9 completed, 4 recruiting, 1 terminated. 3 Phase 2, 2 Not applicable, 1 Phase 1, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 3 | Completed 2, Terminated 1 |
| Not applicable | 2 | Completed 2 |
| Phase 1 | 1 | Completed 1 |
| Phase 3 | 1 | Completed 1 |
Follow Ophthalmoplegia, Chronic Progressive External: new trials by email Literature Review of Ophthalmoplegia, Chronic Progressive External trials Research Report on Ophthalmoplegia, Chronic Progressive External
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 |
|---|---|---|---|---|---|---|
| Prospective Natural History Study of POLG Disease PolG, Primary Mitochondrial Myopathy | Recruiting | The POLG Foundation | 300 | 2026-06-17 | 2026-09-15 | |
| Mitochondrial Video Assessment Source Material Study PolG, Primary Mitochondrial Myopathy | Completed | The Emmes Company, LLC | 28 | 2023-06-16 | 2024-11-14 | |
| Genomic Profiling of Mitochondrial Disease - Imaging Analysis for Precise Mitochondrial Medicine Mitochondrial Myopathies, Mitochondrial DNA Mutation | Completed | Mayo Clinic | 9 | 2022-05-01 | 2024-06-12 | |
| Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD) Elamipretide, Placebo - Mitochondrial Myopathies, Mitochondrial Pathology | Phase 3 | Completed | Stealth BioTherapeutics Inc. | 102 | 2022-04-29 | 2025-10-24 |
| 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 Trial Comparing Two Non-Surgical Treatments for Severe Blepharoptosis Magnetic Levator Prosthesis (MLP), Kinesiotape Frontalis Sling (KTFS) - Blepharoptosis, Ptosis, Eyelid | Not applicable | Completed | Massachusetts Eye and Ear Infirmary | 16 | 2021-06-01 | 2025-04-22 |
| 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 |
| Nicotinamide Riboside and Mitochondrial Biogenesis Nicotinamide Riboside - Mitochondrial Diseases, Mitochondrial Myopathies | Not applicable | Completed | Cambridge University Hospitals NHS Foundation Trust | 13 | 2017-12-08 | 2023-07-20 |
| A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease Cysteamine Bitartrate - Mitochondrial Diseases | Phase 2 | Terminated | Amgen | 22 | 2015-05-19 | 2024-12-27 |
| Inherited Retinal Degenerative Disease Registry Eye Diseases Hereditary, Retinal Disease | Recruiting | Foundation Fighting Blindness | 20000 | 2014-06-01 | 2026-05-19 | |
| MRI Study - Chronic Progressive External Ophthalmoplegia Magnetic Resonance Imaging - Chronic Progressive External Ophthalmoplegia | Completed | Copenhagen University Hospital, Rigshospitalet | 133 | 2014-06-01 | 2016-04-14 | |
| Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease Cysteamine Bitartrate - Inherited Mitochondrial Disease, Including Leigh Syndrome | Phase 2 | Completed | Amgen | 36 | 2014-05-01 | 2024-12-27 |
| North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC) Mitochondrial Disorders, Mitochondrial Genetic Disorders | Recruiting | Columbia University | 1000 | 2011-01-31 | 2026-02-04 | |
| Global Registry and Natural History Study for Mitochondrial Disorders Mitochondrial Diseases, Kearns-Sayre Syndrome | Recruiting | LMU Klinikum | 6000 | 2009-02-01 | 2026-08-07 |
Mitochondrial Diseases (8) · Mitochondrial Myopathies (7) · MELAS Syndrome (6) · Kearns-Sayre Syndrome (5) · Leigh Disease (4) · Optic Atrophy, Hereditary, Leber (3) · Acidosis, Lactic (2) · Barth Syndrome (2) · Epilepsies, Myoclonic (2) · MERRF Syndrome (2) · Mitochondrial Encephalomyopathies (2) · Mitochondrial complex I deficiency (2) · Neuropathy ataxia and retinitis pigmentosa (2) · Stroke (2)
Chronic Disease (14) · Cranial Nerve Diseases (14) · Disease Attributes (14) · Eye Diseases (14) · Metabolic Diseases (14) · Muscular Diseases (14) · Musculoskeletal Diseases (14) · Nervous System Diseases (14) · Neurologic Manifestations (14) · Neuromuscular Diseases (14) · Nutritional and Metabolic Diseases (14) · Ocular Motility Disorders (14) · Ophthalmoplegia (14) · Paralysis (14) · Pathologic Processes (14)
Cysteamine (2) · Magnetic Resonance Spectroscopy (1) · arginyl-2,'6'-dimethyltyrosyl-lysyl-phenylalaninamide (1) · nicotinamide-beta-riboside (1)
Amgen (2) · Abliva AB (1) · Cambridge University Hospitals NHS Foundation Trust (1) · Columbia University (1) · Copenhagen University Hospital, Rigshospitalet (1) · Foundation Fighting Blindness (1) · Khondrion BV (1) · LMU Klinikum (1) · Massachusetts Eye and Ear Infirmary (1) · Mayo Clinic (1) · Stealth BioTherapeutics Inc. (1) · The Emmes Company, LLC (1) · The POLG Foundation (1)
United States (9) · United Kingdom (5) · Denmark (3) · Germany (3) · Netherlands (3) · Australia (2) · Italy (2) · Norway (2) · Spain (2) · Austria (1) · Canada (1) · Finland (1) · Hungary (1) · New Zealand (1) · Sweden (1)
Amel Kaara (1) · Kevin Houston, OD MSc (1) · Matilda Hugerth, MSc (1) · Michelangelo Mancuso, Prof. Dr. (1) · Michio Hirano, MD (1) · Omar Hikmat (1) · Patrick Chinnery, Prof. (1) · Ralitza Gavrilova, MD (1) · Thomas Klopstock, Prof. Dr. (1) · Todd Durham, PhD (1)
R01EY029437 (1), U54NS078059 (1)