Study of ORL-1B in Patients With Biotinidase Deficiency

An Open-label, Single-arm, Phase 2 Study of ORL-1B in Patients With Biotinidase Deficiency

Status

Completed

Phases

Phase 1Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT03269045

Enrollment

Registered

2017-08-31

Start date

2013-08-31

Completion date

2018-01-01

Last updated

2018-01-17

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Biotinidase Deficiency

Brief summary

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

Interventions

DRUGORL-1B

Oral ORL-1B

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

No minimum to 18 Years

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.

Design outcomes

Primary

Measure	Time frame	Description
Improvement in seizure frequency	12 months	Statistically significant improvement in seizure frequency after the ORL-1B treatment.

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026