Leber Hereditary Optic Neuropathy: Clinical Trials

10 trials for Leber Hereditary Optic Neuropathy, newest first

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5 completed, 3 recruiting, 2 unknown. 4 Not applicable, 3 Phase 2, 3 Phase 3, 1 Phase 1.

By phase and status

PhaseTrialsStatus
Not applicable4Recruiting 3, Completed 1
Phase 23Unknown 2, Completed 1
Phase 33Completed 2, Unknown 1
Phase 11Completed 1

Follow Leber Hereditary Optic Neuropathy: new trials by email Literature Review of Leber Hereditary Optic Neuropathy trials Research Report on Leber Hereditary Optic Neuropathy

Trials newest first · largest first

Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.

TrialPhaseStatusSponsorEnrollmentStartUpdated
Metabolomics Analysis According to the Retinal Nerve Fiber Layer in Patients With NOHL Mutations (MétabOCT)
Optical coherent tomography - Healthy Subjects, Leber Hereditary Optic Neuropathy
Not applicableRecruitingHôpital Necker-Enfants Malades902023-03-102025-11-18
New Non-invasive Modalities for Assessing Retinal Structure and Function
LSFG-NAVI - Ischemic Optic Neuropathy, Branch Retinal Artery Occlusion
Not applicableRecruitingRandy Kardon5002019-05-062026-09-08
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 3CompletedGenSight Biologics982018-03-122026-04-16
RESCUE and REVERSE Long-term Follow-up
GS010, Sham Intravitreal Injection - Leber Hereditary Optic Neuropathy
Phase 3CompletedGenSight Biologics622018-01-092026-03-02
REALITY LHON Registry
Patient-reported outcomes (PROs) - Leber Hereditary Optic Neuropathy
CompletedGenSight Biologics442018-01-032026-03-02
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 3UnknownHuazhong University of Science and Technology1592017-12-272020-09-17
Stem Cell Ophthalmology Treatment Study II
Arm 1 - Retinal Disease, Age-Related Macular Degeneration
Not applicableRecruitingMD Stem Cells5002016-01-012026-06-29
Safety Evaluation of Gene Therapy in Leber Hereditary Optic Neuropathy (LHON) Patients
GS010 - Leber Hereditary Optic Neuropathy
Phase 1 / Phase 2CompletedGenSight Biologics192014-02-132026-03-02
Trial of Cyclosporine in the Acute Phase of Leber Hereditary Optic Neuropathy
cyclosporine - Leber Hereditary Optic Neuropathy
Phase 2UnknownUniversity Hospital, Angers122011-07-012014-06-27
Safety and Efficacy Study of rAAV2-ND4 Treatment of Leber Hereditary Optic Neuropathy (LHON)
rAAV2-ND4 - Leber Hereditary Optic Neuropathy
Not applicableCompletedBin Li92011-04-012018-01-31

Narrower conditions

Optic Atrophy, Hereditary, Leber (10)  ·  Eye Diseases (4)  ·  Eye Diseases, Hereditary (3)  ·  Mitochondrial Diseases (3)  ·  Genetic Diseases, Inborn (2)  ·  Nervous System Diseases (2)  ·  Neurodegenerative Diseases (2)  ·  Optic Nerve Diseases (2)  ·  Optic Neuropathy, Ischemic (2)  ·  Retinal Diseases (2)  ·  Acute zonal occult outer retinopathy (1)  ·  Blindness (1)  ·  Cone Dystrophy (1)  ·  Cone-Rod Dystrophies (1)  ·  Doyne honeycomb retinal dystrophy (1)

Broader conditions

Congenital, Hereditary, and Neonatal Diseases and Abnormalities (10)  ·  Cranial Nerve Diseases (10)  ·  Heredodegenerative Disorders, Nervous System (10)  ·  Metabolic Diseases (10)  ·  Nutritional and Metabolic Diseases (10)  ·  Optic Atrophies, Hereditary (9)  ·  Optic Atrophy (9)  ·  Genetic Diseases, Inborn (8)  ·  Nervous System Diseases (8)  ·  Neurodegenerative Diseases (8)  ·  Optic Nerve Diseases (8)  ·  Eye Diseases, Hereditary (7)  ·  Mitochondrial Diseases (7)  ·  Eye Diseases (6)  ·  Cardiovascular Diseases (2)

Interventions

Cyclosporine (1)  ·  Patient Reported Outcome Measures (1)  ·  salicylhydroxamic acid (1)

Sponsors

GenSight Biologics (4)  ·  Bin Li (1)  ·  Huazhong University of Science and Technology (1)  ·  Hôpital Necker-Enfants Malades (1)  ·  MD Stem Cells (1)  ·  Randy Kardon (1)  ·  University Hospital, Angers (1)

Countries

France (6)  ·  United States (5)  ·  Italy (3)  ·  United Kingdom (3)  ·  China (2)  ·  Spain (2)  ·  Austria (1)  ·  Belgium (1)  ·  Germany (1)  ·  Taiwan (1)  ·  United Arab Emirates (1)

Top investigators

Nancy Newman, MD (2)  ·  Bin Li, PhD,MD (1)  ·  Christophe Orssaud, MD (1)  ·  Jeffrey Weiss, MD (1)  ·  Patrick Yu-Wai-Man, PhD (1)  ·  Randy H Kardon, MD, PhD (1)  ·  Steven Levy, MD (1)  ·  Yong Zhang, PhD,MD (1)  ·  bin Li, PhD,MD (1)

Results publications

PMID 36449262, PMID 26892229, 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 26199618, PMID 26604914, PMID 26606867, PMID 27386292, PMID 27426279, PMID 27749593, PMID 27857759, PMID 27904503, PMID 29270420, PMID 29454364, PMID 30050918, PMID 30878986, PMID 31119146, PMID 31237206, PMID 31620478, PMID 3201231, PMID 32039263, PMID 32231088, PMID 33546345, PMID 39699886, PMID 5063796

NIH funding

2017 (2)