255. Multiple carboxylase deficiency
1 clinical trial,   1 drug   (DrugBank: 0 drug)   0 gene,   0 pathway

Searched query = "Multiple carboxylase deficiency", "Holocarboxylase synthetase deficiency", "HCS deficiency", "Biotinidase deficiency"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

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Public_titleScientific_titleConditionInterventionPrimary_
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Inclusion_
agemin
Inclusion_
agemax
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PhaseCountries
1NCT03269045
(ClinicalTrials.gov)
August 31, 201328/8/2017Study of ORL-1B in Patients With Biotinidase DeficiencyAn Open-label, Single-arm, Phase 2 Study of ORL-1B in Patients With Biotinidase DeficiencyBiotinidase DeficiencyDrug: ORL-1BOrpha LabsNULLCompletedN/A18 YearsAll20Phase 1;Phase 2NULL