Study of ORL-1B in Patients With Biotinidase Deficiency

NCT03269045 — Phase 1 / Phase 2, Completed, 20 participants, Orpha Labs

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

Registry	NCT03269045
Status	Completed
Phase	Phase 1 / Phase 2
Study type	Interventional; na, single group, masking: none, treatment
Enrollment	20 (Actual)
Sponsor	Orpha Labs (Industry)
Conditions	Biotinidase Deficiency
Interventions	ORL-1B (Drug)
Dates	start 2013-08-31, primary completion 2018-01-01, completion 2018-01-01, first posted 2017-08-31, last updated 2018-01-17

-Summary

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

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+Arms and interventions

Arm	Type	Interventions	Description
Treatment with ORL-1B.	Other	Drug: ORL-1B	Pediatric patients with biotinidase deficiency.

ORL-1B (Drug): Oral ORL-1B

+Outcome measures

Primary outcomes

Improvement in seizure frequency [12 months]: Statistically significant improvement in seizure frequency after the ORL-1B treatment.

+Eligibility

Sex: All, ages: any to 18 Years

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.