>
                  <measurement_list>
                    <measurement group_id="O1" value="3"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
            </group_id_list>
            <groups_desc>Preference for deferasirox DT vs deferasirox FCT</groups_desc>
            <non_inferiority_type>Superiority</non_inferiority_type>
            <p_value>&lt;.0001</p_value>
            <method>McNemar</method>
            <param_type>Difference of proportion</param_type>
            <param_value>0.83</param_value>
            <ci_percent>95</ci_percent>
            <ci_n_sides>2-Sided</ci_n_sides>
            <ci_lower_limit>0.75</ci_lower_limit>
            <ci_upper_limit>0.89</ci_upper_limit>
          </analysis>
        </analysis_list>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Number of Participants Preferring Deferasirox FCT, Deferasirox DT or Previous Iron Chelation Therapy at Week 28 Based on Preference Questionnaire (Item 2)</title>
        <description>Number of participants preferring deferasirox FCT, deferasirox DT or previous iron chelation therapy as measured by preference questionnaire (item 2) at Week 28. The preference questionnaire was a 3-item questionnaire. At Week 28, the second item of this questionnaire asked the patients (or parents of young patients from 2 to 9 years old) which medicine did the patient &quot;like best&quot;: &quot;Tablet to dissolve in liquid&quot; (=deferasirox DT), &quot;Film coated tablet (taken once a day)&quot; (=deferasirox FCT), &quot;Sprinkle powder on food&quot; (=deferasirox FCT), &quot;Tablet (taken 3 times a day)&quot; (=previous iron chelation therapy), &quot;Injection&quot; (=previous iron chelation therapy) and &quot;I don't know&quot; (=none of the above). The number of participants who selected each response option for item 2 was assessed. This analysis was performed only for patients who had received iron chelation therapy prior to enrolling in the study and who answered the item 2 of the preference questionnaire.</description>
        <time_frame>Week 28</time_frame>
        <population>Participants in the FAS [to whom study treatment was assigned and who received at least one dose of each study treatment (DT and FCT)] and who received iron chelation therapy prior to enrolling in the study. Only participants with available data for this outcome measure were included in this analysis.</population>
        <group_list>
          <group group_id="O1">
            <title>Deferasirox DT Followed by Deferasirox FCT</title>
            <description>Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT</description>
          </group>
        </group_list>
        <measure>
          <title>Number of Participants Preferring Deferasirox FCT, Deferasirox DT or Previous Iron Chelation Therapy at Week 28 Based on Preference Questionnaire (Item 2)</title>
          <description>Number of participants preferring deferasirox FCT, deferasirox DT or previous iron chelation therapy as measured by preference questionnaire (item 2) at Week 28. The preference questionnaire was a 3-item questionnaire. At Week 28, the second item of this questionnaire asked the patients (or parents of young patients from 2 to 9 years old) which medicine did the patient &quot;like best&quot;: &quot;Tablet to dissolve in liquid&quot; (=deferasirox DT), &quot;Film coated tablet (taken once a day)&quot; (=deferasirox FCT), &quot;Sprinkle powder on food&quot; (=deferasirox FCT), &quot;Tablet (taken 3 times a day)&quot; (=previous iron chelation therapy), &quot;Injection&quot; (=previous iron chelation therapy) and &quot;I don't know&quot; (=none of the above). The number of participants who selected each response option for item 2 was assessed. This analysis was performed only for patients who had received iron chelation therapy prior to enrolling in the study and who answered the item 2 of the preference questionnaire.</description>
          <population>Participants in the FAS [to whom study treatment was assigned and who received at least one dose of each study treatment (DT and FCT)] and who received iron chelation therapy prior to enrolling in the study. Only participants with available data for this outcome measure were included in this analysis.</population>
          <units>Participants</units>
          <param>Count of Participants</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="69"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Preference for deferasirox DT</title>
                  <measurement_list>
                    <measurement group_id="O1" value="6"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Preference for deferasirox FCT</title>
                  <measurement_list>
                    <measurement group_id="O1" value="60"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Preference for previous iron chelation therapy</title>
                  <measurement_list>
                    <measurement group_id="O1" value="3"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Preference for none of the above</title>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
            </group_id_list>
            <groups_desc>Preference of deferasirox FCT vs deferasirox DT</groups_desc>
            <non_inferiority_type>Superiority</non_inferiority_type>
            <p_value>&lt;0.0001</p_value>
            <method>McNemar</method>
            <param_type>Difference of proportion</param_type>
            <param_value>0.78</param_value>
            <ci_percent>95</ci_percent>
            <ci_n_sides>2-Sided</ci_n_sides>
            <ci_lower_limit>0.65</ci_lower_limit>
            <ci_upper_limit>0.88</ci_upper_limit>
          </analysis>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
            </group_id_list>
            <groups_desc>Preference for deferasirox FCT vs previous iron chelation therapy</groups_desc>
            <non_inferiority_type>Superiority</non_inferiority_type>
            <p_value>&lt;0.0001</p_value>
            <method>McNemar</method>
            <param_type>Difference of proportion</param_type>
            <param_value>0.83</param_value>
            <ci_percent>95</ci_percent>
            <ci_n_sides>2-Sided</ci_n_sides>
            <ci_lower_limit>0.71</ci_lower_limit>
            <ci_upper_limit>0.91</ci_upper_limit>
          </analysis>
        </analysis_list>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Number of Participants Preferring Deferasirox DT or Previous Iron Chelation Therapy at Week 4 and Week 24 Based on Preference Questionnaire (Item 2)</title>
        <description>Number of participants preferring deferasirox DT or previous iron chelation therapy as measured by preference questionnaire (item 2) at Week 4 and 24. The preference questionnaire was a 3-item questionnaire. At Week 4 and 24, the second item of the preference questionnaire asked the patients (or parents of young patients from 2 to 9 years old) which medicine did the patient &quot;like best&quot;: &quot;Tablet to dissolve in liquid&quot; (=deferasirox DT), &quot;Tablet (taken 3 times a day)&quot; (=previous iron chelation therapy), &quot;Injection&quot; (=previous iron chelation therapy) and &quot;I don't know&quot; (=none of the above). The number of participants who selected each response option for item 2 was assessed. This analysis was performed only for patients who had received iron chelation therapy prior to enrolling in the study and who answered item 2 of the preference questionnaire.</description>
        <time_frame>Week 4 and Week 24</time_frame>
        <population>Participants in the FAS [to whom study treatment was assigned and who received at least one dose of each study treatment (DT and FCT)] and who received iron chelation therapy prior to enrolling in the study. Number analyzed signified number of participants with available data for this outcome measure at specified timepoints.</population>
        <group_list>
          <group group_id="O1">
            <title>Deferasirox DT Followed by Deferasirox FCT</title>
            <description>Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT</description>
          </group>
        </group_list>
        <measure>
          <title>Number of Participants Preferring Deferasirox DT or Previous Iron Chelation Therapy at Week 4 and Week 24 Based on Preference Questionnaire (Item 2)</title>
          <description>Number of participants preferring deferasirox DT or previous iron chelation therapy as measured by preference questionnaire (item 2) at Week 4 and 24. The preference questionnaire was a 3-item questionnaire. At Week 4 and 24, the second item of the preference questionnaire asked the patients (or parents of young patients from 2 to 9 years old) which medicine did the patient &quot;like best&quot;: &quot;Tablet to dissolve in liquid&quot; (=deferasirox DT), &quot;Tablet (taken 3 times a day)&quot; (=previous iron chelation therapy), &quot;Injection&quot; (=previous iron chelation therapy) and &quot;I don't know&quot; (=none of the above). The number of participants who selected each response option for item 2 was assessed. This analysis was performed only for patients who had received iron chelation therapy prior to enrolling in the study and who answered item 2 of the preference questionnaire.</description>
          <population>Participants in the FAS [to whom study treatment was assigned and who received at least one dose of each study treatment (DT and FCT)] and who received iron chelation therapy prior to enrolling in the study. Number analyzed signified number of participants with available data for this outcome measure at specified timepoints.</population>
          <units>Participants</units>
          <param>Count of Participants</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="70"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Week 4</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="70"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <title>Preference for deferasirox DT</title>
                  <measurement_list>
                    <measurement group_id="O1" value="57"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Preference for previous iron chelation therapy</title>
                  <measurement_list>
                    <measurement group_id="O1" value="11"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Preference for none of the above</title>
                  <measurement_list>
                    <measurement group_id="O1" value="2"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 24</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="69"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <title>Preference for deferasirox DT</title>
                  <measurement_list>
                    <measurement group_id="O1" value="52"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Preference for previous iron chelation therapy</title>
                  <measurement_list>
                    <measurement group_id="O1" value="11"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Preference for none of the above</title>
                  <measurement_list>
                    <measurement group_id="O1" value="6"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
            </group_id_list>
            <groups_desc>Deferasirox DT vs previous iron chelation therapy at Week 4</groups_desc>
            <non_inferiority_type>Superiority</non_inferiority_type>
            <p_value>&lt; 0.0001</p_value>
            <method>McNemar</method>
            <param_type>Difference of proportion</param_type>
            <param_value>0.66</param_value>
            <ci_percent>95</ci_percent>
            <ci_n_sides>2-Sided</ci_n_sides>
            <ci_lower_limit>0.51</ci_lower_limit>
            <ci_upper_limit>0.77</ci_upper_limit>
          </analysis>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
            </group_id_list>
            <groups_desc>Deferasirox DT vs previous iron chelation therapy at Week 24</groups_desc>
            <non_inferiority_type>Superiority</non_inferiority_type>
            <p_value>&lt; 0.0001</p_value>
            <method>McNemar</method>
            <param_type>Difference of proportion</param_type>
            <param_value>0.59</param_value>
            <ci_percent>95</ci_percent>
            <ci_n_sides>2-Sided</ci_n_sides>
            <ci_lower_limit>0.44</ci_lower_limit>
            <ci_upper_limit>0.72</ci_upper_limit>
          </analysis>
        </analysis_list>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Number of Participants Selecting Each Reason for Treatment Preference as Assessed by the Preference Questionnaire at Week 28 and Week 48</title>
        <description>The preference questionnaire was a 3 item questionnaire. The first item asked the patients (or parents of young patients from 2 to 9 years old) which medicine they were taking. The second item asked which of the medicines did the patient &quot;Like best&quot;. Finally, the third item asked the patient why he/she preferred the medicine they chose in the second item. The number of participants who selected each response option for item 3 was assessed. Participants could select multiple reasons for treatment preference at each timepoint.</description>
        <time_frame>Week 28 and Week 48</time_frame>
        <population>FAS: Participants to whom study treatment was assigned and who received at least one dose of each study treatment (DT and FCT).</population>
        <group_list>
          <group group_id="O1">
            <title>Deferasirox DT Followed by Deferasirox FCT</title>
            <description>Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT</description>
          </group>
        </group_list>
        <measure>
          <title>Number of Participants Selecting Each Reason for Treatment Preference as Assessed by the Preference Questionnaire at Week 28 and Week 48</title>
          <description>The preference questionnaire was a 3 item questionnaire. The first item asked the patients (or parents of young patients from 2 to 9 years old) which medicine they were taking. The second item asked which of the medicines did the patient &quot;Like best&quot;. Finally, the third item asked the patient why he/she preferred the medicine they chose in the second item. The number of participants who selected each response option for item 3 was assessed. Participants could select multiple reasons for treatment preference at each timepoint.</description>
          <population>FAS: Participants to whom study treatment was assigned and who received at least one dose of each study treatment (DT and FCT).</population>
          <units>Participants</units>
          <param>Count of Participants</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="139"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Aftertaste (Week 28)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="9"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Can correctly prepare the medicine (Week 28)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="35"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Convenience (it's not a problem to take your medicine) (Week 28)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="109"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Easier to remember to take the medicine (Week 28)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="57"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Gain my personal time with family and friends (Week 28)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="35"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>No/ less pain on the injection site (Week 28)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="31"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>No/ less side effects (Week 28)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="50"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Number of pills (Week 28)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="46"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Number of times you have to take the medicine (Week 28)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="42"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Other (Week 28)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="5"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Taste (Week 28)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="30"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Aftertaste (Week 48)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="17"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Can correctly prepare the medicine (Week 48)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="48"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Convenience (it's not a problem to take your medicine) (Week 48)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="103"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Easier to remember to take the medicine (Week 48)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="61"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Gain my personal time with family and friends (Week 48)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="36"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>No/ less pain on the injection site (Week 48)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="37"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>No/ less side effects (Week 48)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="48"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Number of pills (Week 48)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="41"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Number of times you have to take the medicine (Week 48)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="47"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Other (Week 48)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Taste (Week 48)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="51"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Percentage of Consumed Tablet Counts During Deferasirox DT and Deferasirox FCT Treatment Periods</title>
        <description>The percentage of consumed tablet counts (compliance) was calculated for each treatment period in the core phase: deferasirox DT (period 1) and deferasirox FCT (period 2). Compliance was defined as the total tablet count consumed divided by total tablet count prescribed and multiplied by 100. Total tablet count consumed was calculated as total number of tablets dispensed minus total number of tablets lost/wasted or returned. Total tablet count prescribed was calculated as the number of tablets that the patient should have taken during this period. If a patient did not return the study drug, the compliance was not calculated.</description>
        <time_frame>Deferasirox DT: From Baseline up to Week 24. Deferasirox FCT: From Week 25 up to Week 48</time_frame>
        <population>FAS: Participants to whom study treatment was assigned and who received at least one dose of each study treatment (DT and FCT). Number analyzed signified number of participants with available data for this outcome measure for each treatment period</population>
        <group_list>
          <group group_id="O1">
            <title>Deferasirox DT Followed by Deferasirox FCT</title>
            <description>Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT</description>
          </group>
        </group_list>
        <measure>
          <title>Percentage of Consumed Tablet Counts During Deferasirox DT and Deferasirox FCT Treatment Periods</title>
          <description>The percentage of consumed tablet counts (compliance) was calculated for each treatment period in the core phase: deferasirox DT (period 1) and deferasirox FCT (period 2). Compliance was defined as the total tablet count consumed divided by total tablet count prescribed and multiplied by 100. Total tablet count consumed was calculated as total number of tablets dispensed minus total number of tablets lost/wasted or returned. Total tablet count prescribed was calculated as the number of tablets that the patient should have taken during this period. If a patient did not return the study drug, the compliance was not calculated.</description>
          <population>FAS: Participants to whom study treatment was assigned and who received at least one dose of each study treatment (DT and FCT). Number analyzed signified number of participants with available data for this outcome measure for each treatment period</population>
          <units>Percentage of tablet counts</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="139"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Deferasirox DT (Baseline up to Week 24)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="139"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="98.68" spread="22.373"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Deferasirox FCT (From Week 25 up to Week 48)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="138"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="95.07" spread="15.368"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
            </group_id_list>
            <groups_desc>Compliance of deferasirox DT vs deferasirox FCT</groups_desc>
            <non_inferiority_type>Superiority</non_inferiority_type>
            <p_value>0.1191</p_value>
            <method>ANCOVA</method>
            <param_type>Least squares mean</param_type>
            <param_value>-3.6</param_value>
            <dispersion_type>Standard Error of the Mean</dispersion_type>
            <dispersion_value>2.30</dispersion_value>
            <ci_percent>95</ci_percent>
            <ci_n_sides>2-Sided</ci_n_sides>
            <ci_lower_limit>-8.1</ci_lower_limit>
            <ci_upper_limit>0.9</ci_upper_limit>
          </analysis>
        </analysis_list>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change Over Time in Aftertaste Score of Palatability Questionnaire</title>
        <description>The palatability questionnaire consisted of 4 items, three items measuring taste and one item measuring aftertaste. The aftertaste item scored on a 5-point response scale with the response option: Very good = 1, Good = 2, Neither good nor bad = 3, Bad = 4, Very bad = 5. This item offered an additional response option of &quot;no aftertaste&quot;. The aftertaste score was calculated among participants who had an aftertaste. Higher aftertaste scores indicated a worse aftertaste.&#xD;
For participants less than (&lt;) 10 years old, an observer palatability questionnaire was administered. Items and scoring algorithm remained the same as for participants greater than or equal to (≥) 10 years old.&#xD;
Change in aftertaste score over time was assessed</description>
        <time_frame>Week 4, 24, 28 and 48</time_frame>
        <population>FAS: Participants to whom study treatment was assigned and who received at least one dose of each study treatment (DT and FCT). Number analyzed signified number of participants with available data for this outcome measure at specified timepoints.</population>
        <group_list>
          <group group_id="O1">
            <title>Deferasirox DT Followed by Deferasirox FCT</title>
            <description>Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT</description>
          </group>
        </group_list>
        <measure>
          <title>Change Over Time in Aftertaste Score of Palatability Questionnaire</title>
          <description>The palatability questionnaire consisted of 4 items, three items measuring taste and one item measuring aftertaste. The aftertaste item scored on a 5-point response scale with the response option: Very good = 1, Good = 2, Neither good nor bad = 3, Bad = 4, Very bad = 5. This item offered an additional response option of &quot;no aftertaste&quot;. The aftertaste score was calculated among participants who had an aftertaste. Higher aftertaste scores indicated a worse aftertaste.&#xD;
For participants less than (&lt;) 10 years old, an observer palatability questionnaire was administered. Items and scoring algorithm remained the same as for participants greater than or equal to (≥) 10 years old.&#xD;
Change in aftertaste score over time was assessed</description>
          <population>FAS: Participants to whom study treatment was assigned and who received at least one dose of each study treatment (DT and FCT). Number analyzed signified number of participants with available data for this outcome measure at specified timepoints.</population>
          <units>Score on a Scale</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="139"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>From Week 4 to Week 24 in participants treated with deferasirox DT at Week 24</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="102"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.1" spread="0.80"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>From Week 4 to Week 24 in participants treated with deferasirox FCT at Week 24</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="6"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.5" spread="1.22"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>From Week 24 to Week 28 in participants treated with deferasirox DT at Week 28</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="0"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
            </class>
            <class>
              <title>From Week 24 to Week 28 in participants treated with deferasirox FCT at Week 28</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="106"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.5" spread="1.16"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>From Week 24 to Week 48 in participants treated with deferasirox DT at Week 48</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="0"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
            </class>
            <class>
              <title>From Week 24 to Week 48 in participants treated with deferasirox FCT at Week 48</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="98"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.5" spread="1.08"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change Over Time in Palatability Score of Palatability Questionnaire</title>
        <description>The palatability questionnaire consisted of 4 items, three items measuring taste and one item measuring aftertaste. Among the taste items, first one measured taste on a 5-point response scale. The other two items measured what happened after taking the medicine and how the perceived amount of liquid taken with the medicine was. Responses to these 3 items were combined and converted into a single palatability score using a scoring matrix: each combination of responses on each of 3 items corresponded to a predefined palatability score. E.g. if a participant responded &quot;bad&quot; to item 1, &quot;vomited &lt;30min&quot; to item 2 and &quot;not enough&quot; to item 3, then the palatability score assigned was 0. This score ranged from 0 to 11; higher scores indicated better palatability. For participants &lt;10 years old, an observer palatability questionnaire was administered. Items and scoring algorithm were the same as for participants ≥10 years old. Change in palatability score over time was assessed</description>
        <time_frame>Week 4, 24, 28 and 48</time_frame>
        <population>FAS: Participants to whom study treatment was assigned and who received at least one dose of each study treatment (DT and FCT). Number analyzed signified number of participants with available data for this outcome measure at specified timepoints.</population>
        <group_list>
          <group group_id="O1">
            <title>Deferasirox DT Followed by Deferasirox FCT</title>
            <description>Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT</description>
          </group>
        </group_list>
        <measure>
          <title>Change Over Time in Palatability Score of Palatability Questionnaire</title>
          <description>The palatability questionnaire consisted of 4 items, three items measuring taste and one item measuring aftertaste. Among the taste items, first one measured taste on a 5-point response scale. The other two items measured what happened after taking the medicine and how the perceived amount of liquid taken with the medicine was. Responses to these 3 items were combined and converted into a single palatability score using a scoring matrix: each combination of responses on each of 3 items corresponded to a predefined palatability score. E.g. if a participant responded &quot;bad&quot; to item 1, &quot;vomited &lt;30min&quot; to item 2 and &quot;not enough&quot; to item 3, then the palatability score assigned was 0. This score ranged from 0 to 11; higher scores indicated better palatability. For participants &lt;10 years old, an observer palatability questionnaire was administered. Items and scoring algorithm were the same as for participants ≥10 years old. Change in palatability score over time was assessed</description>
          <population>FAS: Participants to whom study treatment was assigned and who received at least one dose of each study treatment (DT and FCT). Number analyzed signified number of participants with available data for this outcome measure at specified timepoints.</population>
          <units>Score on a Scale</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="139"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>From Week 4 to Week 24- participants treated with deferasirox DT at Week 24</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="131"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.1" spread="2.46"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>From Week 4 to Week 24 in participants treated with deferasirox FCT at Week 24</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="7"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.0" spread="0.00"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>From Week 24 to Week 28 in participants treated with deferasirox DT at Week 28</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="0"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
            </class>
            <class>
              <title>From Week 24 to Week 28 in participants treated with deferasirox FCT at Week 28</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="137"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1.1" spread="2.77"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>From Week 24 to Week 48 in participants treated with deferasirox DT at Week 48</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="0"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
            </class>
            <class>
              <title>From Week 24 to Week 48 in participants treated with deferasirox FCT at Week 48</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="134"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1.3" spread="2.54"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change From Baseline in Adherence Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire</title>
        <description>The mSICT patient reported outcome (PRO) consisted of 15 items that represented 3 domains: Adherence, Preference, and Concerns. The adherence domain score consisted of 6 adherence items, measured using a 5-point response scale. The adherence score was calculated by summing these 6 items, with scores ranging from 6 to 30. Higher scores indicated worse adherence.&#xD;
For participants &lt;10 years old, an observer version (ObsRO) was administered. The adherence score remained the same as for participants ≥10 years old.</description>
        <time_frame>Baseline (week 2 or, if missing, week 3), week 24, 28 and 48</time_frame>
        <population>FAS: Participants to whom study treatment was assigned and who received at least one dose of each study treatment (DT and FCT). Number analyzed signified number of participants with available data for this outcome measure at specified timepoints</population>
        <group_list>
          <group group_id="O1">
            <title>Deferasirox DT Followed by Deferasirox FCT</title>
            <description>Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline in Adherence Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire</title>
          <description>The mSICT patient reported outcome (PRO) consisted of 15 items that represented 3 domains: Adherence, Preference, and Concerns. The adherence domain score consisted of 6 adherence items, measured using a 5-point response scale. The adherence score was calculated by summing these 6 items, with scores ranging from 6 to 30. Higher scores indicated worse adherence.&#xD;
For participants &lt;10 years old, an observer version (ObsRO) was administered. The adherence score remained the same as for participants ≥10 years old.</description>
          <population>FAS: Participants to whom study treatment was assigned and who received at least one dose of each study treatment (DT and FCT). Number analyzed signified number of participants with available data for this outcome measure at specified timepoints</population>
          <units>Score on a scale</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="139"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Week 24- participants ≥ 10 years treated with deferasirox DT (PRO)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="72"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.3" spread="3.37"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 24 - participants ≥ 10 years treated with deferasirox FCT (PRO)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="4"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-1.0" spread="2.94"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 28 - participants ≥ 10 years treated with deferasirox DT (PRO)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="0"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
            </class>
            <class>
              <title>Week 28 - participants ≥ 10 years treated with deferasirox FCT (PRO)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="77"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.5" spread="3.59"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 48 - participants ≥ 10 years treated with deferasirox DT (PRO)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="0"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
            </class>
            <class>
              <title>Week 48 - participants ≥ 10 years treated with deferasirox FCT (PRO)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="75"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.5" spread="3.29"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 24 - participants &lt; 10 years treated with deferasirox DT (ObsRO)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="56"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.2" spread="3.35"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 24 - participants &lt; 10 years treated with deferasirox FCT (ObsRO)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="3"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.7" spread="4.16"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 28 - participants &lt; 10 years treated with deferasirox DT (ObsRO)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="0"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
            </class>
            <class>
              <title>Week 28 - participants &lt; 10 years treated with deferasirox FCT (ObsRO)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="58"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.8" spread="3.68"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 48 - participants &lt; 10 years treated with deferasirox DT (ObsRO)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="0"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
            </class>
            <class>
              <title>Week 48 - participants &lt; 10 years treated with deferasirox FCT (ObsRO)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="55"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1.1" spread="3.79"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change From Baseline in Preference Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire</title>
        <description>The mSICT patient reported outcome (PRO) consisted of 15 items that represented 3 domains: Adherence, Preference, and Concerns.&#xD;
The preference/satisfaction domain score consisted of 2 preference/satisfaction items, measured using a 5-point response scale. The preference score was calculated by summing these 2 items, with scores ranging from 2 to 10. Higher scores indicated worse satisfaction.&#xD;
For participants &lt; 10 years old, an observer version (ObsRO) was administered. Preference score remained the same as for participants ≥ 10 years old.</description>
        <time_frame>Baseline (week 2 or, if missing, week 3), week 24, 28 and 48</time_frame>
        <population>FAS: Participants to whom study treatment was assigned and who received at least one dose of each study treatment (DT and FCT). Number analyzed signified number of participants with available data for this outcome measure at specified timepoints</population>
        <group_list>
          <group group_id="O1">
            <title>Deferasirox DT Followed by Deferasirox FCT</title>
            <description>Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline in Preference Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire</title>
          <description>The mSICT patient reported outcome (PRO) consisted of 15 items that represented 3 domains: Adherence, Preference, and Concerns.&#xD;
The preference/satisfaction domain score consisted of 2 preference/satisfaction items, measured using a 5-point response scale. The preference score was calculated by summing these 2 items, with scores ranging from 2 to 10. Higher scores indicated worse satisfaction.&#xD;
For participants &lt; 10 years old, an observer version (ObsRO) was administered. Preference score remained the same as for participants ≥ 10 years old.</description>
          <population>FAS: Participants to whom study treatment was assigned and who received at least one dose of each study treatment (DT and FCT). Number analyzed signified number of participants with available data for this outcome measure at specified timepoints</population>
          <units>Score on a scale</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="139"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Week 24 - participants ≥ 10 treated with deferasirox DT (PRO)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="72"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.5" spread="1.21"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 24 - participants ≥ 10 treated with deferasirox FCT (PRO)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="4"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.5" spread="3.70"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 28 - participants ≥ 10 treated with deferasirox DT (PRO)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="0"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
            </class>
            <class>
              <title>Week 28 - participants ≥ 10 treated with deferasirox FCT (PRO)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="77"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-1.1" spread="1.83"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 48 - participants ≥ 10 treated with deferasirox DT (PRO)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="0"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
            </class>
            <class>
              <title>Week 48 - participants ≥ 10 treated with deferasirox FCT (PRO)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="75"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.9" spread="2.02"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 24 - participants &lt; 10 years treated with deferasirox DT (ObsRO)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="55"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.0" spread="1.49"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 24 - participants &lt; 10 years treated with deferasirox FCT (ObsRO)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="3"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.0" spread="1.73"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 28 - participants &lt; 10 years treated with deferasirox DT (ObsRO)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="0"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
            </class>
            <class>
              <title>Week 28 - participants &lt; 10 years treated with deferasirox FCT (ObsRO)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="57"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.8" spread="1.74"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 48 - participants &lt; 10 years treated with deferasirox DT (ObsRO)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="0"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
            </class>
            <class>
              <title>Week 48 - participants &lt; 10 years treated with deferasirox FCT (ObsRO)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="54"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.9" spread="1.75"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change From Baseline in Concerns Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire</title>
        <description>The mSICT patient reported outcome (PRO) consisted of 15 items that represented 3 domains: Adherence, Preference, and Concerns.&#xD;
The concerns domain score consisted of 3 items to address any concerns or worries with the medication. All 3 items were measured on a 5-point response scale. The concerns score was calculated by summing the 3 items, with scores ranging from 3 to 15. Higher scores indicated fewer concerns. For participants &lt; 10 years old, an observer version (ObsRO) was administered. Concerns score remained the same as for participants ≥ 10 years old.</description>
        <time_frame>Baseline (week 2 or, if missing, week 3), week 24, 28 and 48</time_frame>
        <population>FAS: Participants to whom study treatment was assigned and who received at least one dose of each study treatment (DT and FCT). Number analyzed signified number of participants with available data for this outcome measure at specified timepoints</population>
        <group_list>
          <group group_id="O1">
            <title>Deferasirox DT Followed by Deferasirox FCT</title>
            <description>Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline in Concerns Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire</title>
          <description>The mSICT patient reported outcome (PRO) consisted of 15 items that represented 3 domains: Adherence, Preference, and Concerns.&#xD;
The concerns domain score consisted of 3 items to address any concerns or worries with the medication. All 3 items were measured on a 5-point response scale. The concerns score was calculated by summing the 3 items, with scores ranging from 3 to 15. Higher scores indicated fewer concerns. For participants &lt; 10 years old, an observer version (ObsRO) was administered. Concerns score remained the same as for participants ≥ 10 years old.</description>
          <population>FAS: Participants to whom study treatment was assigned and who received at least one dose of each study treatment (DT and FCT). Number analyzed signified number of participants with available data for this outcome measure at specified timepoints</population>
          <units>Score on a scale</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="139"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Week 24 - participants ≥ 10 treated with deferasirox DT (PRO)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="72"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.1" spread="1.99"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 24 - participants ≥ 10 treated with deferasirox FCT (PRO)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="4"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-1.0" spread="1.15"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 28 - participants ≥ 10 treated with deferasirox DT (PRO)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="0"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
            </class>
            <class>
              <title>Week 28 - participants ≥ 10 treated with deferasirox FCT (PRO)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="77"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.3" spread="1.84"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 48 - participants ≥ 10 treated with deferasirox DT (PRO)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="0"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
            </class>
            <class>
              <title>Week 48 - participants ≥ 10 treated with deferasirox FCT (PRO)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="75"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.5" spread="1.80"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 24 - participants &lt; 10 years treated with deferasirox DT (ObsRO)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="55"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.4" spread="1.33"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 24 - participants &lt; 10 years treated with deferasirox FCT (ObsRO)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="3"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.7" spread="1.15"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 28 - participants &lt; 10 years treated with deferasirox DT (ObsRO)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="0"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
            </class>
            <class>
              <title>Week 28 - participants &lt; 10 years treated with deferasirox FCT (ObsRO)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="58"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.1" spread="2.05"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 48 - participants &lt; 10 years treated with deferasirox DT (ObsRO)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="0"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
            </class>
            <class>
              <title>Week 48 - participants &lt; 10 years treated with deferasirox FCT (ObsRO)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="54"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.1" spread="1.58"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change From Baseline in Gastrointestinal (GI) Symptom Score Based on GI Questionnaire</title>
        <description>The GI symptom score was calculated from responses to 5 questions of the GI questionnaire, each with a possible score of 1 to 5, for an overall possible score range of 5 to 25, where a lower score represents a less severe GI symptom and a higher score represents a more severe GI symptom.&#xD;
An observer GI symptom questionnaire was administered to those patients who were &lt; 10 years old. The questionnaire was completed by the parents of the participants. All items and the scoring algorithm remained the same as for participants ≥ 10 years old.</description>
        <time_frame>Baseline (week -1 or, if missing, week -2), week 24, 28 and 48</time_frame>
        <population>FAS: Participants to whom study treatment was assigned and who received at least one dose of each study treatment (DT and FCT). Number analyzed signified number of participants with available data for this outcome measure at specified timepoints</population>
        <group_list>
          <group group_id="O1">
            <title>Deferasirox DT Followed by Deferasirox FCT</title>
            <description>Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline in Gastrointestinal (GI) Symptom Score Based on GI Questionnaire</title>
          <description>The GI symptom score was calculated from responses to 5 questions of the GI questionnaire, each with a possible score of 1 to 5, for an overall possible score range of 5 to 25, where a lower score represents a less severe GI symptom and a higher score represents a more severe GI symptom.&#xD;
An observer GI symptom questionnaire was administered to those patients who were &lt; 10 years old. The questionnaire was completed by the parents of the participants. All items and the scoring algorithm remained the same as for participants ≥ 10 years old.</description>
          <population>FAS: Participants to whom study treatment was assigned and who received at least one dose of each study treatment (DT and FCT). Number analyzed signified number of participants with available data for this outcome measure at specified timepoints</population>
          <units>Score on a scale</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="139"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Week 24 - participants ≥ 10 years treated with deferasirox DT</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="71"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1.4" spread="5.53"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 24 - participants ≥ 10 years treated with deferasirox FCT</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="4"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="5.8" spread="12.82"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 28 - participants ≥ 10 years treated with deferasirox DT</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="0"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
            </class>
            <class>
              <title>Week 28 - participants ≥ 10 years treated with deferasirox FCT</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="75"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-1.9" spread="4.33"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 48 - participants ≥ 10 years treated with deferasirox DT</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="0"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
            </class>
            <class>
              <title>Week 48 -participants ≥ 10 years treated with deferasirox FCT</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="75"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-2.2" spread="4.74"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 24 - participants &lt; 10 years treated with deferasirox DT</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="60"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.2" spread="2.78"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 24 - participants &lt; 10 years treated with deferasirox FCT</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="3"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-2.0" spread="2.65"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 28 - participants &lt; 10 years treated with deferasirox DT</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="0"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
            </class>
            <class>
              <title>Week 28 - participants &lt; 10 years treated with deferasirox FCT</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="62"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.5" spread="2.49"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 48 - participants&lt; 10 years treated with deferasirox DT</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="0"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
            </class>
            <class>
              <title>Week 48 - participants &lt; 10 years treated with deferasirox FCT</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="61"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.6" spread="2.33"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change From Baseline in Serum Ferritin Levels</title>
        <description>Absolute change from baseline over time in serum ferritin levels</description>
        <time_frame>From Baseline (Day 1) up to 96 weeks</time_frame>
        <population>FAS: Participants to whom study treatment was assigned and who received at least one dose of each study treatment (DT and FCT). Number analyzed signified number of participants with available data for this outcome measure at specified timepoints</population>
        <group_list>
          <group group_id="O1">
            <title>Deferasirox DT Followed by Deferasirox FCT</title>
            <description>Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline in Serum Ferritin Levels</title>
          <description>Absolute change from baseline over time in serum ferritin levels</description>
          <population>FAS: Participants to whom study treatment was assigned and who received at least one dose of each study treatment (DT and FCT). Number analyzed signified number of participants with available data for this outcome measure at specified timepoints</population>
          <units>microgram/liter (ug/L)</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="139"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Week 2 - participants treated with deferasirox DT</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="108"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="363.654" spread="323.3828"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 4 - participants treated with deferasirox DT</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="120"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="393.126" spread="457.8032"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 8 - participants treated with deferasirox DT</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="121"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="462.560" spread="427.7138"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 12 - participants treated with deferasirox DT</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="129"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="510.455" spread="548.6222"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 16 - participants treated with deferasirox DT</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="131"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="519.038" spread="508.7432"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 20 - participants treated with deferasirox DT</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="134"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="540.618" spread="548.2318"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 24 - participants treated with deferasirox DT</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="130"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="644.849" spread="629.6687"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 28 - participants treated with deferasirox FCT</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="114"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="679.610" spread="701.6219"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 32 - participants treated with deferasirox FCT</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="126"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="623.600" spread="629.5417"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 36 - participants treated with deferasirox FCT</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="127"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="679.011" spread="674.6716"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 40 - participants treated with deferasirox FCT</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="120"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="739.016" spread="659.0840"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 44 - participants treated with deferasirox FCT</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="120"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="748.063" spread="647.3000"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 48 - participants treated with deferasirox FCT</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="120"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="833.700" spread="788.0650"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 52 - participants treated with deferasirox FCT</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="113"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="760.229" spread="798.0836"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 56 - participants treated with deferasirox FCT</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="106"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="903.622" spread="832.9872"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 60 - participants treated with deferasirox FCT</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="102"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="980.644" spread="994.4445"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 64 - participants treated with deferasirox FCT</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="92"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="863.579" spread="854.8592"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 68 - participants treated with deferasirox FCT</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="91"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="921.725" spread="860.8738"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 72 - participants treated with deferasirox FCT</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="90"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="971.112" spread="872.7306"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 76 - participants treated with deferasirox FCT</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="90"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="888.917" spread="869.0805"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 80 - participants treated with deferasirox FCT</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="77"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1005.758" spread="868.4338"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 84 - participants treated with deferasirox FCT</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="87"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1013.683" spread="924.5828"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 88 - participants treated with deferasirox FCT</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="80"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1117.849" spread="985.0767"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 92 - participants treated with deferasirox FCT</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="74"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1222.429" spread="1027.2565"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 96 - participants treated with deferasirox FCT</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="74"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1137.347" spread="972.1286"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
    </outcome_list>

    <reported_events>
      <time_frame>Adverse events (AEs) and serious AEs (including all-cause mortality data table) were collected from day of first dose to 30 days after last dose of study medication, assessed up to approximately 30 weeks for deferasirox DT treatment (core phase), 39 weeks for deferasirox FCT treatment (core phase) and 52 weeks for deferasirox FCT treatment (extension phase).</time_frame>
      <desc>Any sign or symptom that occurred during the study treatment plus the 30 days post treatment. Safety analyses were performed in all participants who received at least one dose of either study treatment (DT or FCT), and at least one safety assessment on or after Day 1. If, for a participant, AE start date was in deferasirox DT period and end date was in deferasirox FCT period, the participant was counted in both periods.</desc>
      <group_list>
        <group group_id="E1">
          <title>Deferasirox DT (Core Phase)</title>
          <description>Participants who were treated with deferasirox DT once daily in the core phase</description>
        </group>
        <group group_id="E2">
          <title>Deferasirox FCT (Core Phase)</title>
          <description>Participants who were treated with deferasirox FCT once daily in the core phase</description>
        </group>
        <group group_id="E3">
          <title>Deferasirox FCT (Extension Phase)</title>
          <description>Participants who were treated with deferasirox FCT once daily in the extension phase</description>
        </group>
      </group_list>
      <serious_events>
        <default_vocab>MedDRA (23.1)</default_vocab>
        <default_assessment>Systematic Assessment</default_assessment>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, all-cause mortality</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="116"/>
              </event>
              <event>
                <sub_title>Total, serious adverse events</sub_title>
                <counts group_id="E1" subjects_affected="13" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="6" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="16" subjects_at_risk="116"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Blood and lymphatic system disorders</title>
            <event_list>
              <event>
                <sub_title>Haemolysis</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="116"/>
              </event>
              <event>
                <sub_title>Leukopenia</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="116"/>
              </event>
              <event>
                <sub_title>Neutropenia</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="116"/>
              </event>
              <event>
                <sub_title>Thrombocytopenia</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="116"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Cardiac disorders</title>
            <event_list>
              <event>
                <sub_title>Cardiac failure chronic</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="116"/>
              </event>
              <event>
                <sub_title>Cardiac failure congestive</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="116"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Eye disorders</title>
            <event_list>
              <event>
                <sub_title>Optic nerve cupping</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="116"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Gastrointestinal disorders</title>
            <event_list>
              <event>
                <sub_title>Pancreatitis</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="116"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>General disorders</title>
            <event_list>
              <event>
                <sub_title>Pyrexia</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="2" subjects_at_risk="116"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Hepatobiliary disorders</title>
            <event_list>
              <event>
                <sub_title>Cholangitis acute</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="116"/>
              </event>
              <event>
                <sub_title>Jaundice cholestatic</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="116"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Infections and infestations</title>
            <event_list>
              <event>
                <sub_title>Appendicitis</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="116"/>
              </event>
              <event>
                <sub_title>Cellulitis</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="116"/>
              </event>
              <event>
                <sub_title>Diarrhoea infectious</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="116"/>
              </event>
              <event>
                <sub_title>Ecthyma</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="116"/>
              </event>
              <event>
                <sub_title>Gastrointestinal infection</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="116"/>
              </event>
              <event>
                <sub_title>Influenza</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="116"/>
              </event>
              <event>
                <sub_title>Otitis media</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="116"/>
              </event>
              <event>
                <sub_title>Respiratory tract infection</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="116"/>
              </event>
              <event>
                <sub_title>Viral infection</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="116"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Injury, poisoning and procedural complications</title>
            <event_list>
              <event>
                <sub_title>Abdominal injury</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="116"/>
              </event>
              <event>
                <sub_title>Femur fracture</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="116"/>
              </event>
              <event>
                <sub_title>Gastrointestinal injury</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="116"/>
              </event>
              <event>
                <sub_title>Road traffic accident</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="116"/>
              </event>
              <event>
                <sub_title>Splenic rupture</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="116"/>
              </event>
              <event>
                <sub_title>Ulna fracture</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="116"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Investigations</title>
            <event_list>
              <event>
                <sub_title>Alanine aminotransferase increased</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="116"/>
              </event>
              <event>
                <sub_title>Aspartate aminotransferase increased</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="116"/>
              </event>
              <event>
                <sub_title>Blood bilirubin increased</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="116"/>
              </event>
              <event>
                <sub_title>Hepatic enzyme increased</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="116"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Metabolism and nutrition disorders</title>
            <event_list>
              <event>
                <sub_title>Hypokalaemia</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="116"/>
              </event>
              <event>
                <sub_title>Metabolic acidosis</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="116"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Neoplasms benign, malignant and unspecified (incl cysts and polyps)</title>
            <event_list>
              <event>
                <sub_title>Cholangiocarcinoma</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="116"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Nervous system disorders</title>
            <event_list>
              <event>
                <sub_title>Polyneuropathy</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="116"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Renal and urinary disorders</title>
            <event_list>
              <event>
                <sub_title>Acute kidney injury</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="116"/>
              </event>
              <event>
                <sub_title>Proteinuria</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="116"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Skin and subcutaneous tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Angioedema</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="116"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Vascular disorders</title>
            <event_list>
              <event>
                <sub_title>Hypertension</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="116"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </serious_events>
      <other_events>
        <frequency_threshold>5</frequency_threshold>
        <default_vocab>MedDRA (23.1)</default_vocab>
        <default_assessment>Systematic Assessment</default_assessment>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, other adverse events</sub_title>
                <counts group_id="E1" subjects_affected="82" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="59" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="49" subjects_at_risk="116"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Gastrointestinal disorders</title>
            <event_list>
              <event>
                <sub_title>Diarrhoea</sub_title>
                <counts group_id="E1" subjects_affected="16" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="5" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="2" subjects_at_risk="116"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>General disorders</title>
            <event_list>
              <event>
                <sub_title>Pyrexia</sub_title>
                <counts group_id="E1" subjects_affected="7" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="8" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="5" subjects_at_risk="116"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Hepatobiliary disorders</title>
            <event_list>
              <event>
                <sub_title>Hepatomegaly</sub_title>
                <counts group_id="E1" subjects_affected="8" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="116"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Infections and infestations</title>
            <event_list>
              <event>
                <sub_title>Nasopharyngitis</sub_title>
                <counts group_id="E1" subjects_affected="6" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="7" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="3" subjects_at_risk="116"/>
              </event>
              <event>
                <sub_title>Pharyngitis</sub_title>
                <counts group_id="E1" subjects_affected="7" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="6" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="6" subjects_at_risk="116"/>
              </event>
              <event>
                <sub_title>Upper respiratory tract infection</sub_title>
                <counts group_id="E1" subjects_affected="8" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="8" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="4" subjects_at_risk="116"/>
              </event>
              <event>
                <sub_title>Urinary tract infection</sub_title>
                <counts group_id="E1" subjects_affected="9" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="3" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="3" subjects_at_risk="116"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Investigations</title>
            <event_list>
              <event>
                <sub_title>Serum ferritin increased</sub_title>
                <counts group_id="E1" subjects_affected="8" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="2" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="116"/>
              </event>
              <event>
                <sub_title>Urine protein/creatinine ratio increased</sub_title>
                <counts group_id="E1" subjects_affected="13" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="12" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="7" subjects_at_risk="116"/>
              </event>
              <event>
                <sub_title>Hepatic enzyme increased</sub_title>
                <counts group_id="E1" subjects_affected="5" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="4" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="6" subjects_at_risk="116"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Renal and urinary disorders</title>
            <event_list>
              <event>
                <sub_title>Proteinuria</sub_title>
                <counts group_id="E1" subjects_affected="8" subjects_at_risk="148"/>
                <counts group_id="E2" subjects_affected="9" subjects_at_risk="140"/>
                <counts group_id="E3" subjects_affected="12" subjects_at_risk="116"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </other_events>
    </reported_events>

    <certain_agreements>
      <pi_employee>Principal Investigators are NOT employed by the organization sponsoring the study.</pi_employee>
      <restrictive_agreement>The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (ie, data from all sites) in the clinical trial.</restrictive_agreement>
    </certain_agreements>
    <point_of_contact>
      <name_or_title>Study director</name_or_title>
      <organization>Novartis Pharmaceuticals</organization>
      <phone>862-778-8300</phone>
      <email>novartis.email@novartis.com</email>
    </point_of_contact>
  </clinical_results>
</clinical_study>


download date: ClinicalTrials.gov processed this data on September 20, 2023
link text: Link to the current ClinicalTrials.gov record.
url: https://clinicaltrials.gov/ct2/show/NCT02993224
org study id: CICL670FIC05
secondary id: 2016-002282-61
nct id: NCT02993224
lead sponsor: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
Study to evaluate patient preference of deferasirox film-coated tablet (FCT) or deferasirox
      dispersible tablet (DT) in patient with transfusion - dependent thalassemia or
      non-transfusion -dependent thalassemia as measured by preference questionnaire at Week 48
This was an open-label, multicenter, single arm, phase II study aimed at collecting data on
      preference for iron chelation therapy in patients with transfusion-dependent thalassemia
      (TDT) or non-transfusion-dependent thalassemia (NTDT) throughout a 48 week treatment period.
      Participants who were either chelator-naïve, or who were previously treated with iron
      chelators (excluding deferasirox) for at least 6 months continuously, were eligible to
      participate in this study.

      The study was divided into 2 phases:

        1. Core Phase:

             -  Screening phase which lasted for a maximum of 4 weeks to determine patient
                eligibility followed by

             -  Period 1: Participants were treated with deferasirox DT from Baseline visit Day 1
                to Week 24

             -  Period 2: Participants were treated with deferasirox FCT from Week 25 to Week 48:

           At the discretion of the investigator, patients could switch from deferasirox DT to
           deferasirox FCT at any time during Period 1 of the Core phase, and vice versa, from
           deferasirox FCT to deferasirox DT at any time during Period 2 of the Core phase.
           Re-switching treatments was not allowed within each period.

        2. Extension Phase:

      Participants could continue deferasirox FCT formulation as per the judgment of the
      investigator, through an extension phase for a maximum of 48 weeks months from the last dose
      of deferasirox FCT received at the end of period 2 in the Core Phase or until one of the end
      of study criteria defined is met, whichever came first. Participation in the extension phase
      was optional.

      The end of study was defined as the earliest occurrence of one of the following:

        -  The patient reached Week 96 in the Extension phase.

        -  Deferasirox FCT was locally reimbursed for this indication (only applicable for the
           Extension phase)

        -  Another clinical study or post-trial access program became available that could continue
           to provide deferasirox FCT in this patient population and all patients ongoing were
           eligible to be transferred to that clinical study.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Other
masking: None (Open Label)
measure: Number of Participants Preferring Deferasirox FCT or DT at Week 48 Based on Preference Questionnaire (Item 2)
time frame: Week 48
description: Number of participants preferring deferasirox FCT or DT as measured by preference questionnaire (item 2) at Week 48. The preference questionnaire was a 3-item questionnaire. At Week 48, the second item of the preference questionnaire asked the patients (or parents of young patients from 2 to 9 years old) which medicine did the patient "like best": "Tablet to dissolve in liquid" (=deferasirox DT), "Film coated tablet" (=deferasirox FCT), "Sprinkle powder on food" (=deferasirox FCT) and "I don't know" (=none of the above). The number of participants who selected each response option for item 2 was assessed. The analysis was performed for participants who answered the item 2 of the preference questionnaire.
measure: Number of Participants Preferring Deferasirox FCT, Deferasirox DT or Previous Iron Chelation Therapy at Week 28 Based on Preference Questionnaire (Item 2)
time frame: Week 28
description: Number of participants preferring deferasirox FCT, deferasirox DT or previous iron chelation therapy as measured by preference questionnaire (item 2) at Week 28. The preference questionnaire was a 3-item questionnaire. At Week 28, the second item of this questionnaire asked the patients (or parents of young patients from 2 to 9 years old) which medicine did the patient "like best": "Tablet to dissolve in liquid" (=deferasirox DT), "Film coated tablet (taken once a day)" (=deferasirox FCT), "Sprinkle powder on food" (=deferasirox FCT), "Tablet (taken 3 times a day)" (=previous iron chelation therapy), "Injection" (=previous iron chelation therapy) and "I don't know" (=none of the above). The number of participants who selected each response option for item 2 was assessed. This analysis was performed only for patients who had received iron chelation therapy prior to enrolling in the study and who answered the item 2 of the preference questionnaire.
measure: Number of Participants Preferring Deferasirox DT or Previous Iron Chelation Therapy at Week 4 and Week 24 Based on Preference Questionnaire (Item 2)
time frame: Week 4 and Week 24
description: Number of participants preferring deferasirox DT or previous iron chelation therapy as measured by preference questionnaire (item 2) at Week 4 and 24. The preference questionnaire was a 3-item questionnaire. At Week 4 and 24, the second item of the preference questionnaire asked the patients (or parents of young patients from 2 to 9 years old) which medicine did the patient "like best": "Tablet to dissolve in liquid" (=deferasirox DT), "Tablet (taken 3 times a day)" (=previous iron chelation therapy), "Injection" (=previous iron chelation therapy) and "I don't know" (=none of the above). The number of participants who selected each response option for item 2 was assessed. This analysis was performed only for patients who had received iron chelation therapy prior to enrolling in the study and who answered item 2 of the preference questionnaire.
measure: Number of Participants Selecting Each Reason for Treatment Preference as Assessed by the Preference Questionnaire at Week 28 and Week 48
time frame: Week 28 and Week 48
description: The preference questionnaire was a 3 item questionnaire. The first item asked the patients (or parents of young patients from 2 to 9 years old) which medicine they were taking. The second item asked which of the medicines did the patient "Like best". Finally, the third item asked the patient why he/she preferred the medicine they chose in the second item. The number of participants who selected each response option for item 3 was assessed. Participants could select multiple reasons for treatment preference at each timepoint.
measure: Percentage of Consumed Tablet Counts During Deferasirox DT and Deferasirox FCT Treatment Periods
time frame: Deferasirox DT: From Baseline up to Week 24. Deferasirox FCT: From Week 25 up to Week 48
description: The percentage of consumed tablet counts (compliance) was calculated for each treatment period in the core phase: deferasirox DT (period 1) and deferasirox FCT (period 2). Compliance was defined as the total tablet count consumed divided by total tablet count prescribed and multiplied by 100. Total tablet count consumed was calculated as total number of tablets dispensed minus total number of tablets lost/wasted or returned. Total tablet count prescribed was calculated as the number of tablets that the patient should have taken during this period. If a patient did not return the study drug, the compliance was not calculated.
measure: Change Over Time in Aftertaste Score of Palatability Questionnaire
time frame: Week 4, 24, 28 and 48
description: The palatability questionnaire consisted of 4 items, three items measuring taste and one item measuring aftertaste. The aftertaste item scored on a 5-point response scale with the response option: Very good = 1, Good = 2, Neither good nor bad = 3, Bad = 4, Very bad = 5. This item offered an additional response option of "no aftertaste". The aftertaste score was calculated among participants who had an aftertaste. Higher aftertaste scores indicated a worse aftertaste.
For participants less than (<) 10 years old, an observer palatability questionnaire was administered. Items and scoring algorithm remained the same as for participants greater than or equal to (≥) 10 years old.
Change in aftertaste score over time was assessed
measure: Change Over Time in Palatability Score of Palatability Questionnaire
time frame: Week 4, 24, 28 and 48
description: The palatability questionnaire consisted of 4 items, three items measuring taste and one item measuring aftertaste. Among the taste items, first one measured taste on a 5-point response scale. The other two items measured what happened after taking the medicine and how the perceived amount of liquid taken with the medicine was. Responses to these 3 items were combined and converted into a single palatability score using a scoring matrix: each combination of responses on each of 3 items corresponded to a predefined palatability score. E.g. if a participant responded "bad" to item 1, "vomited <30min" to item 2 and "not enough" to item 3, then the palatability score assigned was 0. This score ranged from 0 to 11; higher scores indicated better palatability. For participants <10 years old, an observer palatability questionnaire was administered. Items and scoring algorithm were the same as for participants ≥10 years old. Change in palatability score over time was assessed
measure: Change From Baseline in Adherence Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire
time frame: Baseline (week 2 or, if missing, week 3), week 24, 28 and 48
description: The mSICT patient reported outcome (PRO) consisted of 15 items that represented 3 domains: Adherence, Preference, and Concerns. The adherence domain score consisted of 6 adherence items, measured using a 5-point response scale. The adherence score was calculated by summing these 6 items, with scores ranging from 6 to 30. Higher scores indicated worse adherence.
For participants <10 years old, an observer version (ObsRO) was administered. The adherence score remained the same as for participants ≥10 years old.
measure: Change From Baseline in Preference Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire
time frame: Baseline (week 2 or, if missing, week 3), week 24, 28 and 48
description: The mSICT patient reported outcome (PRO) consisted of 15 items that represented 3 domains: Adherence, Preference, and Concerns.
The preference/satisfaction domain score consisted of 2 preference/satisfaction items, measured using a 5-point response scale. The preference score was calculated by summing these 2 items, with scores ranging from 2 to 10. Higher scores indicated worse satisfaction.
For participants < 10 years old, an observer version (ObsRO) was administered. Preference score remained the same as for participants ≥ 10 years old.
measure: Change From Baseline in Concerns Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire
time frame: Baseline (week 2 or, if missing, week 3), week 24, 28 and 48
description: The mSICT patient reported outcome (PRO) consisted of 15 items that represented 3 domains: Adherence, Preference, and Concerns.
The concerns domain score consisted of 3 items to address any concerns or worries with the medication. All 3 items were measured on a 5-point response scale. The concerns score was calculated by summing the 3 items, with scores ranging from 3 to 15. Higher scores indicated fewer concerns. For participants < 10 years old, an observer version (ObsRO) was administered. Concerns score remained the same as for participants ≥ 10 years old.
measure: Change From Baseline in Gastrointestinal (GI) Symptom Score Based on GI Questionnaire
time frame: Baseline (week -1 or, if missing, week -2), week 24, 28 and 48
description: The GI symptom score was calculated from responses to 5 questions of the GI questionnaire, each with a possible score of 1 to 5, for an overall possible score range of 5 to 25, where a lower score represents a less severe GI symptom and a higher score represents a more severe GI symptom.
An observer GI symptom questionnaire was administered to those patients who were < 10 years old. The questionnaire was completed by the parents of the participants. All items and the scoring algorithm remained the same as for participants ≥ 10 years old.
measure: Change From Baseline in Serum Ferritin Levels
time frame: From Baseline (Day 1) up to 96 weeks
description: Absolute change from baseline over time in serum ferritin levels
arm group label: Deferasirox DT followed by deferasirox FCT
arm group type: Experimental
description: Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT
intervention type: Drug
intervention name: Deferasirox dispersable tablet (DT)
description: Deferasirox DT was provided as 125 mg, 250 mg, 500 mg dispersible tablets for oral use. The strengths provided in an individual country could differ and reflected the strengths available commercially in each country.
For iron chelation naive participants, the starting dose on Baseline Day 1 was 20 mg/kg/day in TDT and 10 mg/kg/day in NTDT.
For iron chelation (deferoxamine and/or deferiprone) pre-treated participants, the starting dose was equivalent to the dose of deferoxamine received (for participants pre-treated with deferoxamine) and based on their serum ferritin levels (for participants pre-treated with deferiprone).
Participants took deferasirox DT once daily for 24 weeks (core phase). The required number of deferasirox DT tablets were to be dispersed with gentle stirring in a glass of water.
arm group label: Deferasirox DT followed by deferasirox FCT
intervention type: Drug
intervention name: Deferasirox film coated tablet (FCT)
description: Deferasirox FCT was provided as 90 mg, 180 mg, 360 mg film coated tablets for oral use. The FCT starting dose on Week 25 was 14 mg/kg/day in TDT and 7 mg/kg/day in NTDT.
Participants took deferasirox FCT once daily for 24 weeks during the core phase and up to 48 weeks during the extension phase. For patients with difficulties in swallowing deferasirox FCT, it was allowed to crush the film-coated tablets and administer the study drug by sprinkling the full dose on soft food (like yogurt or apple puree).
arm group label: Deferasirox DT followed by deferasirox FCT
criteria: 
gender: All
minimum age: 2 Years
maximum age: N/A
healthy volunteers: No
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country: Egypt
country: Lebanon
country: Morocco
country: Saudi Arabia
country: Thailand
country: Turkey
country: Vietnam
responsible party type: Sponsor
mesh term: Thalassemia
mesh term: Deferasirox
sharing ipd: Yes
ipd description: Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.
This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
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