254 completed, 74 recruiting, 34 unknown, 21 active not recruiting. 111 Phase 2, 98 Not applicable, 66 Phase 1, 46 Phase 3, 15 Phase 4.
Follow Mitochondrial Diseases: new trials by email Literature Review of Mitochondrial Diseases trials Research Report on Mitochondrial Diseases
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 |
| Role of AMPKalpha2 in Myonuclear Accretion in Mitochondrial Myopathies Analysis of signaling pathway : AMPK - MItochondrial Myopathies | Not yet recruiting | Hospices Civils de Lyon | 12 | 2027-01-01 | 2026-11-05 | |
| Efficacy and Safety of Modafinil to Treat Fatigue in People With Mitochondrial Disease. Modafinil (M), Placebo - Mitochondrial Disease | Phase 3 | Not yet recruiting | Kenneth Myers, MD | 15 | 2026-12-01 | 2026-10-26 |
| GV101 for Friedreich's Ataxia (FA) Treatment GV101, Placebo - Friedreich's Ataxia | Phase 2 | Not yet recruiting | Graviton Bioscience Corporation | 48 | 2026-11-01 | 2026-10-15 |
| The Precision Mito- Mito Map COMPASS Study Mito Map Energy Score and Home Functional Assessments - Mitochondrial Diseases, Myalgic Encephalomyelitis Chronic Fatigue Syndrome | Not yet recruiting | AI Agent Vault, Inc. d/b/a Precision Mito | 400 | 2026-11-01 | 2026-10-02 | |
| A Study to Evaluate the Efficacy and Safety of Subcutaneous Nomlabofusp in Subjects With Friedreich's Ataxia Nomlabofusp, Placebo - Friedreich Ataxia, Friedreich's Ataxia | Phase 3 | Recruiting | Larimar Therapeutics, Inc. | 150 | 2026-10-26 | 2026-10-09 |
| A Study to Learn How BIIB141 (Omaveloxolone) Affects the Health of Participants With Friedrich's Ataxia Who Took it During Pregnancy and/or During Breastfeeding and About the Health of Their Babies Omaveloxolone - Friedreich Ataxia | Recruiting | Biogen | 10 | 2026-10-26 | 2026-07-10 | |
| A Study to Investigate the Safety, Tolerability, and Pharmacokinetics of ATI-105 in Healthy Adults ATI-105, Placebo - Friedreich Ataxia, Cardiomyopathies | Phase 1 | Not yet recruiting | Adjuvia Therapeutics, Inc. | 60 | 2026-10-26 | 2026-10-09 |
| A Study to Learn More About Friedrich's Ataxia in People Living in the Gulf Cooperation Council (GCC) Region of the World Friedreich's Ataxia | Not yet recruiting | Biogen | 40 | 2026-10-15 | 2026-10-21 | |
| Natural History of MADD Multiple Acyl-CoA Dehydrogenase Deficiency | Recruiting | Icahn School of Medicine at Mount Sinai | 50 | 2026-10-01 | 2026-09-16 | |
| A Dose-ranging Study of TTI-0102 in Adults and Children With Leigh Syndrome Spectrum (LSS) TTI-0102: cysteamine-pantetheine disulfide, D-Mannitol - Leigh Syndrome | Phase 2 | Not yet recruiting | Thiogenesis Therapeutics, Inc. | 18 | 2026-10-01 | 2026-07-24 |
| Mitigating Mitochondrial RNA Release During Aging to Control Inflammation and Senescence Skin biopsy and venous blood sampling - Aging, Inflammation | Not applicable | Not yet recruiting | Mario Negri Institute for Pharmacological Research | 90 | 2026-09-01 | 2026-05-19 |
| MItochondrial Intervention for Resilience in Older Adults With Coenzyme Q10 Q10Vital® (CoQ10, ubiquinone), Placebo - Frailty, Aging | Not applicable | Not yet recruiting | University Medical Centre Ljubljana | 100 | 2026-09-01 | 2026-08-19 |
| Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA) Vatiquinone - Friedreich's Ataxia | Phase 3 | Not yet recruiting | PTC Therapeutics | 120 | 2026-08-15 | 2026-08-14 |
| Validation of the Snouda Metabolic Score for Phenotyping and Guiding Reversal in Type 2 Diabetes Phenotype-Matched Dietary Protocol, Structured Progressive Exercise Protocol, Targeted Nutritional Supplementation - Type 2 Diabetes Mellitus (T2DM), Insulin Resistance | Not applicable | Enrolling by invitation | Salah Snouda | 150 | 2026-08-01 | 2026-08-18 |
| Digital Outcome Assessment Using AI Active Gaming and Motion Capture in Friedreich Ataxia Laboratory validation session (VSimulator), Home-based active gaming assessment - Friedreich Ataxia | Not yet recruiting | University of Exeter | 40 | 2026-07-30 | 2026-07-28 | |
| A Longitudinal Natural History Study of OPA1-Associated Autosomal-Dominant Optic Atrophy OPA1 Gene Mutation, Optic Atrophy, Autosomal Dominant | Recruiting | Ludwig-Maximilians - University of Munich | 50 | 2026-07-16 | 2026-07-28 | |
| Cardiac Output and Fatigue in Friedreich's Ataxia Aerobic Exercise - Friedreich's Ataxia | Not applicable | Recruiting | Scott Barbuto | 30 | 2026-07-15 | 2026-09-22 |
| Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy LX2006, Usual Care - Friedreich Ataxia, Cardiomyopathy, Secondary | Phase 2 | Recruiting | Lexeo Therapeutics | 26 | 2026-06-25 | 2026-09-02 |
| Metabolic and Functional Study of γδ T Cells in Critically Ill Patients Sepsis, Critical Illness | Recruiting | Union Hospital, Tongji Medical College, Huazhong University of Science and Technology | 105 | 2026-06-21 | 2026-10-27 |
Showing 20 of 456. Sign in to see all of them.
Friedreich Ataxia (110) · Multiple Acyl Coenzyme A Dehydrogenase Deficiency (60) · Optic Atrophy, Hereditary, Leber (44) · Mitochondrial Myopathies (42) · HIV Infections (41) · Cytomegalovirus Infections (39) · Acquired Immunodeficiency Syndrome (37) · MELAS Syndrome (37) · Cytomegalovirus Retinitis (30) · Retinitis (25) · AIDS-Related Opportunistic Infections (20) · Leigh Disease (20) · Ataxia (13) · Genetic Diseases, Inborn (13)
Nutritional and Metabolic Diseases (455) · Metabolic Diseases (452) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (306) · Nervous System Diseases (296) · Genetic Diseases, Inborn (293) · Central Nervous System Diseases (202) · Brain Diseases (196) · Heredodegenerative Disorders, Nervous System (179) · Neurodegenerative Diseases (169) · Metabolism, Inborn Errors (132) · Spinal Cord Diseases (116) · Cerebellar Diseases (110) · Pathological Conditions, Signs and Symptoms (108) · Spinocerebellar Degenerations (108)
Ganciclovir (44) · alpha-tocotrienol quinone (16) · idebenone (15) · Blood Specimen Collection (11) · Valganciclovir (9) · Foscarnet (8) · omaveloxolone (7) · arginyl-2,'6'-dimethyltyrosyl-lysyl-phenylalaninamide (6) · coenzyme Q10 (6) · Exercise (5) · 6-hydroxy-2,5,7,8-tetramethylchroman-2-carboxylic acid (4) · Arginine (4) · Magnetic Resonance Spectroscopy (4) · Resveratrol (4) · Ubiquinone (4)
National Institute of Allergy and Infectious Diseases (NIAID) (22) · Children's Hospital of Philadelphia (14) · Hoffmann-La Roche (12) · Santhera Pharmaceuticals (12) · Centre Hospitalier Universitaire de Nice (10) · PTC Therapeutics (10) · Copenhagen University Hospital, Rigshospitalet (9) · GenSight Biologics (8) · Larimar Therapeutics, Inc. (8) · Biogen (7) · Columbia University (7) · Khondrion BV (6) · Stealth BioTherapeutics Inc. (6) · University of South Florida (6) · Amgen (5)
United States (242) · France (67) · Italy (49) · Germany (46) · United Kingdom (44) · Canada (32) · Spain (30) · Netherlands (28) · Australia (25) · Belgium (22) · Denmark (22) · Austria (16) · Brazil (10) · China (10) · New Zealand (7)
Michio Hirano, MD (6) · David Lynch, MD, PhD (4) · David Lynch, MD (3) · John Vissing, MD (3) · Magdy Shenouda, M.D. (3) · Matteo Fiorenza, Ph.D. (3) · Amel Karaa, MD (2) · Arnold Munnich, M.D. (2) · David Lynch, MD PhD (2) · Hardy WD (2) · MA Jacobson (2)
PMID 36449262, PMID 34108929, PMID 40739200, PMID 17562928, PMID 21779958, PMID 21788663, PMID 26606867, PMID 26892229, PMID 33246973, PMID 34645790, PMID 38700045, PMID 39807668, PMID 42307907, PMID 11182283, PMID 11972582, PMID 12013896, PMID 12508073, PMID 12771225, PMID 12826641, PMID 1463007, PMID 14661862, PMID 15090651, PMID 15498605, PMID 15684706, PMID 16267188, PMID 16621917, PMID 1674640, PMID 16801664, PMID 16849828, PMID 16900450, PMID 16971696, PMID 16971699, PMID 17080429, PMID 17287825, PMID 17353390, PMID 17483096, PMID 17486094, PMID 17502776, PMID 17549623, PMID 17826341, PMID 18238797, PMID 18414145, PMID 18441370, PMID 18441371, PMID 18467430, PMID 18546365, PMID 18725595, PMID 18774912, PMID 19103785, PMID 19125351
2025 (5), U54NS078059 (5), 2018 (4), 2017 (3), 2015 (2), P01HD032062 (2), R01AR083552 (2), 2008 (1), 2019 (1), 2023 (1), K01HD084690 (1), K23AI070078 (1), K23MH077287 (1), N01AI30025 (1), R01AG034995 (1)