8 completed, 2 no longer available, 2 terminated, 1 withdrawn. 10 Phase 2, 2 Phase 1, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 10 | Completed 7, Terminated 2, Withdrawn 1 |
| Phase 1 | 2 | Completed 2 |
| Phase 3 | 1 | Terminated 1 |
Follow EPI-743: new trials by email Literature Review of EPI-743 trials Research Report on EPI-743
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia Vatiquinone - Friedreich Ataxia | Phase 2 | Completed | PTC Therapeutics | 5 | 2022-10-12 | 2025-08-22 |
| A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy Vatiquinone, Placebo - Mitochondrial Diseases, Drug Resistant Epilepsy | Phase 2 / Phase 3 | Terminated | PTC Therapeutics | 68 | 2020-09-28 | 2026-03-31 |
| Protective Effects of EPI-743 on Noise-Induced Hearing Loss EPI-743, Placebo - Noise-induced Hearing Loss | Phase 2 | Completed | Edison Pharmaceuticals Inc | 77 | 2014-10-31 | 2020-11-18 |
| An Exploratory Open Label Study of EPI-743 (Vincerinone TM) in Children With Autism Spectrum Disorder EPI-743 - Autism Spectrum Disorder | Phase 2 | Withdrawn | Edison Pharmaceuticals Inc | 2014-10-31 | 2022-01-21 | |
| Phase 2 Study of EPI-743 in Children With Pearson Syndrome EPI-743 - Pearson Syndrome | Phase 2 | Terminated | Edison Pharmaceuticals Inc | 2 | 2014-08-31 | 2020-11-18 |
| Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome EPI-743 - Leigh Syndrome | Phase 2 | Completed | PTC Therapeutics | 30 | 2014-01-31 | 2024-10-23 |
| EPI-743 in Friedreich's Ataxia Point Mutations EPI-743 - Friedreich's Ataxia | Phase 2 | Completed | University of South Florida | 4 | 2013-10-01 | 2016-06-29 |
| Phase 2 Study of EPI-743 for Treatment of Rett Syndrome EPI-743, Placebo - Rett Syndrome | Phase 2 | Completed | Edison Pharmaceuticals Inc | 24 | 2013-01-01 | 2018-07-26 |
| EPI-743 in Cobalamin C Defect: Effects on Visual and Neurological Impairment Epi-743, Placebo supplementation - Methylmalonic Aciduria and Homocystinuria,Cblc Type, Genetic Disease | Phase 2 | Completed | Bambino Gesù Hospital and Research Institute | 30 | 2013-01-01 | 2017-04-25 |
| Open-Trial of EPI-743 for Adults With Tourette Syndrome EPI-743 - Tourette Syndrome | Phase 1 | Completed | Yale University | 10 | 2012-10-01 | 2014-03-05 |
| EPI-743 for Metabolism or Mitochondrial Disorders EPI-743, Placebo - Mitochondrial Disease, Neurology | Phase 1 / Phase 2 | Completed | National Human Genome Research Institute (NHGRI) | 20 | 2012-09-01 | 2021-04-14 |
| EPI-743 for Mitochondrial Respiratory Chain Diseases EPI-743 - Mitochondrial Diseases | No longer available | PTC Therapeutics | 2024-01-23 | |||
| Emergency Administration of EPI-743 to a Single Patient With Leber's Hereditary Optic Neuropathy [LHON] EPI-743 - Leber's Hereditary Optic Neuropathy | No longer available | Edison Pharmaceuticals Inc | 2014-11-25 |
Mitochondrial Diseases (6) · Friedreich Ataxia (3) · Leigh Disease (3) · MELAS Syndrome (2) · Rett Syndrome (2) · Angelman Syndrome (1) · Autism Spectrum Disorder (1) · Autistic Disorder (1) · Diffuse Cerebral Sclerosis of Schilder (1) · Drug Resistant Epilepsy (1) · Genetic Diseases, Inborn (1) · Hearing Loss, Noise-Induced (1) · MERRF Syndrome (1) · Methylmalonic acidemia with homocystinuria (1) · Mitochondrial encephalopathy (1)
Edison Pharmaceuticals Inc (5) · PTC Therapeutics (4) · Bambino Gesù Hospital and Research Institute (1) · National Human Genome Research Institute (NHGRI) (1) · University of South Florida (1) · Yale University (1)
United States (10) · Italy (3) · Canada (1) · France (1) · Japan (1) · Poland (1) · Spain (1) · Sweden (1) · United Kingdom (1)
Bruce Cohen, MD (1) · Carlo Dionisi-Vici, MD (1) · Colleen Le Prell, Ph.D (1) · Daniela Ricci, MD,PhD (1) · Diego Martinelli, MD, PhD (1) · Giancarlo Iarossi, MD (1) · Gregory Enns, MB, ChB (1) · Matthew Klein, MD, FACS (1) · Michael H Bloch, MD, MS (1) · Theresa A Zesiewicz, MD (1) · Vinay Penematsa, MD (1) · William A Gahl, M.D. (1)