Study of ORL-1B in Patients With Biotinidase Deficiency

Phase 2CompletedNCT03269045

Orpha Labs20 enrolled

Overview

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

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Biotinidase Deficiency

Interventions

ORL-1BDRUG

Oral ORL-1B

Eligibility

Sex: ALLMax age: 18 Years

Medical Language ↔ Plain English

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.

Outcomes

Primary Outcomes

Improvement in seizure frequency

Statistically significant improvement in seizure frequency after the ORL-1B treatment.

Time frame: 12 months

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.