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. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT03269045 (ClinicalTrials.gov) | August 31, 2013 | 28/8/2017 | 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 | NULL | Completed | N/A | 18 Years | All | 20 | Phase 1;Phase 2 | NULL |