Study of ORL-1B in Patients With Biotinidase Deficiency

Orpha Labs

Status and phase: Completed; Phase 2; Phase 1

Conditions: Biotinidase Deficiency

Treatments: Drug: ORL-1B

Study type: Interventional

Funder types: Industry

Identifiers

NCT03269045

Biot-1

Details and patient eligibility

About

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

Enrollment: 20 patients

Sex: All

Ages: Under 18 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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20 participants in 1 patient group

Treatment with ORL-1B.

Other group

Description:

Pediatric patients with biotinidase deficiency.

Treatment:

Drug: ORL-1B