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
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agemin
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1NCT02473445
(ClinicalTrials.gov)
May 19, 201510/6/2015A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial DiseaseA 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 DiseaseMitochondrial DiseasesDrug: Cysteamine BitartrateHorizon Pharma USA, Inc.NULLTerminated6 Years17 YearsAll22Phase 2United States
2NCT02023866
(ClinicalTrials.gov)
May 201417/12/2013Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial DiseaseAn 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 DiseaseInherited Mitochondrial Disease, Including Leigh SyndromeDrug: Cysteamine BitartrateHorizon Pharma USA, Inc.NULLCompleted6 Years17 YearsAll36Phase 2United States