5 completed, 2 active not recruiting, 2 recruiting, 1 unknown. 11 Phase 3, 3 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 11 | Completed 5, Active not recruiting 2, Recruiting 2, Unknown 1, Withdrawn 1 |
| Phase 2 | 3 | Active not recruiting 1, Recruiting 1, Unknown 1 |
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 |
|---|---|---|---|---|---|---|
| Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION) sepofarsen, Placebo IVT - Leber Congenital Amaurosis 10, Blindness | Phase 3 | Recruiting | Laboratoires Thea | 32 | 2025-06-04 | 2026-10-21 |
| A Phase 3, Multicenter, Randomized, Double-Masked, Sham-Controlled Clinical Trial for Leber's Hereditary Optic Neuropathy (LHON) Associated With ND4 Mutation rAAV2-ND4, Sham (No Treatment) - Leber Hereditary Optic Neuropathy (LHON) | Phase 3 | Active not recruiting | Wuhan Neurophth Biotechnology Limited Company | 95 | 2024-09-19 | 2026-02-12 |
| 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 |
| 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 |
| Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year GS010, Placebo - Leber Hereditary Optic Neuropathy | Phase 3 | Completed | GenSight Biologics | 98 | 2018-03-12 | 2026-04-16 |
| RESCUE and REVERSE Long-term Follow-up GS010, Sham Intravitreal Injection - Leber Hereditary Optic Neuropathy | Phase 3 | Completed | GenSight Biologics | 62 | 2018-01-09 | 2026-03-02 |
| 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 |
| Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 Mutation GS010, Sham Intravitreal Injection - Optic, Atrophy, Hereditary, Leber | Phase 3 | Completed | GenSight Biologics | 39 | 2016-02-23 | 2026-03-02 |
| Efficacy Study of GS010 for Treatment of Vision Loss From 7 Months to 1 Year From Onset in LHON Due to the ND4 Mutation (REVERSE) GS010, Sham Intravitreal Injection - Optic, Atrophy, Hereditary, Leber | Phase 3 | Completed | GenSight Biologics | 37 | 2016-01-01 | 2026-03-02 |
| A Randomized, Double-blind, Placebo-controlled Trial of Curcumin in Leber's Hereditary Optic Neuropathy (LHON) curcumin - Optic Atrophy, Hereditary, Leber | Phase 3 | Completed | Mahidol University | 70 | 2005-05-01 | 2012-12-24 |
| Study With Idebenone in Patients With Chronic Vision Loss Due to Leber's Hereditary Optic Neuropathy (LHON) Idebenone, Placebo - Leber's Hereditary Optic Neuropathy | Phase 3 | Withdrawn | Santhera Pharmaceuticals | 2014-08-22 |
Eye Diseases (6) · Eye Diseases, Hereditary (6) · Mitochondrial Diseases (4) · Genetic Diseases, Inborn (3) · Nervous System Diseases (3) · Neurodegenerative Diseases (3) · Blindness (2) · Eye Abnormalities (2) · Leber Congenital Amaurosis (2) · Leber Congenital Amaurosis 10 (2) · Meckel Syndrome, Type 4 (2) · Neurologic Manifestations (2) · Retinal Diseases (2) · Sensation Disorders (2)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (11) · Cranial Nerve Diseases (11) · Heredodegenerative Disorders, Nervous System (11) · Metabolic Diseases (11) · Nutritional and Metabolic Diseases (11) · Optic Atrophy (11) · Optic Nerve Diseases (11) · Optic Atrophies, Hereditary (10) · Genetic Diseases, Inborn (8) · Nervous System Diseases (8) · Neurodegenerative Diseases (8) · Mitochondrial Diseases (7) · Eye Diseases (5) · Eye Diseases, Hereditary (5) · Congenital Abnormalities (2)
salicylhydroxamic acid (3) · Curcumin (1) · idebenone (1)
GenSight Biologics (4) · Wuhan Neurophth Biotechnology Limited Company (2) · Huazhong University of Science and Technology (1) · Laboratoires Thea (1) · Mahidol University (1) · ProQR Therapeutics (1) · Santhera Pharmaceuticals (1)
France (6) · United Kingdom (6) · United States (6) · Germany (5) · Italy (5) · Belgium (3) · China (3) · Brazil (2) · Canada (2) · Netherlands (2) · Spain (2) · Australia (1) · Taiwan (1) · Thailand (1)
Nancy Newman, MD (2) · Bin Li, MD (1) · Bin Li, PhD,MD (1) · Nancy J. Newman, MD (1) · Patrick Yu Wai Man, MDPhDFRCOpht (1) · Patrick Yu-Wai-Man, PhD (1) · Professor Wanicha L Chuenkongkaew, MD (1) · Yong Zhang, PhD,MD (1)
PMID 36449262, PMID 34108929, PMID 1463007, PMID 1674640, PMID 18441370, PMID 18441371, PMID 18774912, PMID 1928099, PMID 22612072, PMID 22644094, PMID 23063736, PMID 24396955, PMID 25950380, PMID 26606867, PMID 26892229, PMID 27386292, PMID 27426279, PMID 27749593, PMID 31237206, PMID 3201231, PMID 33451738, PMID 39699886, PMID 5063796
2015 (2), 2017 (2), 2018 (1)