Leber Hereditary Optic Neuropathy (LHON): Clinical Trials

5 trials for Leber Hereditary Optic Neuropathy (LHON), newest first

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2 active not recruiting, 1 completed, 1 not yet recruiting, 1 terminated. 3 Phase 1, 2 Phase 2, 1 Phase 3.

By phase and status

PhaseTrialsStatus
Phase 13Active not recruiting 1, Not yet recruiting 1, Terminated 1
Phase 22Active not recruiting 1, Terminated 1
Phase 31Active not recruiting 1

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

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
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 1Not yet recruitingUniversity Hospital, Angers132026-04-012025-12-18
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 3Active not recruitingWuhan Neurophth Biotechnology Limited Company952024-09-192026-02-12
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 2TerminatedNeurophth Therapeutics Inc112023-08-152024-09-20
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 2Active not recruitingNeurophth Therapeutics Inc122023-05-222024-09-04
Leber Hereditary Optic Neuropathy (LHON) Historical Case Record Survey
Leber Hereditary Optic Neuropathy (LHON)
CompletedSanthera Pharmaceuticals3062013-08-012014-05-15

Narrower conditions

Optic Atrophy, Hereditary, Leber (5)  ·  Mitochondrial Diseases (1)  ·  Optic Nerve Diseases (1)

Broader conditions

Congenital, Hereditary, and Neonatal Diseases and Abnormalities (5)  ·  Cranial Nerve Diseases (5)  ·  Eye Diseases (5)  ·  Eye Diseases, Hereditary (5)  ·  Genetic Diseases, Inborn (5)  ·  Heredodegenerative Disorders, Nervous System (5)  ·  Metabolic Diseases (5)  ·  Nervous System Diseases (5)  ·  Neurodegenerative Diseases (5)  ·  Nutritional and Metabolic Diseases (5)  ·  Optic Atrophies, Hereditary (5)  ·  Optic Atrophy (5)  ·  Mitochondrial Diseases (4)  ·  Optic Nerve Diseases (4)

Interventions

Needles (1)  ·  Syringes (1)  ·  salicylhydroxamic acid (1)

Sponsors

Neurophth Therapeutics Inc (2)  ·  Santhera Pharmaceuticals (1)  ·  University Hospital, Angers (1)  ·  Wuhan Neurophth Biotechnology Limited Company (1)

Countries

China (2)  ·  France (2)  ·  United States (2)  ·  Belgium (1)  ·  Denmark (1)  ·  Italy (1)  ·  Slovenia (1)

Top investigators

Jose-Alain Sahel, MD (1)

NIH funding

2025 (1)