2 completed, 1 active not recruiting, 1 recruiting, 1 terminated. 5 Phase 2, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 5 | Completed 2, Active not recruiting 1, Recruiting 1, Terminated 1 |
| 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 | Active not recruiting | Tisento Therapeutics | 44 | 2025-08-18 | 2026-10-13 |
| A Phase 2b Study of Zagociguat in Patients With MELAS zagociguat 15mg, zagociguat 30mg, Placebo - Mitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome) | Phase 2 | Completed | Tisento Therapeutics | 43 | 2024-09-10 | 2026-10-23 |
| OMT-28 in Patients With Primary Mitochondrial Disease (PMD) (PMD-OPTION) OMT-28 - Primary Mitochondrial Disease | Phase 2 | Completed | Omeicos Therapeutics GmbH | 28 | 2023-05-22 | 2026-01-22 |
| Deoxynucleosides Pyrimidines as Treatment for Mitochondrial Depletion Syndrome deoxycytidine and deoxythymidine - Mitochondrial Diseases, Mitochondrial Encephalomyopathy | Phase 2 | Recruiting | Kenneth Myers, MD | 200 | 2021-10-18 | 2026-10-19 |
| 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 (4) · Acidosis, Lactic (3) · MERRF Syndrome (2) · Mitochondrial Diseases (2) · Cardiomyopathies (1) · Deafness (1) · Diffuse Cerebral Sclerosis of Schilder (1) · Drug Resistant Epilepsy (1) · Leigh Disease (1) · Mitochondrial Encephalomyopathies (1) · Muscular Diseases (1) · Nerve Degeneration (1) · Noninsulin-dependent diabetes mellitus with deafness (1) · Pontocerebellar Hypoplasia Type 6 (1)
Brain Diseases (5) · Brain Diseases, Metabolic (5) · Central Nervous System Diseases (5) · Metabolic Diseases (5) · Mitochondrial Myopathies (5) · Musculoskeletal Diseases (5) · Nervous System Diseases (5) · Neuromuscular Diseases (5) · Nutritional and Metabolic Diseases (5) · Brain Diseases, Metabolic, Inborn (4) · Cardiovascular Diseases (4) · Cerebral Small Vessel Diseases (4) · Cerebrovascular Disorders (4) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (4) · Genetic Diseases, Inborn (4)
Deoxycytidine (1) · Thymidine (1) · alpha-tocotrienol quinone (1)
Tisento Therapeutics (2) · Kenneth Myers, MD (1) · Omeicos Therapeutics GmbH (1) · PTC Therapeutics (1)
Italy (4) · Canada (3) · Germany (3) · United Kingdom (3) · United States (3) · Australia (2) · France (1) · Japan (1) · Netherlands (1) · Poland (1) · Spain (1) · Sweden (1)
Kenneth Alexis MD Myers, MD PhD FRCPC (1) · Robert Fischer, MD (1) · Vinay Penematsa, MD (1)
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