3 completed, 2 no longer available, 1 active not recruiting, 1 not yet recruiting. 5 Phase 3, 3 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 5 | Completed 2, Active not recruiting 1, Not yet recruiting 1, Terminated 1 |
| Phase 2 | 3 | Completed 2, Terminated 1 |
Follow Vatiquinone: new trials by email Literature Review of Vatiquinone trials Research Report on Vatiquinone
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 |
|---|---|---|---|---|---|---|
| Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA) Vatiquinone - Friedreich's Ataxia | Phase 3 | Not yet recruiting | PTC Therapeutics | 120 | 2026-08-15 | 2026-08-14 |
| A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia Vatiquinone - Friedreich Ataxia | Phase 3 | Active not recruiting | PTC Therapeutics | 130 | 2022-12-08 | 2025-10-30 |
| 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 Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease Vatiquinone - Inherited Mitochondrial Disease | Phase 3 | Completed | PTC Therapeutics | 101 | 2022-06-22 | 2025-12-22 |
| A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia Vatiquinone, Placebo - Friedreich Ataxia | Phase 2 / Phase 3 | Completed | PTC Therapeutics | 146 | 2020-12-17 | 2026-04-13 |
| 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 |
| 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 | |||
| Vatiquinone Expanded Access Protocol Vatiquinone - Mitochondrial Disease | No longer available | Medical University of South Carolina | 2026-04-09 |
Friedreich Ataxia (4) · Diffuse Cerebral Sclerosis of Schilder (2) · Drug Resistant Epilepsy (2) · Leigh Disease (2) · MELAS Syndrome (2) · MERRF Syndrome (2) · Mitochondrial Diseases (2) · Mitochondrial Encephalomyopathies (1) · Mitochondrial encephalopathy (1) · Nerve Degeneration (1) · Optic Atrophy, Hereditary, Leber (1) · Pontocerebellar Hypoplasia Type 6 (1) · Seizures (1) · Status Epilepticus (1)
PTC Therapeutics (6) · Edison Pharmaceuticals Inc (1) · Medical University of South Carolina (1)
United States (7) · France (5) · Spain (5) · Canada (4) · Italy (4) · Brazil (3) · Australia (2) · Germany (2) · Japan (2) · New Zealand (2) · Poland (2) · United Kingdom (2) · Belgium (1) · Sweden (1)
Vinay Penematsa, MD (2) · Thomas Koch, MD (1)