2 terminated. 2 Phase 2, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 2 | Terminated 2 |
| Phase 3 | 1 | Terminated 1 |
Follow Pyruvate Metabolism, Inborn Errors: new trials by email Literature Review of Pyruvate Metabolism, Inborn Errors trials Research Report on Pyruvate Metabolism, Inborn Errors
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 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 |
| A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease Cysteamine Bitartrate - Mitochondrial Diseases | Phase 2 | Terminated | Amgen | 22 | 2015-05-19 | 2024-12-27 |
Leigh Disease (2) · Mitochondrial Diseases (2) · Acidosis, Lactic (1) · Diffuse Cerebral Sclerosis of Schilder (1) · Drug Resistant Epilepsy (1) · Epilepsies, Myoclonic (1) · MELAS Syndrome (1) · MERRF Syndrome (1) · Mitochondrial Encephalomyopathies (1) · Mitochondrial encephalopathy (1) · Nerve Degeneration (1) · Ophthalmoplegia, Chronic Progressive External (1) · Optic Atrophy, Hereditary, Leber (1) · Pontocerebellar Hypoplasia Type 6 (1) · Seizures (1)
Brain Diseases (2) · Brain Diseases, Metabolic (2) · Brain Diseases, Metabolic, Inborn (2) · Carbohydrate Metabolism, Inborn Errors (2) · Central Nervous System Diseases (2) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (2) · Epilepsy (2) · Epilepsy, Generalized (2) · Epileptic Syndromes (2) · Genetic Diseases, Inborn (2) · Metabolic Diseases (2) · Metabolism, Inborn Errors (2) · Mitochondrial Myopathies (2) · Muscular Diseases (2) · Musculoskeletal Diseases (2)
Cysteamine (1) · alpha-tocotrienol quinone (1)
Amgen (1) · PTC Therapeutics (1)
United States (2) · Canada (1) · France (1) · Italy (1) · Japan (1) · Poland (1) · Spain (1) · Sweden (1) · United Kingdom (1)