Study of ORL-1B in Patients With Biotinidase Deficiency

Phase 1

Completed

• Conditions: Biotinidase Deficiency
• Interventions: Drug: ORL-1B

• Registration Number: NCT03269045
• Lead Sponsor: Orpha Labs

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study Start: 2013-08-31
• Study First Submitted: 2017-08-28
• Study First Submitted QC: 2017-08-29
• Study First Posted: 2017-08-31
• Status Verified: 2018-01
• Primary Completion: 2018-01-01
• Study Completion: 2018-01-01
• Last Update Submitted: 2018-01-13
• Last Update Posted: 2018-01-17

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 20

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Treatment with ORL-1B.	ORL-1B	Pediatric patients with biotinidase deficiency.

Primary Outcome Measures

Name	Time	Method
Improvement in seizure frequency	12 months	Statistically significant improvement in seizure frequency after the ORL-1B treatment.