4 completed. 4 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 4 | Completed 4 |
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 |
|---|---|---|---|---|---|---|
| Use of Phenylbutyrate Therapy for Patients With Pyruvate Dehydrogenase Complex Deficiency. sodium phenylbutyrate - Pyruvate Dehydrogenase Complex Deficiency | Phase 2 | Completed | Fondazione Telethon | 1 | 2018-10-01 | 2021-10-13 |
| Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease Cysteamine Bitartrate - Inherited Mitochondrial Disease, Including Leigh Syndrome | Phase 2 | Completed | Amgen | 36 | 2014-05-01 | 2024-12-27 |
| 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 |
| Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome Placebo, EPI-743 15 mg/kg, EPI-743 5 mg/kg - Leigh Syndrome | Phase 2 | Completed | PTC Therapeutics | 35 | 2012-10-31 | 2020-08-31 |
Leigh Disease (3) · Mitochondrial Diseases (2) · Epilepsies, Myoclonic (1) · Mitochondrial Encephalomyopathies (1) · Ophthalmoplegia, Chronic Progressive External (1) · Optic Atrophy, Hereditary, Leber (1) · Pyruvate Dehydrogenase Complex Deficiency Disease (1)
Brain Diseases (4) · Brain Diseases, Metabolic (4) · Brain Diseases, Metabolic, Inborn (4) · Carbohydrate Metabolism, Inborn Errors (4) · Central Nervous System Diseases (4) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (4) · Genetic Diseases, Inborn (4) · Metabolic Diseases (4) · Metabolism, Inborn Errors (4) · Nervous System Diseases (4) · Nutritional and Metabolic Diseases (4) · Heredodegenerative Disorders, Nervous System (2) · Mitochondrial Diseases (2) · Neurologic Manifestations (2)
alpha-tocotrienol quinone (2) · 4-phenylbutyric acid (1) · Cysteamine (1)
PTC Therapeutics (2) · Amgen (1) · Fondazione Telethon (1)
United States (3) · Italy (1)
Bruce Cohen, MD (1)