Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: Open-Label, Dose-Escalating Study to Assess Safety, Tolerability, Efficacy, Pharmacokinetics and Pharmacodynamics of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited M
Brief Summary: Patients with mitochondrial diseases, who are ≥ 2 years old and < 18 years, will be included in this study. It is anticipated that approximately one half of subjects will have genetically confirmed Leigh Syndrome. Up to 25 patients will be enrolled if there is no toxicity up to the level of 1.3 g/m2/day of RP103. Interim analyses will occur after 4 and then 12 subjects complete the study through Week 24. If the study is not stopped early, final analysis will occur after 25 subjects have completed through Week 24.