21. Mitochondrial disease Clinical trials / Disease details

Clinical trials : 33 / Drugs : 42 - (DrugBank : 32) / Drug target genes : 47 - Drug target pathways : 67

2 trials found

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