13 completed, 3 not yet recruiting, 3 recruiting, 1 active not recruiting. 6 Phase 1, 3 Not applicable, 1 Phase 2, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 6 | Completed 3, Recruiting 2, Not yet recruiting 1 |
| Not applicable | 3 | Completed 1, Not yet recruiting 1, Terminated 1 |
| Phase 2 | 1 | Completed 1 |
| Phase 3 | 1 | Not yet recruiting 1 |
Follow Mitochondrial Disease: new trials by email Literature Review of Mitochondrial Disease trials Research Report on Mitochondrial Disease
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 |
|---|---|---|---|---|---|---|
| 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 |
| Development of a New Technique for Quantifying Mitochondrial DNA in Single Muscle Fibers Quantification of Mitochondrial DNA Heteroplasmy and Copy Number by Digital PCR in Mitochondrial Diseases - Mitochondrial Disease | Not applicable | Not yet recruiting | Centre Hospitalier Universitaire de Nice | 4 | 2026-06-01 | 2026-04-06 |
| Pilot Study of the Efficacy of Nicotinamide (Vitamin B3) in Leber's Hereditary Optic Neuropathy Nicotinamide treatment - Leber Hereditary Optic Neuropathy (LHON), Leber's Hereditary Optic Neuropathy (LHON) | Phase 1 | Not yet recruiting | University Hospital, Angers | 13 | 2026-04-01 | 2025-12-18 |
| Validation of Nanosensor Oxygen Measurement Nanosensor - MItochondrial Myopathies, Mitochondrial Disease | Phase 1 | Recruiting | Children's Hospital of Philadelphia | 70 | 2025-01-02 | 2026-10-07 |
| 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 |
| Drug-drug Interaction Study of KL1333 in Healthy Subjects KL1333, Flurbiprofen, Dextromethorphan - Mitochondrial Disease | Phase 1 | Completed | Abliva AB | 14 | 2020-11-10 | 2021-10-20 |
| Acute Infection in Mitochondrial Disease: Metabolism, Infection and Immunity Mitochondrial Disease | Recruiting | National Human Genome Research Institute (NHGRI) | 400 | 2020-10-21 | 2026-09-10 | |
| Assessment of Small Fiber Neuropathy in Rare Diseases Using Sudoscan Sudoscan, Skin biopsy, QSART - Small Fiber Neuropathy, Fabry Disease | Not applicable | Completed | Massachusetts General Hospital | 102 | 2016-08-01 | 2021-10-07 |
| GDF-15 as a Biomarker for Mitochondrial Disease Mitochondrial Disease, Metabolic Myopathy | Completed | Copenhagen University Hospital, Rigshospitalet | 97 | 2016-06-01 | 2017-08-17 | |
| Calf Muscle Strength in Mitochondrial Diseases MRI and muscle dynamometer - Mitochondrial Disease | Completed | Copenhagen University Hospital, Rigshospitalet | 37 | 2016-04-01 | 2017-08-17 | |
| A Dose-escalating Clinical Trial With KH176 KH176, placebo - MELAS, LHON | Phase 1 | Completed | Khondrion BV | 32 | 2015-05-01 | 2021-10-18 |
| Survey on Supplement Use in Mitochondrial Disease Mitochondrial Disease | Completed | University of South Florida | 162 | 2014-09-01 | 2016-09-22 | |
| Magnetic Resonance Imaging (MRI) Muscle Phenotyping in Mitochondrial Disease Mitochondrial Disease | Completed | University of Pennsylvania | 40 | 2014-05-01 | 2024-08-29 | |
| Mitochondrial Dysfunction in Autism Spectrum Disorder Autism Spectrum Disorder, Autism | Active not recruiting | University of Arkansas | 297 | 2012-10-16 | 2026-01-22 | |
| EPI-743 for Metabolism or Mitochondrial Disorders EPI-743, Placebo - Mitochondrial Disease, Neurology | Phase 1 / Phase 2 | Completed | National Human Genome Research Institute (NHGRI) | 20 | 2012-09-01 | 2021-04-14 |
| Tissue Sample Study for Mitochondrial Disorders Mitochondrial Disorders, Mitochondrial Disease | Enrolling by invitation | Columbia University | 6900 | 2012-02-01 | 2026-01-07 | |
| Energy Requirements in Mitochondrial Disease Mitochondrial Disease, Chronic Metabolic Disorder | Completed | University of British Columbia | 23 | 2012-02-01 | 2017-07-18 | |
| Defining 31Phosphorous Magnetic Resonance Spectroscopy Characteristics in Patients With Mitochondrial Myopathy Mitochondrial Disease | Completed | Massachusetts General Hospital | 8 | 2011-02-01 | 2012-04-30 | |
| Mechanisms of Mitochondrial Defects in Gulf War Syndrome Skin biopsy, Blood Collection - Gulf War Syndrome, Mitochondrial Disease | Completed | Medical Neurogenetics, LLC | 26 | 2009-05-01 | 2015-04-13 | |
| Economic and Medical Evaluation of the Whole Mitochondrial DNA Screening by Surveyor and Mitochips Techniques Mitochondrial Disease | Completed | Centre Hospitalier Universitaire de Nice | 550 | 2009-03-01 | 2012-12-18 |
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Mitochondrial Diseases (24) · MELAS Syndrome (3) · Leigh Disease (2) · Muscular Diseases (2) · Asperger Syndrome (1) · Autism Spectrum Disorder (1) · Autistic Disorder (1) · Deafness (1) · Diabetes Mellitus (1) · Ehlers-Danlos Syndrome (1) · Fabry Disease (1) · Fatigue (1) · Genetic Diseases, Inborn (1) · Learning Disabilities (1) · Mitochondrial Myopathies (1)
Metabolic Diseases (24) · Nutritional and Metabolic Diseases (24) · Nervous System Diseases (11) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (8) · Neuromuscular Diseases (8) · Genetic Diseases, Inborn (7) · Musculoskeletal Diseases (7) · Brain Diseases (5) · Brain Diseases, Metabolic (5) · Brain Diseases, Metabolic, Inborn (5) · Central Nervous System Diseases (5) · Metabolism, Inborn Errors (5) · Muscular Diseases (5) · Cardiovascular Diseases (4) · Cerebral Small Vessel Diseases (4)
alpha-tocotrienol quinone (2) · 6-hydroxy-2,5,7,8-tetramethylchroman-2-carboxylic acid (1) · Acetylcysteine (1) · Blood Specimen Collection (1) · Bupropion (1) · Caffeine (1) · Dextromethorphan (1) · Flurbiprofen (1) · Magnetic Resonance Spectroscopy (1) · Midazolam (1) · Modafinil (1) · Omeprazole (1) · Phlebotomy (1) · Probucol (1) · Sevoflurane (1)
Centre Hospitalier Universitaire de Nice (2) · Copenhagen University Hospital, Rigshospitalet (2) · Massachusetts General Hospital (2) · National Human Genome Research Institute (NHGRI) (2) · Abliva AB (1) · Children's Hospital of Philadelphia (1) · Columbia University (1) · Kenneth Myers, MD (1) · Khondrion BV (1) · Medical Neurogenetics, LLC (1) · Medical University of South Carolina (1) · Michio Hirano, MD (1) · RiboNova Inc. (1) · University Hospital, Angers (1) · University of Arkansas (1)
United States (14) · France (3) · Canada (2) · Denmark (2) · Belgium (1) · United Kingdom (1)
Michio Hirano, MD (3) · Amel Karaa, MD (2) · Bruce J Tromberg, PhD (1) · Danield I Sessler, MD (1) · Eliza M Gordon-Lipkin, M.D. (1) · Howard Parsons (1) · John M Shoffner, MD (1) · Nanna S Nielsen, B.Sc (1) · Nanna S. Nielsen, B.Sc. (1) · Rajavel Elango, PhD (1) · Ramona Meni Salvarinova Zivkovic (1) · Shana E McCormack, MD (1) · Shannon Rose, PhD (1) · Sumit Parikh, MD (1) · Sylvia Stockler-Ipsiroglu (1)
2025 (1), P01HD032062 (1)