21. ミトコンドリア病
［臨床試験数：23，薬物数：29（DrugBank：28），標的遺伝子数：44，標的パスウェイ数：65］
Searched query = "Mitochondrial disease"
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 | NCT02473445 (ClinicalTrials.gov) | May 19, 2015 | 10/6/2015 | A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease | A Long-Term Open-Label Extension Study of RP103-MITO-001 to Assess the Safety, Tolerability and Efficacy of Cysteamine Bitartrate Delayed-release Capsules (RP103) for Treatment of Children With Inherited Mitochondrial Disease | Mitochondrial Diseases | Drug: Cysteamine Bitartrate | Horizon Pharma USA, Inc. | NULL | Terminated | 6 Years | 17 Years | All | 22 | Phase 2 | United States |
| 2 | NCT02023866 (ClinicalTrials.gov) | May 2014 | 17/12/2013 | Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease | An Open-Label, Dose-Escalating Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics and Pharmacodynamics of Cysteamine Bitartrate Delayed-release Capsules (RP103) for Treatment of Children With Inherited Mitochondrial Disease | Inherited Mitochondrial Disease, Including Leigh Syndrome | Drug: Cysteamine Bitartrate | Horizon Pharma USA, Inc. | NULL | Completed | 6 Years | 17 Years | All | 36 | Phase 2 | United States |