<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/NCT03269045</url>
  </required_header>
  <id_info>
    <org_study_id>Biot-1</org_study_id>
    <nct_id>NCT03269045</nct_id>
  </id_info>
  <brief_title>Study of ORL-1B in Patients With Biotinidase Deficiency</brief_title>
  <official_title>An Open-label, Single-arm, Phase 2 Study of ORL-1B in Patients With Biotinidase Deficiency</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Orpha Labs</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Orpha Labs</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
    <is_us_export>No</is_us_export>
  </oversight_info>
  <brief_summary>
    <textblock>
      An Open-label, Single-arm, Phase 2 Study of Biotin in Patients With Biotinidase Deficiency.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">August 31, 2013</start_date>
  <completion_date type="Actual">January 1, 2018</completion_date>
  <primary_completion_date type="Actual">January 1, 2018</primary_completion_date>
  <phase>Phase 1/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>Improvement in seizure frequency</measure>
    <time_frame>12 months</time_frame>
    <description>Statistically significant improvement in seizure frequency after the ORL-1B treatment.</description>
  </primary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">20</enrollment>
  <condition>Biotinidase Deficiency</condition>
  <arm_group>
    <arm_group_label>Treatment with ORL-1B.</arm_group_label>
    <arm_group_type>Other</arm_group_type>
    <description>Pediatric patients with biotinidase deficiency.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>ORL-1B</intervention_name>
    <description>Oral ORL-1B</description>
    <arm_group_label>Treatment with ORL-1B.</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Diagnosis of biotinidase deficiency.&#xD;
&#xD;
          -  Less than 18 years old.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Diagnosis of any other disease that is not a manifestation of biotinidase deficiency.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>N/A</minimum_age>
    <maximum_age>18 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <verification_date>January 2018</verification_date>
  <study_first_submitted>August 28, 2017</study_first_submitted>
  <study_first_submitted_qc>August 29, 2017</study_first_submitted_qc>
  <study_first_posted type="Actual">August 31, 2017</study_first_posted>
  <last_update_submitted>January 13, 2018</last_update_submitted>
  <last_update_submitted_qc>January 13, 2018</last_update_submitted_qc>
  <last_update_posted type="Actual">January 17, 2018</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>Biotinidase Deficiency</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </patient_data>
  <!-- Results have not yet been posted for this study                                          -->
</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/NCT03269045
org study id: Biot-1
nct id: NCT03269045
lead sponsor: 
has dmc: Yes
is fda regulated drug: No
is fda regulated device: No
is us export: No
An Open-label, Single-arm, Phase 2 Study of Biotin in Patients With Biotinidase Deficiency.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Improvement in seizure frequency
time frame: 12 months
description: Statistically significant improvement in seizure frequency after the ORL-1B treatment.
arm group label: Treatment with ORL-1B.
arm group type: Other
description: Pediatric patients with biotinidase deficiency.
intervention type: Drug
intervention name: ORL-1B
description: Oral ORL-1B
arm group label: Treatment with ORL-1B.
criteria: 
gender: All
minimum age: N/A
maximum age: 18 Years
healthy volunteers: No
responsible party type: Sponsor
mesh term: Biotinidase Deficiency
sharing ipd: No