3 completed, 2 recruiting, 1 not yet recruiting. 6 Phase 1, 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 6 | Completed 3, Recruiting 2, Not yet recruiting 1 |
| Phase 2 | 1 | Completed 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 |
|---|---|---|---|---|---|---|
| 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 |
| A Dose-escalating Clinical Trial With KH176 KH176, placebo - MELAS, LHON | Phase 1 | Completed | Khondrion BV | 32 | 2015-05-01 | 2021-10-18 |
| 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 |
Mitochondrial Diseases (6) · MELAS Syndrome (2) · Deafness (1) · Diabetes Mellitus (1) · Leigh Disease (1) · Mitochondrial Myopathies (1) · Muscular Diseases (1) · Optic Atrophy, Hereditary, Leber (1) · Optic Nerve Diseases (1)
Metabolic Diseases (6) · Nutritional and Metabolic Diseases (6) · Nervous System Diseases (5) · Musculoskeletal Diseases (4) · Neuromuscular Diseases (4) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (3) · Genetic Diseases, Inborn (3) · Muscular Diseases (3) · Brain Diseases (2) · Brain Diseases, Metabolic (2) · Brain Diseases, Metabolic, Inborn (2) · Cardiovascular Diseases (2) · Central Nervous System Diseases (2) · Cerebral Small Vessel Diseases (2) · Cerebrovascular Disorders (2)
6-hydroxy-2,5,7,8-tetramethylchroman-2-carboxylic acid (1) · Acetylcysteine (1) · Bupropion (1) · Caffeine (1) · Dextromethorphan (1) · Flurbiprofen (1) · Midazolam (1) · Omeprazole (1) · alpha-tocotrienol quinone (1) · repaglinide (1)
Abliva AB (1) · Children's Hospital of Philadelphia (1) · Khondrion BV (1) · Michio Hirano, MD (1) · National Human Genome Research Institute (NHGRI) (1) · University Hospital, Angers (1)
United States (3) · Belgium (1) · France (1) · United Kingdom (1)
Michio Hirano, MD (1) · William A Gahl, M.D. (1) · Zarazuela Zolkipli-Cunningham, MBChB, MRCP (1)
2025 (1)