<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/NCT01734889</url>
  </required_header>
  <id_info>
    <org_study_id>Sobi.NTBC-002</org_study_id>
    <nct_id>NCT01734889</nct_id>
  </id_info>
  <brief_title>Taste and Palatability of Orfadin Suspension</brief_title>
  <official_title>Taste and Palatability of Orfadin Suspension. An Open, Non-controlled 3 Day Study in Pediatric Patients With Hereditary Tyrosinemia Type 1 Treated With Orfadin.</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Swedish Orphan Biovitrum</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Swedish Orphan Biovitrum</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      The purpose of this study is to verify that pediatric patients, especially those who are not&#xD;
      old enough to swallow capsules, accept the taste and palatability of a new suspension.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This is an open, non-randomized, non-controlled, multiple-dose study in 18 pediatric&#xD;
      patients. The treatment period is three days, and during the study the subjects will rate the&#xD;
      taste and palatability of the suspension or (for younger children) their parents will rate&#xD;
      the child´s acceptance of the suspension.&#xD;
&#xD;
      The study consists of a screening period, a 3 day treatment period and a 1 week follow-up&#xD;
      period.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date>October 2012</start_date>
  <completion_date type="Actual">March 2013</completion_date>
  <primary_completion_date type="Actual">March 2013</primary_completion_date>
  <phase>Phase 1</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>The Taste Score for the Last Dose of the Suspension on Day 3 for Subjects 5 - &lt;18 Years</measure>
    <time_frame>Day 3</time_frame>
    <description>Patients rated the taste of the suspension. The following grading was applied: 5 (very good taste), 4 (good taste), 3 (neither good nor bad taste), 2 (bad taste) and 1 (very bad taste).</description>
  </primary_outcome>
  <primary_outcome>
    <measure>The Acceptability Score for the Last Dose of the Suspension on Day 3 for Subjects &lt; 5 Years</measure>
    <time_frame>Day 3</time_frame>
    <description>The parents of patients aged &lt;5 years rated their child´s acceptability of the suspension. The following grading was applied: 5 (very well), 4 (well), 3 (neither well nor badly), 2 (badly) and 1 (very badly).</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>The Palatability Scores on Day 1 (Subjects 5 - &lt; 18 Years)</measure>
    <time_frame>Day 1</time_frame>
    <description>Patients rated the palatability of the suspension. The following grading was applied: 5 (very good), 4 (good), 3 (neither good nor bad), 2 (bad) and 1 (very bad).</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The Palatability Scores on Day 2 (Subjects 5 - &lt; 18 Years)</measure>
    <time_frame>Day 2</time_frame>
    <description>Patients rated the palatability of the suspension. The following grading was applied: 5 (very good), 4 (good), 3 (neither good nor bad), 2 (bad) and 1 (very bad).</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The Palatability Scores on Day 3 (Subjects 5 - &lt; 18 Years)</measure>
    <time_frame>Day 3</time_frame>
    <description>Patients rated the palatability of the suspension. The following grading was applied: 5 (very good), 4 (good), 3 (neither good nor bad), 2 (bad) and 1 (very bad).</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">18</enrollment>
  <condition>Hereditary Tyrosinemia, Type I</condition>
  <arm_group>
    <arm_group_label>Orfadin suspension</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Drug: nitisinone, oral suspension</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Nitisinone</intervention_name>
    <description>Oral suspension</description>
    <arm_group_label>Orfadin suspension</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Patients with HT-1 currently managed on Orfadin (nitisinone) capsules.&#xD;
&#xD;
          -  Age from 1 month to less than 18 years.&#xD;
&#xD;
          -  Signed informed consent.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Any medical condition which in the opinion of the investigator makes the subject&#xD;
             unsuitable for inclusion.&#xD;
&#xD;
          -  Enrollment in another concurrent clinical study, or intake of an investigational&#xD;
             medicinal product (IMP), within one month prior to inclusion in this study.&#xD;
&#xD;
          -  Foreseeable inability to cooperate with given instructions or study procedures.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>1 Month</minimum_age>
    <maximum_age>17 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Erik Brouwer, MD</last_name>
    <role>Study Director</role>
    <affiliation>Swedish Orphan Biovitrum</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Hopital Necker</name>
      <address>
        <city>Paris</city>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Universitätsklinikum Düsseldorf</name>
      <address>
        <city>Düsseldorf</city>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Universitätsklinikum Heidelberg</name>
      <address>
        <city>Heidelberg</city>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Klinikum der Universität München</name>
      <address>
        <city>München</city>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Birmingham Children's Hospital</name>
      <address>
        <city>Birmingham</city>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Evelina Children's Hospital, St Thomas' Hospital</name>
      <address>
        <city>London</city>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>St Mary's Hospital</name>
      <address>
        <city>Manchester</city>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>France</country>
    <country>Germany</country>
    <country>United Kingdom</country>
  </location_countries>
  <verification_date>October 2014</verification_date>
  <study_first_submitted>November 22, 2012</study_first_submitted>
  <study_first_submitted_qc>November 27, 2012</study_first_submitted_qc>
  <study_first_posted type="Estimate">November 28, 2012</study_first_posted>
  <results_first_submitted>January 21, 2014</results_first_submitted>
  <results_first_submitted_qc>January 21, 2014</results_first_submitted_qc>
  <results_first_posted type="Estimate">March 4, 2014</results_first_posted>
  <last_update_submitted>October 31, 2014</last_update_submitted>
  <last_update_submitted_qc>October 31, 2014</last_update_submitted_qc>
  <last_update_posted type="Estimate">November 6, 2014</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>nitisinone</keyword>
  <keyword>Orfadin</keyword>
  <keyword>suspension</keyword>
  <keyword>taste</keyword>
  <keyword>acceptability</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Tyrosinemias</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Nitisinone</mesh_term>
  </intervention_browse>

  <clinical_results>

    <participant_flow>
      <recruitment_details>The study recruitment was performed at seven medical clinics in three countries (United Kingdom, Germany and France). First subject was enrolled on 09 November 2012 and last subject´s last visit was on 01 March 2013.</recruitment_details>
      <group_list>
        <group group_id="P1">
          <title>Orfadin Suspension</title>
          <description>Drug: nitisinone, oral suspension 4 mg/mL, twice daily dosing, total daily dose according to current prescribed dose at screening</description>
        </group>
      </group_list>
      <period_list>
        <period>
          <title>Overall Study</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="18"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="18"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="0"/>
              </participants_list>
            </milestone>
          </milestone_list>
        </period>
      </period_list>
    </participant_flow>

    <baseline>
      <group_list>
        <group group_id="B1">
          <title>Orfadin Suspension</title>
          <description>Drug: nitisinone, oral suspension 4 mg/mL, twice daily dosing, total daily dose according to current prescribed dose at screening</description>
        </group>
      </group_list>
      <analyzed_list>
        <analyzed>
          <units>Participants</units>
          <scope>Overall</scope>
          <count_list>
            <count group_id="B1" value="18"/>
          </count_list>
        </analyzed>
      </analyzed_list>
      <measure_list>
        <measure>
          <title>Age</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>&lt;=18 years</title>
                  <measurement_list>
                    <measurement group_id="B1" value="18"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Between 18 and 65 years</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>&gt;=65 years</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <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="8.03" spread="5.60"/>
                  </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="9"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Male</title>
                  <measurement_list>
                    <measurement group_id="B1" value="9"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Region of Enrollment</title>
          <units>participants</units>
          <param>Number</param>
          <class_list>
            <class>
              <title>France</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="2"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Germany</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="4"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>United Kingdom</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="12"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </measure_list>
    </baseline>

    <outcome_list>
      <outcome>
        <type>Primary</type>
        <title>The Taste Score for the Last Dose of the Suspension on Day 3 for Subjects 5 - &lt;18 Years</title>
        <description>Patients rated the taste of the suspension. The following grading was applied: 5 (very good taste), 4 (good taste), 3 (neither good nor bad taste), 2 (bad taste) and 1 (very bad taste).</description>
        <time_frame>Day 3</time_frame>
        <population>Full analysis set: All subjects who received at least one dose of study drug and had at least one taste or acceptability assessments</population>
        <group_list>
          <group group_id="O1">
            <title>Age 5-&lt;18 Years</title>
            <description>Drug: nitisinone, oral suspension 4 mg/mL, twice daily dosing, total daily dose according to current prescribed dose at screening</description>
          </group>
        </group_list>
        <measure>
          <title>The Taste Score for the Last Dose of the Suspension on Day 3 for Subjects 5 - &lt;18 Years</title>
          <description>Patients rated the taste of the suspension. The following grading was applied: 5 (very good taste), 4 (good taste), 3 (neither good nor bad taste), 2 (bad taste) and 1 (very bad taste).</description>
          <population>Full analysis set: All subjects who received at least one dose of study drug and had at least one taste or acceptability assessments</population>
          <units>units on a scale</units>
          <param>Median</param>
          <dispersion>Full Range</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="12"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="4.0" lower_limit="2.0" upper_limit="5.0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Primary</type>
        <title>The Acceptability Score for the Last Dose of the Suspension on Day 3 for Subjects &lt; 5 Years</title>
        <description>The parents of patients aged &lt;5 years rated their child´s acceptability of the suspension. The following grading was applied: 5 (very well), 4 (well), 3 (neither well nor badly), 2 (badly) and 1 (very badly).</description>
        <time_frame>Day 3</time_frame>
        <population>Full analysis set: All subjects who received at least one dose of study drug and had at least one taste or acceptability assessments</population>
        <group_list>
          <group group_id="O1">
            <title>Age &lt;5 Years</title>
            <description>Drug: nitisinone, oral suspension 4 mg/mL, twice daily dosing, total daily dose according to current prescribed dose at screening</description>
          </group>
        </group_list>
        <measure>
          <title>The Acceptability Score for the Last Dose of the Suspension on Day 3 for Subjects &lt; 5 Years</title>
          <description>The parents of patients aged &lt;5 years rated their child´s acceptability of the suspension. The following grading was applied: 5 (very well), 4 (well), 3 (neither well nor badly), 2 (badly) and 1 (very badly).</description>
          <population>Full analysis set: All subjects who received at least one dose of study drug and had at least one taste or acceptability assessments</population>
          <units>units on a scale</units>
          <param>Median</param>
          <dispersion>Full Range</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="6"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="5.0" lower_limit="4.0" upper_limit="5.0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>The Palatability Scores on Day 1 (Subjects 5 - &lt; 18 Years)</title>
        <description>Patients rated the palatability of the suspension. The following grading was applied: 5 (very good), 4 (good), 3 (neither good nor bad), 2 (bad) and 1 (very bad).</description>
        <time_frame>Day 1</time_frame>
        <population>Full analysis set: All subjects who received at least one dose of study drug and had at least taste or acceptability assessment.</population>
        <group_list>
          <group group_id="O1">
            <title>Age 5-&lt;18 Years</title>
            <description>Drug: nitisinone, oral suspension 4 mg/mL, twice daily dosing, total daily dose according to current prescribed dose at screening</description>
          </group>
        </group_list>
        <measure>
          <title>The Palatability Scores on Day 1 (Subjects 5 - &lt; 18 Years)</title>
          <description>Patients rated the palatability of the suspension. The following grading was applied: 5 (very good), 4 (good), 3 (neither good nor bad), 2 (bad) and 1 (very bad).</description>
          <population>Full analysis set: All subjects who received at least one dose of study drug and had at least taste or acceptability assessment.</population>
          <units>units on a scale</units>
          <param>Median</param>
          <dispersion>Full Range</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="12"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="4.0" lower_limit="3.0" upper_limit="5.0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>The Palatability Scores on Day 2 (Subjects 5 - &lt; 18 Years)</title>
        <description>Patients rated the palatability of the suspension. The following grading was applied: 5 (very good), 4 (good), 3 (neither good nor bad), 2 (bad) and 1 (very bad).</description>
        <time_frame>Day 2</time_frame>
        <population>Full analysis set: All subjects who received at least one dose of study drug and had at least taste or acceptability assessment.</population>
        <group_list>
          <group group_id="O1">
            <title>Age 5-&lt;18 Years</title>
            <description>Drug: nitisinone, oral suspension 4 mg/mL, twice daily dosing, total daily dose according to current prescribed dose at screening</description>
          </group>
        </group_list>
        <measure>
          <title>The Palatability Scores on Day 2 (Subjects 5 - &lt; 18 Years)</title>
          <description>Patients rated the palatability of the suspension. The following grading was applied: 5 (very good), 4 (good), 3 (neither good nor bad), 2 (bad) and 1 (very bad).</description>
          <population>Full analysis set: All subjects who received at least one dose of study drug and had at least taste or acceptability assessment.</population>
          <units>units on a scale</units>
          <param>Median</param>
          <dispersion>Full Range</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="12"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="4.0" lower_limit="2.0" upper_limit="5.0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>The Palatability Scores on Day 3 (Subjects 5 - &lt; 18 Years)</title>
        <description>Patients rated the palatability of the suspension. The following grading was applied: 5 (very good), 4 (good), 3 (neither good nor bad), 2 (bad) and 1 (very bad).</description>
        <time_frame>Day 3</time_frame>
        <population>Full analysis set: All subjects who received at least one dose of study drug and had at least taste or acceptability assessment.</population>
        <group_list>
          <group group_id="O1">
            <title>Age 5-&lt;18 Years</title>
            <description>Drug: nitisinone, oral suspension 4 mg/mL, twice daily dosing, total daily dose according to current prescribed dose at screening</description>
          </group>
        </group_list>
        <measure>
          <title>The Palatability Scores on Day 3 (Subjects 5 - &lt; 18 Years)</title>
          <description>Patients rated the palatability of the suspension. The following grading was applied: 5 (very good), 4 (good), 3 (neither good nor bad), 2 (bad) and 1 (very bad).</description>
          <population>Full analysis set: All subjects who received at least one dose of study drug and had at least taste or acceptability assessment.</population>
          <units>units on a scale</units>
          <param>Median</param>
          <dispersion>Full Range</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="12"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="4.0" lower_limit="2.0" upper_limit="5.0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
    </outcome_list>

    <reported_events>
      <time_frame>Adverse events were recorded upon administration of first dose until last study visit 1 week after last dose. Serious Adverse Events were recorded from subject signed informed consent until 28 days after last dose.</time_frame>
      <group_list>
        <group group_id="E1">
          <title>Orfadin Suspension</title>
          <description>Drug: nitisinone, oral suspension 4 mg/mL, twice daily dosing, total daily dose according to current prescribed dose at screening</description>
        </group>
      </group_list>
      <serious_events>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, serious adverse events</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="18"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </serious_events>
      <other_events>
        <frequency_threshold>0</frequency_threshold>
        <default_vocab>MedDRA 15.1</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="4" subjects_at_risk="18"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Gastrointestinal disorders</title>
            <event_list>
              <event>
                <sub_title>Regurgitation</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="18"/>
              </event>
              <event>
                <sub_title>Diarrhoea</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="18"/>
              </event>
              <event>
                <sub_title>Mouth haemorrhage</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="18"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>General disorders</title>
            <event_list>
              <event>
                <sub_title>Pyrexia</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="18"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Infections and infestations</title>
            <event_list>
              <event>
                <sub_title>Nasopharyngitis</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="18"/>
              </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 results are intended to be published as a whole. If no comprehensive publication appears within 12 months of the trial termination, Investigators have the right to publish their results. Such data to be submitted to the Sponsor for review at least 60 days prior to submission for publication, public dissemination, or review by a publication committee. The Sponsor shall be entitled to make a reasoned request that publication be delayed for a period of up to 6 months.</restrictive_agreement>
    </certain_agreements>
    <point_of_contact>
      <name_or_title>Medical Director</name_or_title>
      <organization>Swedish Orphan Biovitrum</organization>
      <phone>+46 8 697 20 00</phone>
      <email>erik.brouwer@sobi.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/NCT01734889
org study id: Sobi.NTBC-002
nct id: NCT01734889
lead sponsor: 
has dmc: No
The purpose of this study is to verify that pediatric patients, especially those who are not
      old enough to swallow capsules, accept the taste and palatability of a new suspension.
This is an open, non-randomized, non-controlled, multiple-dose study in 18 pediatric
      patients. The treatment period is three days, and during the study the subjects will rate the
      taste and palatability of the suspension or (for younger children) their parents will rate
      the child´s acceptance of the suspension.

      The study consists of a screening period, a 3 day treatment period and a 1 week follow-up
      period.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: The Taste Score for the Last Dose of the Suspension on Day 3 for Subjects 5 - <18 Years
time frame: Day 3
description: Patients rated the taste of the suspension. The following grading was applied: 5 (very good taste), 4 (good taste), 3 (neither good nor bad taste), 2 (bad taste) and 1 (very bad taste).
measure: The Acceptability Score for the Last Dose of the Suspension on Day 3 for Subjects < 5 Years
time frame: Day 3
description: The parents of patients aged <5 years rated their child´s acceptability of the suspension. The following grading was applied: 5 (very well), 4 (well), 3 (neither well nor badly), 2 (badly) and 1 (very badly).
measure: The Palatability Scores on Day 1 (Subjects 5 - < 18 Years)
time frame: Day 1
description: Patients rated the palatability of the suspension. The following grading was applied: 5 (very good), 4 (good), 3 (neither good nor bad), 2 (bad) and 1 (very bad).
measure: The Palatability Scores on Day 2 (Subjects 5 - < 18 Years)
time frame: Day 2
description: Patients rated the palatability of the suspension. The following grading was applied: 5 (very good), 4 (good), 3 (neither good nor bad), 2 (bad) and 1 (very bad).
measure: The Palatability Scores on Day 3 (Subjects 5 - < 18 Years)
time frame: Day 3
description: Patients rated the palatability of the suspension. The following grading was applied: 5 (very good), 4 (good), 3 (neither good nor bad), 2 (bad) and 1 (very bad).
arm group label: Orfadin suspension
arm group type: Experimental
description: Drug: nitisinone, oral suspension
intervention type: Drug
intervention name: Nitisinone
description: Oral suspension
arm group label: Orfadin suspension
criteria: 
gender: All
minimum age: 1 Month
maximum age: 17 Years
healthy volunteers: No
last name: Erik Brouwer, MD
role: Study Director
affiliation: Swedish Orphan Biovitrum
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
country: France
country: Germany
country: United Kingdom
responsible party type: Sponsor
mesh term: Tyrosinemias
mesh term: Nitisinone
participant flow: 
baseline: 
outcome list: 
reported events: 
certain agreements: 
point of contact: 