255. 複合カルボキシラーゼ欠損症 ［臨床試験数：1，薬物数：1（DrugBank：0），標的遺伝子数：0，標的パスウェイ数：0］
Searched query = "Multiple carboxylase deficiency", "Holocarboxylase synthetase deficiency", "HCS deficiency", "Biotinidase deficiency"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT03269045 | August 31, 2013 | 22 January 2018 | 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 | Biotinidase Deficiency | Drug: ORL-1B | Orpha Labs | Not recruiting | N/A | 18 Years | All | 20 | Phase 1/Phase 2 |