<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/NCT01671111</url>
  </required_header>
  <id_info>
    <org_study_id>SPD602-301</org_study_id>
    <secondary_id>2011-006322-25</secondary_id>
    <nct_id>NCT01671111</nct_id>
  </id_info>
  <brief_title>Study for Transfusionally Iron Overloaded Children, Adolescents and Adults Using FBS0701 (SSP-004184)</brief_title>
  <official_title>A Phase 2, Open-label, Multicentre, Extension Safety and Tolerability Study for Transfusionally Iron Overloaded Children, Adolescents and Adults Using SSP-004184 (SPD602)</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Shire</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Takeda</source>
  <brief_summary>
    <textblock>
      The purpose of this extension study is to evaluate SSP-004184AQ in patients with&#xD;
      transfusional iron overload and to provide data on long term safety and efficacy.&#xD;
&#xD;
      SSP-004184AQ is an iron chelator under development for chronic daily oral administration to&#xD;
      patients with transfusional iron overload&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Terminated</overall_status>
  <why_stopped>
    This study was terminated due to treatment stop resulting in an inability to draw conclusions&#xD;
    from the data. Evaluation of nonclinical rat findings is ongoing.&#xD;
  </why_stopped>
  <start_date type="Actual">August 14, 2012</start_date>
  <completion_date type="Actual">April 24, 2014</completion_date>
  <primary_completion_date type="Actual">April 24, 2014</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>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Change From Baseline in Ferriscan® R2 Liver Iron Concentrations (LIC) at Week 24 (Cycle 1)</measure>
    <time_frame>Baseline, Week 24 (Cycle 1)</time_frame>
    <description>Efficacy of SSP-004184 was assessed by determining LIC. Abdominal magnetic resonance imaging (MRI) data were collected by using FerriScan R2 standard procedures and used to determine LIC. A negative change from baseline indicates that LIC decreased. A cycle consisted of 8 standard 6-weekly visits and the cycles were repeated each year for the duration of the study.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Change From Baseline in Ferriscan® R2 Liver Iron Concentrations (LIC) at Week 48 (Cycle 1)</measure>
    <time_frame>Baseline, Week 48 (Cycle 1)</time_frame>
    <description>Efficacy of SSP-004184 was assessed by determining LIC. Abdominal MRI data were collected by using FerriScan R2 standard procedures and used to determine LIC. A negative change from baseline indicates that LIC decreased. A cycle consisted of 8 standard 6-weekly visits and the cycles were repeated each year for the duration of the study.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Change From Baseline in Ferriscan® R2 Liver Iron Concentrations (LIC) at Week 24 (Cycle 2)</measure>
    <time_frame>Baseline, Week 24 (Cycle 2)</time_frame>
    <description>Efficacy of SSP-004184 was assessed by determining LIC. Abdominal MRI data were collected by using FerriScan R2 standard procedures and used to determine LIC. A negative change from baseline indicates that LIC decreased. A cycle consisted of 8 standard 6-weekly visits and the cycles were repeated each year for the duration of the study.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Change From Baseline in Cardiac T2* Values at Week 24 (Cycle 1)</measure>
    <time_frame>Baseline, Week 24 (Cycle 1)</time_frame>
    <description>The efficacy of SSP-004184 was assessed by determining cardiac iron load. Cardiac MRI data were collected by using T2* standard procedures and used to determine iron load. A negative change from baseline indicates that iron load increased. A cycle consisted of 8 standard 6-weekly visits and the cycles were repeated each year for the duration of the study.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Change From Baseline in Cardiac T2* Values at Week 48 (Cycle 1)</measure>
    <time_frame>Baseline, Week 48 (Cycle 1)</time_frame>
    <description>The efficacy of SSP-004184 was assessed by determining cardiac iron load. Cardiac MRI data were collected by using T2* standard procedures and used to determine iron load. A negative change from baseline indicates that iron load increased. A cycle consisted of 8 standard 6-weekly visits and the cycles were repeated each year for the duration of the study.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Change From Baseline in Cardiac T2* Values at Week 24 (Cycle 2)</measure>
    <time_frame>Baseline, Week 24 (Cycle 2)</time_frame>
    <description>The efficacy of SSP-004184 was assessed by determining cardiac iron load. Cardiac MRI data were collected by using T2* standard procedures and used to determine iron load. A negative change from baseline indicates that iron load increased. A cycle consisted of 8 standard 6-weekly visits and the cycles were repeated each year for the duration of the study.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline in Serum Ferritin Values at Specified Visits</measure>
    <time_frame>Baseline, Weeks 24 and 48 of Cycle 1, Week 24 of Cycle 2</time_frame>
    <description>A negative change from baseline indicates that serum ferritin decreased. A cycle consisted of 8 standard 6-weekly visits and the cycles were repeated each year for the duration of the study.</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">30</enrollment>
  <condition>Iron Overload Due to Repeated Red Blood Cell Transfusions</condition>
  <arm_group>
    <arm_group_label>SSP-004814AQ</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>SSP-004184AQ</intervention_name>
    <description>Long term extension study of 8-75 mg/kg/d of SSP-004184AQ (equivalent to 7-68 mg/kg/d free acid or &quot;active&quot; form)</description>
    <arm_group_label>SSP-004814AQ</arm_group_label>
    <other_name>SPD602</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Subjects and/or parents willing and able to sign the approved informed consent/and&#xD;
             assent (based on institutional guidelines).&#xD;
&#xD;
          -  An understanding, ability, and willingness to fully comply with study procedures and&#xD;
             restrictions.&#xD;
&#xD;
          -  Has either completed a previous SSP-004184AQ study or prematurely discontinued from&#xD;
             (with agreement from the Investigator and Shire physician) from one of the&#xD;
             SSP-004184AQ studies.&#xD;
&#xD;
          -  If applicable, female subjects should be either:&#xD;
&#xD;
               1. Post-menopausal (12 consecutive months of spontaneous amenorrhea), or&#xD;
&#xD;
               2. Surgically sterile, or&#xD;
&#xD;
               3. Females of child-bearing potential must have a negative urine pregnancy test at&#xD;
                  the Qualification and Enrollment Visit prior to dosing on Day 1. Females of&#xD;
                  child-bearing potential must agree to abstain from sexual activity that could&#xD;
                  result in pregnancy or agree to use acceptable methods of contraception.&#xD;
&#xD;
                  For subjects that did not transition directly from their feeder protocol and have&#xD;
                  initiated treatment with a chelator other than SSP-004184AQ:&#xD;
&#xD;
          -  Willing to discontinue all existing iron chelation therapies for a minimum period of 1&#xD;
             to 5 days prior to first dose of SSP-004184AQ.&#xD;
&#xD;
        (Note: This inclusion criterion is also applicable for subjects that were receiving&#xD;
        chelators other than SSP-004184AQ in their feeder protocol.)&#xD;
&#xD;
          -  Serum ferritin greater than 500 ng/mL at the Qualification and Enrollment Visit.&#xD;
&#xD;
          -  Liver iron concentration (LIC) greater than or equal to 2.0 mg iron per g (equivalent&#xD;
             dry weight, liver) determined by FerriScan® R2 MRI at the Qualification and Enrollment&#xD;
             Visit (Day -28 to Day -8).&#xD;
&#xD;
        (Note: Younger subjects for whom MRI is not feasible will be considered iron overloaded on&#xD;
        the basis of serum ferritin only.) - Mean of the previous 3 pre-transfusion hemoglobin&#xD;
        concentrations greater than or equal to 7.5 g/dL.&#xD;
&#xD;
        Exclusion:&#xD;
&#xD;
          -  Unwilling to remain off all other existing chelation therapies during SSP-004184AQ&#xD;
             dosing and for up to 24 hours from last dose.&#xD;
&#xD;
          -  A history of non-compliance in a prior FerroKin/Shire-sponsored SSP-004184AQ study&#xD;
             (excluding dose suspensions that were medically warranted).&#xD;
&#xD;
          -  Cardiac MRI T2* less than 10.0 milliseconds at the Qualification and Enrollment Visit.&#xD;
&#xD;
          -  Cardiac left ventricular ejection fraction less than 50% at the Qualification and&#xD;
             Enrollment Visit by MRI or below the locally determined normal range by&#xD;
             echocardiography if MRI information is not available.&#xD;
&#xD;
          -  Evidence of clinically relevant oral, cardiovascular, gastrointestinal, endocrine,&#xD;
             pulmonary, neurologic, psychiatric, immunologic, bone marrow, dermatologic, hepatic,&#xD;
             biliary, or renal dysfunction where, in the opinion of the Investigator or the Shire&#xD;
             Study Physician, treatment with SSP-004184AQ is relatively contraindicated.&#xD;
&#xD;
          -  Platelet count below 100,000/µL or absolute neutrophil count less than 1500/mm3 at the&#xD;
             Qualification and Enrollment Visit.&#xD;
&#xD;
          -  Known sensitivity to any ingredient in the SSP-004184AQ formulation.&#xD;
&#xD;
          -  Pregnant or lactating females.&#xD;
&#xD;
        For subjects that did not transition directly from their feeder protocol and have initiated&#xD;
        treatment with a chelator other than SSP-004184AQ:&#xD;
&#xD;
          -  In younger subjects for whom MRI is not feasible, evidence of severe cardiac&#xD;
             dysfunction, as assessed by the Investigator.&#xD;
&#xD;
          -  Non-elective hospitalization within the 30 days prior to receiving the first dose of&#xD;
             SSP 004184AQ.&#xD;
&#xD;
          -  For subjects greater than or equal to 18 years old: ALT greater than 200 IU/L at the&#xD;
             Qualification and Enrollment Visit.&#xD;
&#xD;
        OR For subjects less than 18 years old: ALT greater than 180 IU/L at the Qualification and&#xD;
        Enrollment Visit.&#xD;
&#xD;
        - For subjects greater than or equal to 18 years old: Evidence of severe renal&#xD;
        insufficiency, eg, serum creatinine 1.5X above the upper limit of normal or proteinuria&#xD;
        greater than 1 gm per day or a calculated glomerular filtration rate less than 40 mL/min.&#xD;
&#xD;
        OR For subjects less than 18 years old: Evidence of significant renal insufficiency, eg,&#xD;
        serum creatinine above the upper limit of normal or proteinuria greater than 1 gm per day.&#xD;
&#xD;
          -  Use of any investigational agent within the 30 days prior to receiving the first dose&#xD;
             of SSP-004184AQ.&#xD;
&#xD;
          -  Cardiac left ventricular ejection fraction below the locally determined normal range&#xD;
             in the 12 months prior to Screening by echocardiography or MRI or less than 50% at&#xD;
             Baseline testing by MRI (Echocardiograph is acceptable for LVEF if MRI information is&#xD;
             not available).&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>6 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Study Director</last_name>
    <role>Study Director</role>
    <affiliation>Takeda</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Children's Hospital Oakland</name>
      <address>
        <city>Oakland</city>
        <state>California</state>
        <zip>94609</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's Hospital of Boston</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02115</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>The Hospital for Sick Children</name>
      <address>
        <city>Toronto</city>
        <state>Ontario</state>
        <zip>M5G 1X8</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Toronto General Hospital</name>
      <address>
        <city>Toronto</city>
        <state>Ontario</state>
        <zip>M5G 2C4</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>San Luigi Hospital Thalassemia Centre</name>
      <address>
        <city>Orbassano</city>
        <state>Torino</state>
        <zip>10043</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Ospedale Regionale Microcitemie</name>
      <address>
        <city>Cagliari</city>
        <zip>09121</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Ospedale Galliera</name>
      <address>
        <city>Genova</city>
        <zip>16128</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Siriraj Hospital, Mahidol University</name>
      <address>
        <city>Bangkok</city>
        <zip>10700</zip>
        <country>Thailand</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Whittington Hospital</name>
      <address>
        <city>London</city>
        <zip>N19 5NF</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Canada</country>
    <country>Italy</country>
    <country>Thailand</country>
    <country>United Kingdom</country>
    <country>United States</country>
  </location_countries>
  <verification_date>June 2021</verification_date>
  <study_first_submitted>August 16, 2012</study_first_submitted>
  <study_first_submitted_qc>August 20, 2012</study_first_submitted_qc>
  <study_first_posted type="Estimate">August 23, 2012</study_first_posted>
  <results_first_submitted>July 10, 2015</results_first_submitted>
  <results_first_submitted_qc>July 10, 2015</results_first_submitted_qc>
  <results_first_posted type="Estimate">August 6, 2015</results_first_posted>
  <last_update_submitted>June 11, 2021</last_update_submitted>
  <last_update_submitted_qc>June 11, 2021</last_update_submitted_qc>
  <last_update_posted type="Actual">June 29, 2021</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Iron Overload</mesh_term>
  </condition_browse>

  <clinical_results>

    <participant_flow>
      <pre_assignment_details>A total of 30 participants were enrolled to this open-label extension study (24 participants transferred directly from feeder studies SPD602-201 [NCT01186419], SPD602-202 [NCT01363908], and SPD602-203 [NCT01604941] and 6 participants did not transfer directly and received a chelator other than SSP-004184 after discontinuation from a feeder study).</pre_assignment_details>
      <group_list>
        <group group_id="P1">
          <title>SSP-004184AQ</title>
          <description>Participants received SSP-004184AQ (magnesium salt of free acid or active form, that is, SSP-004184 [SPD602, FBS0701]) capsules orally at a total daily dose of 8-75 milligram per kilogram per day (mg/kg/day) (equivalent to SSP-004184 7-68 mg/kg/day) either once daily or twice daily at the discretion of investigator for up to a maximum of 3 years or until the sponsor decided to stop the study.</description>
        </group>
      </group_list>
      <period_list>
        <period>
          <title>Overall Study</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="30"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="0"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="30"/>
              </participants_list>
            </milestone>
          </milestone_list>
          <drop_withdraw_reason_list>
            <drop_withdraw_reason>
              <title>Adverse Event</title>
              <participants_list>
                <participants group_id="P1" count="2"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Lack of Efficacy</title>
              <participants_list>
                <participants group_id="P1" count="1"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Withdrawal by Subject</title>
              <participants_list>
                <participants group_id="P1" count="2"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Physician Decision</title>
              <participants_list>
                <participants group_id="P1" count="1"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Other</title>
              <participants_list>
                <participants group_id="P1" count="24"/>
              </participants_list>
            </drop_withdraw_reason>
          </drop_withdraw_reason_list>
        </period>
      </period_list>
    </participant_flow>

    <baseline>
      <population>Safety set included all participants who had taken at least 1 dose of investigational product.</population>
      <group_list>
        <group group_id="B1">
          <title>SSP-004184AQ</title>
          <description>Participants received SSP-004184AQ (magnesium salt of free acid or active form, that is, SSP-004184 [SPD602, FBS0701]) capsules orally at a total daily dose of 8-75 mg/kg/day (equivalent to SSP-004184 7-68 mg/kg/day) either once daily or twice daily at the discretion of investigator for up to a maximum of 3 years or until the sponsor decided to stop the study.</description>
        </group>
      </group_list>
      <analyzed_list>
        <analyzed>
          <units>Participants</units>
          <scope>Overall</scope>
          <count_list>
            <count group_id="B1" value="30"/>
          </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="25.5" spread="11.38"/>
                  </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="17"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Male</title>
                  <measurement_list>
                    <measurement group_id="B1" value="13"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </measure_list>
    </baseline>

    <outcome_list>
      <outcome>
        <type>Primary</type>
        <title>Change From Baseline in Ferriscan® R2 Liver Iron Concentrations (LIC) at Week 24 (Cycle 1)</title>
        <description>Efficacy of SSP-004184 was assessed by determining LIC. Abdominal magnetic resonance imaging (MRI) data were collected by using FerriScan R2 standard procedures and used to determine LIC. A negative change from baseline indicates that LIC decreased. A cycle consisted of 8 standard 6-weekly visits and the cycles were repeated each year for the duration of the study.</description>
        <time_frame>Baseline, Week 24 (Cycle 1)</time_frame>
        <population>Full analysis set (FAS) included all participants in the Safety set who had at least 1 post-baseline primary efficacy assessment. Here, 'n' signifies the number of FAS participants evaluable for the respective time points.</population>
        <group_list>
          <group group_id="O1">
            <title>SSP-004184AQ</title>
            <description>Participants received SSP-004184AQ (magnesium salt of free acid or active form, that is, SSP-004184 [SPD602, FBS0701]) capsules orally at a total daily dose of 8-75 mg/kg/day (equivalent to SSP-004184 7-68 mg/kg/day) either once daily or twice daily at the discretion of investigator for up to a maximum of 3 years or until the sponsor decided to stop the study.</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline in Ferriscan® R2 Liver Iron Concentrations (LIC) at Week 24 (Cycle 1)</title>
          <description>Efficacy of SSP-004184 was assessed by determining LIC. Abdominal magnetic resonance imaging (MRI) data were collected by using FerriScan R2 standard procedures and used to determine LIC. A negative change from baseline indicates that LIC decreased. A cycle consisted of 8 standard 6-weekly visits and the cycles were repeated each year for the duration of the study.</description>
          <population>Full analysis set (FAS) included all participants in the Safety set who had at least 1 post-baseline primary efficacy assessment. Here, 'n' signifies the number of FAS participants evaluable for the respective time points.</population>
          <units>milligram per gram (mg/g) dry tissue</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="23"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Baseline (n=23)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="9.9" spread="8.10"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Change at Week 24 (Cycle 1) (n=13)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-1.1" spread="1.86"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
            </group_id_list>
            <non_inferiority_type>Superiority or Other (legacy)</non_inferiority_type>
            <p_value>0.0560</p_value>
            <p_value_desc>P-value was from paired t-test for post treatment timepoint versus baseline.</p_value_desc>
            <method>paired t-test</method>
          </analysis>
        </analysis_list>
      </outcome>
      <outcome>
        <type>Primary</type>
        <title>Change From Baseline in Ferriscan® R2 Liver Iron Concentrations (LIC) at Week 48 (Cycle 1)</title>
        <description>Efficacy of SSP-004184 was assessed by determining LIC. Abdominal MRI data were collected by using FerriScan R2 standard procedures and used to determine LIC. A negative change from baseline indicates that LIC decreased. A cycle consisted of 8 standard 6-weekly visits and the cycles were repeated each year for the duration of the study.</description>
        <time_frame>Baseline, Week 48 (Cycle 1)</time_frame>
        <population>FAS participants evaluable for this outcome.</population>
        <group_list>
          <group group_id="O1">
            <title>SSP-004184AQ</title>
            <description>Participants received SSP-004184AQ (magnesium salt of free acid or active form, that is, SSP-004184 [SPD602, FBS0701]) capsules orally at a total daily dose of 8-75 mg/kg/day (equivalent to SSP-004184 7-68 mg/kg/day) either once daily or twice daily at the discretion of investigator for up to a maximum of 3 years or until the sponsor decided to stop the study.</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline in Ferriscan® R2 Liver Iron Concentrations (LIC) at Week 48 (Cycle 1)</title>
          <description>Efficacy of SSP-004184 was assessed by determining LIC. Abdominal MRI data were collected by using FerriScan R2 standard procedures and used to determine LIC. A negative change from baseline indicates that LIC decreased. A cycle consisted of 8 standard 6-weekly visits and the cycles were repeated each year for the duration of the study.</description>
          <population>FAS participants evaluable for this outcome.</population>
          <units>mg/g dry tissue</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="9"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-1.0" spread="1.68"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
            </group_id_list>
            <non_inferiority_type>Superiority or Other (legacy)</non_inferiority_type>
            <p_value>0.1232</p_value>
            <p_value_desc>P-value was from paired t-test for post treatment timepoint versus baseline.</p_value_desc>
            <method>paired t-test</method>
          </analysis>
        </analysis_list>
      </outcome>
      <outcome>
        <type>Primary</type>
        <title>Change From Baseline in Ferriscan® R2 Liver Iron Concentrations (LIC) at Week 24 (Cycle 2)</title>
        <description>Efficacy of SSP-004184 was assessed by determining LIC. Abdominal MRI data were collected by using FerriScan R2 standard procedures and used to determine LIC. A negative change from baseline indicates that LIC decreased. A cycle consisted of 8 standard 6-weekly visits and the cycles were repeated each year for the duration of the study.</description>
        <time_frame>Baseline, Week 24 (Cycle 2)</time_frame>
        <population>FAS participants evaluable for this outcome.</population>
        <group_list>
          <group group_id="O1">
            <title>SSP-004184AQ</title>
            <description>Participants received SSP-004184AQ (magnesium salt of free acid or active form, that is, SSP-004184 [SPD602, FBS0701]) capsules orally at a total daily dose of 8-75 mg/kg/day (equivalent to SSP-004184 7-68 mg/kg/day) either once daily or twice daily at the discretion of investigator for up to a maximum of 3 years or until the sponsor decided to stop the study.</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline in Ferriscan® R2 Liver Iron Concentrations (LIC) at Week 24 (Cycle 2)</title>
          <description>Efficacy of SSP-004184 was assessed by determining LIC. Abdominal MRI data were collected by using FerriScan R2 standard procedures and used to determine LIC. A negative change from baseline indicates that LIC decreased. A cycle consisted of 8 standard 6-weekly visits and the cycles were repeated each year for the duration of the study.</description>
          <population>FAS participants evaluable for this outcome.</population>
          <units>mg/g dry tissue</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="7"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-1.5" spread="2.23"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
            </group_id_list>
            <non_inferiority_type>Superiority or Other (legacy)</non_inferiority_type>
            <p_value>0.1319</p_value>
            <p_value_desc>P-value was from paired t-test for post treatment timepoint versus baseline.</p_value_desc>
            <method>paired t-test</method>
          </analysis>
        </analysis_list>
      </outcome>
      <outcome>
        <type>Primary</type>
        <title>Change From Baseline in Cardiac T2* Values at Week 24 (Cycle 1)</title>
        <description>The efficacy of SSP-004184 was assessed by determining cardiac iron load. Cardiac MRI data were collected by using T2* standard procedures and used to determine iron load. A negative change from baseline indicates that iron load increased. A cycle consisted of 8 standard 6-weekly visits and the cycles were repeated each year for the duration of the study.</description>
        <time_frame>Baseline, Week 24 (Cycle 1)</time_frame>
        <population>FAS. Here, 'n' signifies the number of FAS participants evaluable for the respective time points.</population>
        <group_list>
          <group group_id="O1">
            <title>SSP-004184AQ</title>
            <description>Participants received SSP-004184AQ (magnesium salt of free acid or active form, that is, SSP-004184 [SPD602, FBS0701]) capsules orally at a total daily dose of 8-75 mg/kg/day (equivalent to SSP-004184 7-68 mg/kg/day) either once daily or twice daily at the discretion of investigator for up to a maximum of 3 years or until the sponsor decided to stop the study.</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline in Cardiac T2* Values at Week 24 (Cycle 1)</title>
          <description>The efficacy of SSP-004184 was assessed by determining cardiac iron load. Cardiac MRI data were collected by using T2* standard procedures and used to determine iron load. A negative change from baseline indicates that iron load increased. A cycle consisted of 8 standard 6-weekly visits and the cycles were repeated each year for the duration of the study.</description>
          <population>FAS. Here, 'n' signifies the number of FAS participants evaluable for the respective time points.</population>
          <units>milliseconds</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="23"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Baseline (n=23)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="35.14" spread="12.242"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Change at Week 24 (Cycle 1) (n=14)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.41" spread="8.069"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
            </group_id_list>
            <non_inferiority_type>Superiority or Other (legacy)</non_inferiority_type>
            <p_value>0.8061</p_value>
            <p_value_desc>P-value was from paired t-test for log-transformed data at post treatment timepoint versus baseline.</p_value_desc>
            <method>paired t-test</method>
          </analysis>
        </analysis_list>
      </outcome>
      <outcome>
        <type>Primary</type>
        <title>Change From Baseline in Cardiac T2* Values at Week 48 (Cycle 1)</title>
        <description>The efficacy of SSP-004184 was assessed by determining cardiac iron load. Cardiac MRI data were collected by using T2* standard procedures and used to determine iron load. A negative change from baseline indicates that iron load increased. A cycle consisted of 8 standard 6-weekly visits and the cycles were repeated each year for the duration of the study.</description>
        <time_frame>Baseline, Week 48 (Cycle 1)</time_frame>
        <population>FAS participants evaluable for this outcome.</population>
        <group_list>
          <group group_id="O1">
            <title>SSP-004184AQ</title>
            <description>Participants received SSP-004184AQ (magnesium salt of free acid or active form, that is, SSP-004184 [SPD602, FBS0701]) capsules orally at a total daily dose of 8-75 mg/kg/day (equivalent to SSP-004184 7-68 mg/kg/day) either once daily or twice daily at the discretion of investigator for up to a maximum of 3 years or until the sponsor decided to stop the study.</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline in Cardiac T2* Values at Week 48 (Cycle 1)</title>
          <description>The efficacy of SSP-004184 was assessed by determining cardiac iron load. Cardiac MRI data were collected by using T2* standard procedures and used to determine iron load. A negative change from baseline indicates that iron load increased. A cycle consisted of 8 standard 6-weekly visits and the cycles were repeated each year for the duration of the study.</description>
          <population>FAS participants evaluable for this outcome.</population>
          <units>milliseconds</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="9"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-3.47" spread="9.619"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
            </group_id_list>
            <non_inferiority_type>Superiority or Other (legacy)</non_inferiority_type>
            <p_value>0.2297</p_value>
            <p_value_desc>P-value was from paired t-test for log-transformed data at post treatment timepoint versus baseline.</p_value_desc>
            <method>paired t-test</method>
          </analysis>
        </analysis_list>
      </outcome>
      <outcome>
        <type>Primary</type>
        <title>Change From Baseline in Cardiac T2* Values at Week 24 (Cycle 2)</title>
        <description>The efficacy of SSP-004184 was assessed by determining cardiac iron load. Cardiac MRI data were collected by using T2* standard procedures and used to determine iron load. A negative change from baseline indicates that iron load increased. A cycle consisted of 8 standard 6-weekly visits and the cycles were repeated each year for the duration of the study.</description>
        <time_frame>Baseline, Week 24 (Cycle 2)</time_frame>
        <population>FAS participants evaluable for this outcome.</population>
        <group_list>
          <group group_id="O1">
            <title>SSP-004184AQ</title>
            <description>Participants received SSP-004184AQ (magnesium salt of free acid or active form, that is, SSP-004184 [SPD602, FBS0701]) capsules orally at a total daily dose of 8-75 mg/kg/day (equivalent to SSP-004184 7-68 mg/kg/day) either once daily or twice daily at the discretion of investigator for up to a maximum of 3 years or until the sponsor decided to stop the study.</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline in Cardiac T2* Values at Week 24 (Cycle 2)</title>
          <description>The efficacy of SSP-004184 was assessed by determining cardiac iron load. Cardiac MRI data were collected by using T2* standard procedures and used to determine iron load. A negative change from baseline indicates that iron load increased. A cycle consisted of 8 standard 6-weekly visits and the cycles were repeated each year for the duration of the study.</description>
          <population>FAS participants evaluable for this outcome.</population>
          <units>milliseconds</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="7"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-3.91" spread="5.551"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
            </group_id_list>
            <non_inferiority_type>Superiority or Other (legacy)</non_inferiority_type>
            <p_value>0.1248</p_value>
            <p_value_desc>P-value was from paired t-test for log-transformed data at post treatment timepoint versus baseline.</p_value_desc>
            <method>paired t-test</method>
          </analysis>
        </analysis_list>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change From Baseline in Serum Ferritin Values at Specified Visits</title>
        <description>A negative change from baseline indicates that serum ferritin decreased. A cycle consisted of 8 standard 6-weekly visits and the cycles were repeated each year for the duration of the study.</description>
        <time_frame>Baseline, Weeks 24 and 48 of Cycle 1, Week 24 of Cycle 2</time_frame>
        <population>FAS. Here, 'n' signifies the number of FAS participants evaluable for the respective time points.</population>
        <group_list>
          <group group_id="O1">
            <title>SSP-004184AQ</title>
            <description>Participants received SSP-004184AQ (magnesium salt of free acid or active form, that is, SSP-004184 [SPD602, FBS0701]) capsules orally at a total daily dose of 8-75 mg/kg/day (equivalent to SSP-004184 7-68 mg/kg/day) either once daily or twice daily at the discretion of investigator for up to a maximum of 3 years or until the sponsor decided to stop the study.</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline in Serum Ferritin Values at Specified Visits</title>
          <description>A negative change from baseline indicates that serum ferritin decreased. A cycle consisted of 8 standard 6-weekly visits and the cycles were repeated each year for the duration of the study.</description>
          <population>FAS. Here, 'n' signifies the number of FAS participants evaluable for the respective time points.</population>
          <units>nanogram per milliliter</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="23"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Baseline (n=23)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="5353.38" spread="4588.423"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Change at Week 24 (Cycle 1) (n=12)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-395.82" spread="569.305"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Change at Week 48 (Cycle 1) (n=9)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-250.87" spread="452.327"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Change at Week 24 (Cycle 2) (n=7)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-384.03" spread="649.990"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
    </outcome_list>

    <reported_events>
      <time_frame>From start of study treatment up to the end of study (1 week after the end of treatment)</time_frame>
      <desc>An adverse event (AE) that occurred during the study was considered a treatment-emergent AE (TEAE) if it had a start date and time on or after the study treatment or if it had a start date before the date and time of the study treatment but increased in intensity on or after the date and time of the study treatment.</desc>
      <group_list>
        <group group_id="E1">
          <title>SSP-004184AQ</title>
          <description>Participants received SSP-004184AQ (magnesium salt of free acid or active form, that is, SSP-004184 [SPD602, FBS0701]) capsules orally at a total daily dose of 8-75 mg/kg/day (equivalent to SSP-004184 7-68 mg/kg/day) either once daily or twice daily at the discretion of investigator for up to a maximum of 3 years or until the sponsor decided to stop the study.</description>
        </group>
      </group_list>
      <serious_events>
        <default_vocab>MedDRA (12.1)</default_vocab>
        <default_assessment>Non-systematic Assessment</default_assessment>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, serious adverse events</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="30"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Congenital, familial and genetic disorders</title>
            <event_list>
              <event>
                <sub_title>Sickle cell anaemia with crisis</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="30"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Infections and infestations</title>
            <event_list>
              <event>
                <sub_title>Bronchitis</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="30"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </serious_events>
      <other_events>
        <frequency_threshold>5</frequency_threshold>
        <default_vocab>MedDRA (12.1)</default_vocab>
        <default_assessment>Non-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="25" subjects_at_risk="30"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Gastrointestinal disorders</title>
            <event_list>
              <event>
                <sub_title>Nausea</sub_title>
                <counts group_id="E1" events="6" subjects_affected="4" subjects_at_risk="30"/>
              </event>
              <event>
                <sub_title>Abdominal pain upper</sub_title>
                <counts group_id="E1" events="3" subjects_affected="3" subjects_at_risk="30"/>
              </event>
              <event>
                <sub_title>Constipation</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="30"/>
              </event>
              <event>
                <sub_title>Diarrhoea</sub_title>
                <counts group_id="E1" events="3" subjects_affected="2" subjects_at_risk="30"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>General disorders</title>
            <event_list>
              <event>
                <sub_title>Influenza like illness</sub_title>
                <counts group_id="E1" events="6" subjects_affected="5" subjects_at_risk="30"/>
              </event>
              <event>
                <sub_title>Asthenia</sub_title>
                <counts group_id="E1" events="8" subjects_affected="4" subjects_at_risk="30"/>
              </event>
              <event>
                <sub_title>Pyrexia</sub_title>
                <counts group_id="E1" events="5" subjects_affected="2" subjects_at_risk="30"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Infections and infestations</title>
            <event_list>
              <event>
                <sub_title>Nasopharyngitis</sub_title>
                <counts group_id="E1" events="6" subjects_affected="5" subjects_at_risk="30"/>
              </event>
              <event>
                <sub_title>Pharyngitis</sub_title>
                <counts group_id="E1" events="3" subjects_affected="3" subjects_at_risk="30"/>
              </event>
              <event>
                <sub_title>Gastroenteritis</sub_title>
                <counts group_id="E1" events="4" subjects_affected="2" subjects_at_risk="30"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Nervous system disorders</title>
            <event_list>
              <event>
                <sub_title>Paraesthesia</sub_title>
                <counts group_id="E1" events="5" subjects_affected="4" subjects_at_risk="30"/>
              </event>
              <event>
                <sub_title>Headache</sub_title>
                <counts group_id="E1" events="3" subjects_affected="3" subjects_at_risk="30"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Psychiatric disorders</title>
            <event_list>
              <event>
                <sub_title>Depression</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="30"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Respiratory, thoracic and mediastinal disorders</title>
            <event_list>
              <event>
                <sub_title>Oropharyngeal pain</sub_title>
                <counts group_id="E1" events="6" subjects_affected="4" subjects_at_risk="30"/>
              </event>
              <event>
                <sub_title>Cough</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="30"/>
              </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>If a multicenter publication is not submitted within twelve (12) months after conclusion, abandonment or termination of the Study at all sites, or after Sponsor confirms there shall be no multicenter Study publication, the Institution and/or such Principal Investigator may publish the results from the Institution site individually.</restrictive_agreement>
    </certain_agreements>
    <limitations_and_caveats>This study was terminated early due to non-clinical safety results. Not all participants completed the study. The available efficacy data were analyzed as specified in the statistical analysis plan; however, no efficacy conclusions were drawn.</limitations_and_caveats>
    <point_of_contact>
      <name_or_title>Study Director</name_or_title>
      <organization>Shire</organization>
      <phone>+1 866 842 5335</phone>
      <email>ClinicalTransparency@shire.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/NCT01671111
org study id: SPD602-301
secondary id: 2011-006322-25
nct id: NCT01671111
lead sponsor: 
The purpose of this extension study is to evaluate SSP-004184AQ in patients with
      transfusional iron overload and to provide data on long term safety and efficacy.

      SSP-004184AQ is an iron chelator under development for chronic daily oral administration to
      patients with transfusional iron overload
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Change From Baseline in Ferriscan® R2 Liver Iron Concentrations (LIC) at Week 24 (Cycle 1)
time frame: Baseline, Week 24 (Cycle 1)
description: Efficacy of SSP-004184 was assessed by determining LIC. Abdominal magnetic resonance imaging (MRI) data were collected by using FerriScan R2 standard procedures and used to determine LIC. A negative change from baseline indicates that LIC decreased. A cycle consisted of 8 standard 6-weekly visits and the cycles were repeated each year for the duration of the study.
measure: Change From Baseline in Ferriscan® R2 Liver Iron Concentrations (LIC) at Week 48 (Cycle 1)
time frame: Baseline, Week 48 (Cycle 1)
description: Efficacy of SSP-004184 was assessed by determining LIC. Abdominal MRI data were collected by using FerriScan R2 standard procedures and used to determine LIC. A negative change from baseline indicates that LIC decreased. A cycle consisted of 8 standard 6-weekly visits and the cycles were repeated each year for the duration of the study.
measure: Change From Baseline in Ferriscan® R2 Liver Iron Concentrations (LIC) at Week 24 (Cycle 2)
time frame: Baseline, Week 24 (Cycle 2)
description: Efficacy of SSP-004184 was assessed by determining LIC. Abdominal MRI data were collected by using FerriScan R2 standard procedures and used to determine LIC. A negative change from baseline indicates that LIC decreased. A cycle consisted of 8 standard 6-weekly visits and the cycles were repeated each year for the duration of the study.
measure: Change From Baseline in Cardiac T2* Values at Week 24 (Cycle 1)
time frame: Baseline, Week 24 (Cycle 1)
description: The efficacy of SSP-004184 was assessed by determining cardiac iron load. Cardiac MRI data were collected by using T2* standard procedures and used to determine iron load. A negative change from baseline indicates that iron load increased. A cycle consisted of 8 standard 6-weekly visits and the cycles were repeated each year for the duration of the study.
measure: Change From Baseline in Cardiac T2* Values at Week 48 (Cycle 1)
time frame: Baseline, Week 48 (Cycle 1)
description: The efficacy of SSP-004184 was assessed by determining cardiac iron load. Cardiac MRI data were collected by using T2* standard procedures and used to determine iron load. A negative change from baseline indicates that iron load increased. A cycle consisted of 8 standard 6-weekly visits and the cycles were repeated each year for the duration of the study.
measure: Change From Baseline in Cardiac T2* Values at Week 24 (Cycle 2)
time frame: Baseline, Week 24 (Cycle 2)
description: The efficacy of SSP-004184 was assessed by determining cardiac iron load. Cardiac MRI data were collected by using T2* standard procedures and used to determine iron load. A negative change from baseline indicates that iron load increased. A cycle consisted of 8 standard 6-weekly visits and the cycles were repeated each year for the duration of the study.
measure: Change From Baseline in Serum Ferritin Values at Specified Visits
time frame: Baseline, Weeks 24 and 48 of Cycle 1, Week 24 of Cycle 2
description: A negative change from baseline indicates that serum ferritin decreased. A cycle consisted of 8 standard 6-weekly visits and the cycles were repeated each year for the duration of the study.
arm group label: SSP-004814AQ
arm group type: Experimental
intervention type: Drug
intervention name: SSP-004184AQ
description: Long term extension study of 8-75 mg/kg/d of SSP-004184AQ (equivalent to 7-68 mg/kg/d free acid or "active" form)
arm group label: SSP-004814AQ
other name: SPD602
criteria: 
gender: All
minimum age: 6 Years
maximum age: N/A
healthy volunteers: No
last name: Study Director
role: Study Director
affiliation: Takeda
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
country: Canada
country: Italy
country: Thailand
country: United Kingdom
country: United States
responsible party type: Sponsor
mesh term: Iron Overload
participant flow: 
baseline: 
outcome list: 
reported events: 
certain agreements: 
limitations and caveats: This study was terminated early due to non-clinical safety results. Not all participants completed the study. The available efficacy data were analyzed as specified in the statistical analysis plan; however, no efficacy conclusions were drawn.
point of contact: 