A Phase 1/2 interventional study of ORL-1B in Biotinidase Deficiency, sponsored by Orpha Labs. Completed. Open to participants aged Up to 18 Years. Per ClinicalTrials.gov, last updated 2018-01-17.
Sponsored by Orpha Labs · Phase 1/2, Interventional, and Treatment
An Open-label, Single-arm, Phase 2 Study of Biotin in Patients With Biotinidase Deficiency.
Exclusion Criteria:
Pediatric patients with biotinidase deficiency.
Drug: ORL-1B
Oral ORL-1B
Improvement in seizure frequency
Statistically significant improvement in seizure frequency after the ORL-1B treatment.
Time frame: 12 months
No study locations are listed for this record.
Plan to share: No
No publications or documents are linked to this record.
This study is completed, as verified in Jan 2018. You cannot join it, but the record below documents what was studied.
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Biotinidase Deficiency