22 completed, 8 recruiting, 3 terminated, 3 unknown. 18 Phase 2, 7 Phase 1, 4 Not applicable, 3 Phase 3, 2 Early Phase 1.
Follow Mitochondrial Encephalomyopathies: new trials by email Literature Review of Mitochondrial Encephalomyopathies trials Research Report on Mitochondrial Encephalomyopathies
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 Basket Clinical Study to Assess Glycerol Tributyrate in Patients With Mitochondrial Encephalopathy, Lactic Acidosis, Stroke-like Episodes (MELAS) or Leber's Hereditary Optic Neuropathy-Plus (LHON-Plus) Glycerol Tributyrate - MELAS Syndrome, Lebers Hereditory Optic Neuropathy With Extra Ocular Symptoms (LHON-Plus) | Phase 1 / Phase 2 | Not yet recruiting | George Washington University | 24 | 2027-03-01 | 2026-10-05 |
| KHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial Diseases Sonlicromanol, Placebo - Mitochondrial Diseases, Maternally Inherited Diabetes and Deafness (MIDD) | Phase 3 | Recruiting | Khondrion BV | 220 | 2026-04-14 | 2026-10-14 |
| 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 Study to Assess TTI-0102 vs Placebo in MELAS Patients TTI-0102, Placebo - MELAS Syndrome, Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like Episodes (MELAS) | Phase 2 | Completed | Thiogenesis Therapeutics, Inc. | 9 | 2025-05-12 | 2026-07-24 |
| 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 | 2026-10-19 |
| Patterns of Neurodevelopmental Disorders Neurodevelopmental Disorders, Autism Spectrum Disorder | Recruiting | Richard Frye | 1000 | 2024-02-01 | 2026-04-16 | |
| Promoting Resilience in Stress Management (PRISM) and Clinical-focused Narrative (CFN) Pilot in Adults With Primary Mitochondrial Disease (PMD). Promoting Resilience in Stress Management (PRISM), Clinical-focused narrative (CFN) - Mitochondrial Diseases, Mitochondrial Myopathies | Phase 1 / Phase 2 | Completed | Children's Hospital of Philadelphia | 16 | 2023-09-22 | 2024-11-29 |
| Effectiveness of Ketogenic Diet in MELAS Syndrome Ketogenic diet - MELAS Syndrome, Ketogenic Dieting | Not applicable | Not yet recruiting | Second Affiliated Hospital, Zhejiang University, School of Medicine | 100 | 2023-09-01 | 2023-08-28 |
| Study of N-acetylcysteine in the Treatment of Patients With the m.3243A>G Mutation and Low Brain Glutathione Levels N-Acetylcysteine - Mitochondrial Disease | Phase 1 | Recruiting | Michio Hirano, MD | 18 | 2023-07-06 | 2026-01-23 |
| Mitochondrial Video Assessment Source Material Study PolG, Primary Mitochondrial Myopathy | Completed | The Emmes Company, LLC | 28 | 2023-06-16 | 2024-11-14 | |
| 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 |
| Natural History Study of Patients with HPDL Mutations Patient Registry, Dry blood spots sampling - Mitochondrial Encephalomyopathies, Hereditary Spastic Paraplegia | Recruiting | University of California, San Diego | 50 | 2023-05-18 | 2025-03-30 | |
| 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 |
| 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 |
| The KHENEREXT Study Oral administration of 100 mg KH176 twice daily - Mitochondrial Diseases, Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation | Phase 2 | Completed | Khondrion BV | 11 | 2021-08-09 | 2024-03-06 |
| Clinical Long Term Evaluation of Glutamine Supplement in MELAS Syndrome Glutamine oral supplement - MELAS Syndrome | Not applicable | Unknown | Hospital Universitario 12 de Octubre | 9 | 2021-07-01 | 2023-09-28 |
| Glutamine Supplement in MELAS (Mitochondrial Encephalopathy, Lactic Acidosis, and Stroke-like Episodes) Syndrome Glutamine oral supplementation - MELAS Syndrome | Not applicable | Completed | Jesús González de la Aleja Tejera | 9 | 2021-06-28 | 2022-02-24 |
| L-Citrulline Dose Finding Safety Study in MELAS L-Citrulline - MELAS Syndrome | Phase 1 | Completed | Baylor College of Medicine | 10 | 2021-04-22 | 2024-06-26 |
| The KHENERGYC Study Sonlicromanol, Placebo - Mitochondrial Diseases, Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation | Phase 2 | Suspended | Khondrion BV | 24 | 2021-02-01 | 2025-01-10 |
| Phase 2a Study of IW-6463 in Adults Diagnosed With Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like Episodes (MELAS) IW-6463 Tablets - MELAS | Phase 2 | Terminated | Tisento Therapeutics | 8 | 2020-11-13 | 2024-08-29 |
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MELAS Syndrome (37) · Mitochondrial Diseases (23) · Leigh Disease (10) · Acidosis, Lactic (8) · Mitochondrial Myopathies (8) · Ophthalmoplegia, Chronic Progressive External (8) · MERRF Syndrome (6) · Mitochondrial encephalopathy (5) · Noninsulin-dependent diabetes mellitus with deafness (4) · Deafness (3) · Kearns-Sayre Syndrome (3) · Optic Atrophy, Hereditary, Leber (3) · Seizures (3) · Barth Syndrome (2)
Brain Diseases, Metabolic (42) · Central Nervous System Diseases (42) · Metabolic Diseases (42) · Musculoskeletal Diseases (42) · Nervous System Diseases (42) · Neuromuscular Diseases (42) · Nutritional and Metabolic Diseases (42) · Brain Diseases (41) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (41) · Genetic Diseases, Inborn (40) · Muscular Diseases (40) · Brain Diseases, Metabolic, Inborn (39) · Metabolism, Inborn Errors (38) · Cardiovascular Diseases (37) · Cerebral Small Vessel Diseases (37)
6-hydroxy-2,5,7,8-tetramethylchroman-2-carboxylic acid (4) · alpha-tocotrienol quinone (3) · Arginine (2) · Cysteamine (2) · Acetylcysteine (1) · Bulk Drugs (1) · Citrulline (1) · Deoxycytidine (1) · Dichloroacetic Acid (1) · Diet, Ketogenic (1) · Influenza Vaccines (1) · Thymidine (1) · cysteamine-pantetheine disulfide (1) · idebenone (1) · nicotinamide-beta-riboside (1)
Khondrion BV (6) · Columbia University (3) · PTC Therapeutics (3) · Tisento Therapeutics (3) · Amgen (2) · Baylor College of Medicine (2) · Abliva AB (1) · Cambridge University Hospitals NHS Foundation Trust (1) · Children's Hospital of Philadelphia (1) · Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (1) · George Washington University (1) · Hospital Universitario 12 de Octubre (1) · Jesús González de la Aleja Tejera (1) · Kenneth Myers, MD (1) · LMU Klinikum (1)
United States (25) · United Kingdom (9) · Germany (7) · Italy (7) · Netherlands (7) · Canada (5) · France (4) · Spain (4) · Denmark (3) · Australia (2) · Japan (2) · Poland (2) · Austria (1) · Belgium (1) · China (1)
Michio Hirano, MD (5) · Vinay Penematsa, MD (2) · Anne Chiaramello, Ph.D. (1) · Chad Glasser, PharmD (1) · Cornelia L Dekker, MD (1) · Darryl C De Vivo, MD (1) · Debra Regier, M.D., Ph.D. (1) · Elizabeth M McCormick, MS (1) · Fernando Scaglia, M.D. (1) · Gregory Enns, MB, ChB (1) · Gregory M Enns, MBChB (1) · Ingrid Tein, MD (1)
PMID 11182283, PMID 12013896, PMID 12508073, PMID 12771225, PMID 12826641, PMID 15090651, PMID 15684706, PMID 16621917, PMID 16971696, PMID 16971699, PMID 17080429, PMID 17353390, PMID 17483096, PMID 17486094, PMID 17549623, PMID 18238797, PMID 18467430, PMID 18546365, PMID 19103785, PMID 19125351, PMID 19138848, PMID 19192035, PMID 19667227, PMID 19766516, PMID 19889013, PMID 20301762, PMID 20301791, PMID 20329599, PMID 20444604, PMID 21483760, PMID 21774628, PMID 21855607, PMID 22084276, PMID 22176657, PMID 22608879, PMID 22711161, PMID 23168274, PMID 23169334, PMID 23230576, PMID 23385875, PMID 23714749, PMID 23817075, PMID 245993, PMID 24708634, PMID 24985751, PMID 25857492, PMID 25993630, PMID 26342080, PMID 26760297, PMID 27554452
U54NS078059 (3), P01HD032062 (2), R21HD061709 (1), UH3TR003897 (1)