21. ミトコンドリア病 Mitochondrial disease Clinical trials / Disease details
臨床試験数 : 33 / 薬物数 : 42 - (DrugBank : 32) / 標的遺伝子数 : 47 - 標的パスウェイ数 : 67
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 |