| Status | Study |
| Recruiting |
Study Name: The Leigh Syndrome Registry Condition: Leigh Syndrome Leigh Disease Date: 2017-04-28 |
| Completed |
Study Name: A Long-Term Extension Study of RP103-MITO-001 (NCT02023866) to Assess RP103 in Children With Inherited Mitochondrial Disease Condition: Mitochondrial Diseases Date: 2015-06-10 Interventions: Drug: Cysteamine Bitartrate Delayed-release Capsules Treatment with experimental drug as detailed in arm |
| Completed |
Study Name: A Dose-escalating Clinical Trial With KH176 Condition: MELAS LHON Leigh Syndrome Date: 2015-04-19 Interventions: Drug: KH176 Drug: placebo |
| Enrolling by invitation |
Study Name: Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome Condition: Leigh Syndrome Date: 2014-08-21 Interventions: Drug: EPI-743 EPI-743 is a quinone oxidation product of alpha tocotrienol |
| Completed |
Study Name: Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease Condition: Inherited Mitochondrial Disease, Including Leigh Syndrome Date: 2013-12-17 Interventions: Drug: Cysteamine Bitartrate Cysteamine Bitartrate Delayed-release Capsules |
| Recruiting |
Study Name: Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford Condition: Rare Disorders Undiagnosed Disorders Disorders of Unknown Date: 2013-02-13 |
| Completed |
Study Name: Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome Condition: Leigh Syndrome Date: 2012-11-01 Interventions: Drug: Placebo Drug: EPI-743 15 mg/kg |
| Recruiting |
Study Name: North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC) Condition: Mitochondrial Disorders Mitochondrial Genetic Disorders Mi Date: 2012-09-25 |
| Enrolling by invitation |
Study Name: Tissue Sample Study for Mitochondrial Disorders Condition: Mitochondrial Disorders Mitochondrial Disease Melas Date: 2012-08-31 |
| Active, not recruiting |
Study Name: EPI-743 for Mitochondrial Respiratory Chain Diseases Condition: Mitochondrial Diseases Date: 2011-06-07 Interventions: Drug: EPI-743 EPI-743 is administered as a dose escalation from 50 mg bid to 100 mg tid |