Leber Hereditary Optic Disease

Clinical trial pipeline · Data from ClinicalTrials.gov

See which Leber Hereditary Optic Disease trials you may qualify for

Total trials

Actively recruiting

Phase 3 trials

Completed

Trial phase breakdown

Trial status

Actively recruiting — 10 trials

RecruitingPhase 3

NCT06891443

Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)

The purpose of this double-masked, randomized, placebo-controlled, paired-eye study is to evaluate the efficacy, safety and tolerability of Sepofarsen in subjec…

United States32 participantsStarted 2025-06-04

Treatment: sepofarsen, Placebo IVT

RecruitingPhase 2

NCT07303296

Efficacy and Safety Study of Bilateral IVT Injection of GS010 at Two Dose Levels in LHON Patients

The goal of this Clinical trial is to assess the safety and efficacy of GS010 at two dose levels on visual acuity and retinal mitochondrial activity in patients…

France14 participantsStarted 2026-01-22

Treatment: GS010 High dose, GS010 Low dose

RecruitingNot Applicable

NCT01694940

North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)

The North American Mitochondrial Disease Consortium (NAMDC) maintains a patient contact registry and tissue biorepository for patients with mitochondrial disord…

United States1,000 participantsStarted 2011-01-31

RecruitingNot Applicable

NCT03475173

New Non-invasive Modalities for Assessing Retinal Structure and Function

This study investigates a new technology to assess the structure and function inside the eye. Retinal imaging of subjects with inner and outer retinal defects t…

United States500 participantsStarted 2019-05-06

Treatment: LSFG-NAVI

RecruitingNot Applicable

NCT06682819

Metabolomics Analysis According to the Retinal Nerve Fiber Layer in Patients With NOHL Mutations (MétabOCT)

Leber hereditary optic neuropathy (LHON), due to mitochondrial DNA (mtDNA) mutations, is responsible for profound visual impairment. However, there is evidence…

France90 participantsStarted 2023-03-10

Treatment: Optical coherent tomography

RecruitingNot Applicable

NCT01793168

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

CoRDS, or the Coordination of Rare Diseases at Sanford, is based at Sanford Research in Sioux Falls, South Dakota. It provides researchers with a centralized, i…

United States, Australia20,000 participantsStarted 2010-07

All recent trials (73 shown)

CompletedPhase 3

NCT03293524

Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year

The goal of this clinical trial is to assess the efficacy and safety of GS010 gene therapy - (lenadogene nolparvovec) in subjects with LHON due to the G11778A N…

United States98 participantsStarted 2018-03-12

Treatment: GS010, Placebo

CompletedPhase 3

NCT02652767

Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 Mutation

The goal of this clinical trial is to assess the effectiveness of GS010, a gene therapy, in improving the visual outcome in participants with Leber Hereditary O…

United States, France, Germany39 participantsStarted 2016-02-23

Treatment: GS010, Sham Intravitreal Injection

Leber Hereditary Optic Disease

Trial phase breakdown

Trial status

Actively recruiting — 10 trials

Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)

Efficacy and Safety Study of Bilateral IVT Injection of GS010 at Two Dose Levels in LHON Patients

North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)

New Non-invasive Modalities for Assessing Retinal Structure and Function

Metabolomics Analysis According to the Retinal Nerve Fiber Layer in Patients With NOHL Mutations (MétabOCT)

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

All recent trials (73 shown)

Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year

Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 Mutation

Trial phase breakdown

Trial status

Actively recruiting — 10 trials

Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)

Efficacy and Safety Study of Bilateral IVT Injection of GS010 at Two Dose Levels in LHON Patients

North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)

New Non-invasive Modalities for Assessing Retinal Structure and Function

Metabolomics Analysis According to the Retinal Nerve Fiber Layer in Patients With NOHL Mutations (MétabOCT)

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

All recent trials (73 shown)

Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year

Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 Mutation

REALITY LHON Registry

Efficacy Study of GS010 for Treatment of Vision Loss From 7 Months to 1 Year From Onset in LHON Due to the ND4 Mutation…

EAP_GS010_single Patient

RESCUE and REVERSE Long-term Follow-up

Safety Evaluation of Gene Therapy in Leber Hereditary Optic Neuropathy (LHON) Patients

A Phase 3, Multicenter, Randomized, Double-Masked, Sham-Controlled Clinical Trial for Leber's Hereditary Optic Neuropath…

Pilot Study of the Efficacy of Nicotinamide (Vitamin B3) in Leber's Hereditary Optic Neuropathy

A Patient Registry Study for Patients Treated With Voretigene Neparvovec in US

Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis

Phase I Trial of Gene Vector to Patients With Retinal Disease Due to RPE65 Mutations

Optic Nerve Head Evaluation Through Multimodal Blood Flow Analysis: a Prospective Observational, Multi-center Study

Psychotherapy Group for Parents of Children With LCA

Safety Study of an Adeno-associated Virus Vector for Gene Therapy of Leber's Hereditary Optic Neuropathy

Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2

Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis

Long-term Follow-up Study in Subjects Who Received Voretigene Neparvovec-rzyl (AAV2-hRPE65v2)

Early Check: Expanded Screening in Newborns

Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease