5 completed, 4 terminated, 2 active not recruiting, 2 recruiting. 15 Phase 2, 6 Phase 1, 3 Phase 3.
Follow Optic Atrophy, Hereditary, Leber: new trials by email Literature Review of Optic Atrophy, Hereditary, Leber trials Research Report on Optic Atrophy, Hereditary, Leber
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 Study of Bilateral IVT Injection of GS010 at Two Dose Levels in LHON Patients GS010 High dose, GS010 Low dose - Leber Hereditary Optic Disease | Phase 2 | Recruiting | GenSight Biologics | 14 | 2026-01-22 | 2026-03-02 |
| Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND1 Mutations NFS-02 Injection - Leber Hereditary Optic Neuropathy (LHON) | Phase 1 / Phase 2 | Terminated | Neurophth Therapeutics Inc | 11 | 2023-08-15 | 2024-09-20 |
| Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND4 Mutations NR082 Injection, Injection needle - Leber Hereditary Optic Neuropathy (LHON) | Phase 1 / Phase 2 | Active not recruiting | Neurophth Therapeutics Inc | 12 | 2023-05-22 | 2024-09-04 |
| Gene Therapy Clinical Trial for the Treatment Of Leber's HereDitary Optic Neuropathy NR082 injection, Sham Injection - Leber's Hereditary Optic Neuropathy (LHON) | Phase 2 / Phase 3 | Recruiting | Wuhan Neurophth Biotechnology Limited Company | 102 | 2021-06-18 | 2022-11-14 |
| Extension Study to Study PQ-110-001 (NCT03140969) QR-110 - Leber Congenital Amaurosis 10, Blindness | Phase 1 / Phase 2 | Terminated | Laboratoires Thea | 9 | 2019-05-13 | 2024-10-15 |
| A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE) sepofarsen, Sham - Leber Congenital Amaurosis 10, Blindness | Phase 2 / Phase 3 | Active not recruiting | ProQR Therapeutics | 36 | 2019-04-04 | 2022-03-17 |
| A Single Intravitreal Injection of rAAV2-ND4 for the Treatment of Leber's Hereditary Optic Neuropathy rAAV2-ND4 - Leber Hereditary Optic Neuropathy | Phase 2 / Phase 3 | Unknown | Huazhong University of Science and Technology | 159 | 2017-12-27 | 2020-09-17 |
| Study to Evaluate QR-110 in Leber's Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene QR-110 - Leber's Congenital Amaurosis | Phase 1 / Phase 2 | Completed | Laboratoires Thea | 11 | 2017-10-16 | 2024-10-15 |
| A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for Treatment of Leber's Hereditary Optic Neuropathy elamipretide (MTP-131) 1% topical ophthalmic solution, Vehicle topical ophthalmic solution - Leber's Hereditary Optic Neuropathy | Phase 2 | Completed | Stealth BioTherapeutics Inc. | 12 | 2016-03-01 | 2021-11-30 |
| 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 |
| 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 |
| Safety Evaluation of Gene Therapy in Leber Hereditary Optic Neuropathy (LHON) Patients GS010 - Leber Hereditary Optic Neuropathy | Phase 1 / Phase 2 | Completed | GenSight Biologics | 19 | 2014-02-13 | 2026-03-02 |
| Near-infrared Light-emitting Diode (NIR-LED) Therapy for Leber's Hereditary Optic Neuropathy (LHON) Near-infrared light-emitting diode (NIR-LED) therapy (Med Light 630 PRO (Medical Devices Inc.)) - Leber's Hereditary Optic Neuropathy (LHON) | Phase 1 / Phase 2 | Terminated | Medical College of Wisconsin | 4 | 2011-07-01 | 2014-09-29 |
| Trial of Cyclosporine in the Acute Phase of Leber Hereditary Optic Neuropathy cyclosporine - Leber Hereditary Optic Neuropathy | Phase 2 | Unknown | University Hospital, Angers | 12 | 2011-07-01 | 2014-06-27 |
| Study to Assess Efficacy,Safety and Tolerability of Idebenone in the Treatment of Leber's Hereditary Optic Neuropathy Idebenone, Placebo - Leber's Hereditary Optic Neuropathy | Phase 2 | Completed | Santhera Pharmaceuticals | 85 | 2007-11-01 | 2013-05-27 |
Eye Diseases (3) · Meckel Syndrome, Type 4 (3) · Blindness (2) · Epilepsies, Myoclonic (2) · Eye Abnormalities (2) · Eye Diseases, Hereditary (2) · Leber Congenital Amaurosis (2) · Leber Congenital Amaurosis 10 (2) · Leigh Disease (2) · Mitochondrial Diseases (2) · Mitochondrial Encephalomyopathies (2) · Neurologic Manifestations (2) · Ophthalmoplegia, Chronic Progressive External (2) · Retinal Diseases (2)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (15) · Cranial Nerve Diseases (15) · Heredodegenerative Disorders, Nervous System (15) · Metabolic Diseases (15) · Nutritional and Metabolic Diseases (15) · Optic Atrophies, Hereditary (15) · Optic Atrophy (15) · Optic Nerve Diseases (15) · Genetic Diseases, Inborn (14) · Nervous System Diseases (14) · Neurodegenerative Diseases (14) · Eye Diseases, Hereditary (13) · Mitochondrial Diseases (13) · Eye Diseases (12) · Pathological Conditions, Signs and Symptoms (4)
Cysteamine (2) · Cyclosporine (1) · Needles (1) · Ophthalmic Solutions (1) · Syringes (1) · Therapeutics (1) · arginyl-2,'6'-dimethyltyrosyl-lysyl-phenylalaninamide (1) · idebenone (1) · salicylhydroxamic acid (1)
Amgen (2) · GenSight Biologics (2) · Laboratoires Thea (2) · Neurophth Therapeutics Inc (2) · Huazhong University of Science and Technology (1) · Medical College of Wisconsin (1) · ProQR Therapeutics (1) · Santhera Pharmaceuticals (1) · Stealth BioTherapeutics Inc. (1) · University Hospital, Angers (1) · Wuhan Neurophth Biotechnology Limited Company (1)
United States (9) · France (4) · Belgium (3) · China (3) · Canada (2) · Germany (2) · United Kingdom (2) · Brazil (1) · Italy (1) · Netherlands (1)
Alfredo Sadun, MD, PhD (1) · Bin Li, MD (1) · Bin Li, PhD,MD (1) · Harry T Whelan, MD (1) · Prof Patrick F Chinnery, MD (1) · Prof Thomas Klopstock, MD (1) · Yong Zhang, PhD,MD (1)
PMID 1463007, PMID 1674640, PMID 18441370, PMID 18441371, PMID 18774912, PMID 1928099, PMID 21788663, PMID 22612072, PMID 22644094, PMID 23063736, PMID 23263355, PMID 24396955, PMID 25950380, PMID 26606867, PMID 26892229, PMID 27386292, PMID 27426279, PMID 27749593, PMID 29454364, PMID 30559420, PMID 31215818, PMID 31237206, PMID 3201231, PMID 33795869, PMID 35379979, PMID 5063796
2018 (2), 2017 (1)