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Study of ORL-1B in Patients With Biotinidase Deficiency

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03269045
Recruitment Status : Completed
First Posted : August 31, 2017
Last Update Posted : January 17, 2018
Sponsor:
Information provided by (Responsible Party):
Orpha Labs

Study Description
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Brief Summary:
An Open-label, Single-arm, Phase 2 Study of Biotin in Patients With Biotinidase Deficiency.

Condition or disease Intervention/treatment Phase
Biotinidase Deficiency Drug: ORL-1B Phase 1 Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 20 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-label, Single-arm, Phase 2 Study of ORL-1B in Patients With Biotinidase Deficiency
Actual Study Start Date : August 31, 2013
Actual Primary Completion Date : January 1, 2018
Actual Study Completion Date : January 1, 2018

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Treatment with ORL-1B.
Pediatric patients with biotinidase deficiency.
 Drug: ORL-1B
Oral ORL-1B


Outcome Measures
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Primary Outcome Measures :
  1. Improvement in seizure frequency [ Time Frame: 12 months ]
    Statistically significant improvement in seizure frequency after the ORL-1B treatment.


Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   up to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Diagnosis of biotinidase deficiency.
  • Less than 18 years old.

Exclusion Criteria:

  • Diagnosis of any other disease that is not a manifestation of biotinidase deficiency.
Contacts and Locations
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No Contacts or Locations Provided
More Information
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Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
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Responsible Party: Orpha Labs
ClinicalTrials.gov Identifier: NCT03269045    
Other Study ID Numbers: Biot-1
First Posted: August 31, 2017    Key Record Dates
Last Update Posted: January 17, 2018
Last Verified: January 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
Additional relevant MeSH terms:
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Biotinidase Deficiency
Multiple Carboxylase Deficiency
Amino Acid Metabolism, Inborn Errors
Metabolism, Inborn Errors
 Genetic Diseases, Inborn
Carbohydrate Metabolism, Inborn Errors
Metabolic Diseases