Study of ORL-1B in Patients With Biotinidase Deficiency

• Status: Completed
• Trial end date: 2018-01-01
• Target enrollment: 0
• Participant gender: All

Summary

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

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Orpha Labs
• Treatments: Biotin

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.