Try the modernized beta website. Learn more about the modernization effort.
Working… Menu

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. Identifier: NCT03269045
Recruitment Status : Completed
First Posted : August 31, 2017
Last Update Posted : January 17, 2018
Information provided by (Responsible Party):
Orpha Labs

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

Layout table for study information
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

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

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

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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

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.
Layout table for additonal information
Responsible Party: Orpha Labs 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

Layout table for additional information
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:
Layout table for MeSH terms
Biotinidase Deficiency
Multiple Carboxylase Deficiency
Amino Acid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Carbohydrate Metabolism, Inborn Errors
Metabolic Diseases