21 completed, 5 recruiting, 3 unknown, 2 not yet recruiting. 15 Phase 2, 7 Phase 1, 4 Not applicable, 3 Phase 3, 2 Early Phase 1.
Follow MELAS Syndrome: new trials by email Literature Review of MELAS Syndrome trials Research Report on MELAS Syndrome
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 | 2026-10-23 |
| 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 |
| 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 |
| 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 |
| 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 |
| The KHENERGYZE Study KH176, Placebo - Mitochondrial Diseases, Mitochondrial Myopathies | Phase 2 | Completed | Khondrion BV | 27 | 2019-10-30 | 2022-08-31 |
| A Phase Ia/Ib, SAD and MAD Study of of KL1333 in Healthy Subjects and Patients With Primary Mitochondrial Disease KL1333, Placebo Oral Tablet - Mitochondrial Diseases, Mitochondrial Respiratory Chain Deficiencies | Phase 1 | Completed | Abliva AB | 72 | 2019-03-18 | 2021-10-20 |
Showing 20 of 37. Sign in to see all of them.
Mitochondrial Diseases (21) · Leigh Disease (8) · Mitochondrial Myopathies (8) · Acidosis, Lactic (7) · MERRF Syndrome (6) · Ophthalmoplegia, Chronic Progressive External (6) · Mitochondrial Encephalomyopathies (4) · Mitochondrial encephalopathy (4) · Noninsulin-dependent diabetes mellitus with deafness (4) · Deafness (3) · Kearns-Sayre Syndrome (3) · Seizures (3) · Barth Syndrome (2) · Diabetes Mellitus (2)
Brain Diseases, Metabolic (37) · Brain Diseases, Metabolic, Inborn (37) · Cardiovascular Diseases (37) · Central Nervous System Diseases (37) · Cerebral Small Vessel Diseases (37) · Cerebrovascular Disorders (37) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (37) · Genetic Diseases, Inborn (37) · Metabolic Diseases (37) · Musculoskeletal Diseases (37) · Nervous System Diseases (37) · Neuromuscular Diseases (37) · Nutritional and Metabolic Diseases (37) · Vascular Diseases (37) · Brain Diseases (36)
6-hydroxy-2,5,7,8-tetramethylchroman-2-carboxylic acid (4) · alpha-tocotrienol quinone (3) · Arginine (2) · Acetylcysteine (1) · Bulk Drugs (1) · Citrulline (1) · Dichloroacetic Acid (1) · Diet, Ketogenic (1) · Influenza Vaccines (1) · cysteamine-pantetheine disulfide (1) · idebenone (1) · nicotinamide-beta-riboside (1)
Khondrion BV (6) · Columbia University (3) · PTC Therapeutics (3) · Tisento Therapeutics (3) · 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) · LMU Klinikum (1) · Michio Hirano (1) · Michio Hirano, MD (1)
United States (21) · United Kingdom (9) · Germany (7) · Italy (7) · Netherlands (7) · Canada (4) · 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 25993630, PMID 29037240, PMID 30058726, PMID 32881886, PMID 35477351, PMID 36334048, PMID 38114794
U54NS078059 (3), P01HD032062 (2), R21HD061709 (1), UH3TR003897 (1)