Study of ORL-1B in Patients With Biotinidase Deficiency
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|ClinicalTrials.gov Identifier: NCT03269045|
Recruitment Status : Completed
First Posted : August 31, 2017
Last Update Posted : January 17, 2018
Information provided by (Responsible Party):
An Open-label, Single-arm, Phase 2 Study of Biotin in Patients With Biotinidase Deficiency.
|Condition or disease||Intervention/treatment||Phase|
|Biotinidase Deficiency||Drug: ORL-1B||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||20 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||An Open-label, Single-arm, Phase 2 Study of ORL-1B in Patients With Biotinidase Deficiency|
|Actual Study Start Date :||August 31, 2013|
|Actual Primary Completion Date :||January 1, 2018|
|Actual Study Completion Date :||January 1, 2018|
Treatment with ORL-1B.
Pediatric patients with biotinidase deficiency.
Primary Outcome Measures :
- Improvement in seizure frequency [ Time Frame: 12 months ]Statistically significant improvement in seizure frequency after the ORL-1B treatment.
No Contacts or Locations Provided