2 terminated. 2 Phase 2, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 2 | Terminated 2 |
| Phase 3 | 1 | Terminated 1 |
Follow Mitochondrial encephalopathy: new trials by email Literature Review of Mitochondrial encephalopathy trials Research Report on Mitochondrial encephalopathy
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 |
|---|---|---|---|---|---|---|
| An Open-label Extension Safety Study of MELAS Patients Who Completed TIS6463-203 (PRIZM) zagociguat 15mg - Mitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome) | Phase 2 | Terminated | Tisento Therapeutics | 37 | 2025-08-18 | 2026-10-30 |
| 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 |
MELAS Syndrome (2) · Acidosis, Lactic (1) · Diffuse Cerebral Sclerosis of Schilder (1) · Drug Resistant Epilepsy (1) · Leigh Disease (1) · MERRF Syndrome (1) · Mitochondrial Diseases (1) · Nerve Degeneration (1) · Pontocerebellar Hypoplasia Type 6 (1) · Seizures (1) · Status Epilepticus (1)
Brain Diseases (2) · Brain Diseases, Metabolic (2) · Brain Diseases, Metabolic, Inborn (2) · Cardiovascular Diseases (2) · Central Nervous System Diseases (2) · Cerebral Small Vessel Diseases (2) · Cerebrovascular Disorders (2) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (2) · Genetic Diseases, Inborn (2) · Metabolic Diseases (2) · Metabolism, Inborn Errors (2) · Mitochondrial Encephalomyopathies (2) · Mitochondrial Myopathies (2) · Muscular Diseases (2) · Musculoskeletal Diseases (2)
PTC Therapeutics (1) · Tisento Therapeutics (1)
Italy (2) · United Kingdom (2) · United States (2) · Australia (1) · Canada (1) · France (1) · Germany (1) · Japan (1) · Poland (1) · Spain (1) · Sweden (1)