Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease
Status:
Completed
Trial end date:
2016-10-01
Target enrollment:
Participant gender:
Summary
To evaluate safety, tolerability and efficacy of cysteamine bitartrate delayed-release
capsules (RP103) administered up to 1.3 g/m²/day in two divided doses, every 12 hours, for up
to 6 months in patients with inherited mitochondrial disease.
Phase:
Phase 2
Details
Lead Sponsor:
Horizon Pharma USA, Inc. Raptor Pharmaceuticals Inc.