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ClinicalTrials.gov public records Last synced Oct 2, 2026, 5:18 AM EDT

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Showing 1–24 of 24 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Leber's Hereditary Optic Neuropathy Clear all
No Longer Available No phase listed Expanded access

Emergency Administration of EPI-743 to a Single Patient With Leber's Hereditary Optic Neuropathy [LHON]

NCT02300753
View directory record Official record
Conditions
Leber's Hereditary Optic Neuropathy
Interventions
EPI-743
Drug
Lead sponsor
Edison Pharmaceuticals Inc
Industry
Eligibility
8 Years to 65 Years
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Nov 25, 2014 · Synced Oct 2, 2026, 5:18 AM EDT
Completed Phase 1 Interventional Results available

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

NCT02161380
View directory record Official record
Conditions
Leber's Hereditary Optic Neuropathy
Interventions
injection of scAAV2-P1ND4v2 1.18x10e9 vg (Low),, injection of scAAV2-P1ND4v2 5.81 X10e9 vg (Med), injection of scAAV2-P1ND4v2 2.4 X10e10vg (High), injection of scAAV2-P1ND4v2 1.0 X10e11vg (Higher)
Drug
Lead sponsor
Byron Lam
Other
Eligibility
15 Years and older
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2025
U.S. locations
1
States / cities
Miami, Florida
Source: ClinicalTrials.gov public record
Updated Jun 5, 2025 · Synced Oct 2, 2026, 5:18 AM EDT
Completed Phase 1Phase 2 Interventional Results available

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

NCT03140969
View directory record Official record
Conditions
Leber's Congenital Amaurosis
Interventions
QR-110
Drug
Lead sponsor
Laboratoires Thea
Industry
Eligibility
6 Years and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
2
States / cities
Iowa City, Iowa • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 15, 2024 · Synced Oct 2, 2026, 5:18 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND4 Mutations

NCT05293626
View directory record Official record
Conditions
Leber Hereditary Optic Neuropathy (LHON)
Interventions
NR082 Injection, Injection needle
Drug · Device
Lead sponsor
Neurophth Therapeutics Inc
Other
Eligibility
18 Years to 75 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
3
States / cities
Palo Alto, California • Aurora, Colorado • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 4, 2024 · Synced Oct 2, 2026, 5:18 AM EDT
Terminated No phase listed Observational Accepts healthy volunteers

Biomechanics of Optic Neuropathy

NCT02982499
View directory record Official record
Conditions
Optic Neuropathy
Interventions
magnetic resonance image (MRI), Optical Coherence Tomography (OCT)
Device
Lead sponsor
University of Miami
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2018
U.S. locations
1
States / cities
Miami, Florida
Source: ClinicalTrials.gov public record
Updated Oct 23, 2020 · Synced Oct 2, 2026, 5:18 AM EDT
Completed Phase 1 Interventional

Safety Study of a Single IVT Injection of QPI-1007 in Chronic Optic Nerve Atrophy and Recent Onset NAION Patients

NCT01064505
View directory record Official record
Conditions
Optic Atrophy, Non-arteritic Anterior Ischemic Optic Neuropathy
Interventions
QPI-1007 at various doses
Drug
Lead sponsor
Quark Pharmaceuticals
Industry
Eligibility
50 Years and older
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
22
States / cities
Phoenix, Arizona • Beverly Hills, California • Los Angeles, California + 19 more
Source: ClinicalTrials.gov public record
Updated May 13, 2013 · Synced Oct 2, 2026, 5:18 AM EDT
Completed Phase 2 Interventional Results available

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

NCT02023866
View directory record Official record
Conditions
Inherited Mitochondrial Disease, Including Leigh Syndrome
Interventions
Cysteamine Bitartrate
Drug
Lead sponsor
Amgen
Industry
Eligibility
6 Years to 17 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
5
States / cities
San Diego, California • Stanford, California • Akron, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 27, 2024 · Synced Oct 2, 2026, 5:18 AM EDT
Completed Phase 3 Interventional Results available

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

NCT02652767
View directory record Official record
Conditions
Optic, Atrophy, Hereditary, Leber
Interventions
GS010, Sham Intravitreal Injection
Biological · Device
Lead sponsor
GenSight Biologics
Industry
Eligibility
15 Years and older
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
3
States / cities
Los Angeles, California • Atlanta, Georgia • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 2, 2026 · Synced Oct 2, 2026, 5:18 AM EDT
Recruiting Phase 3 Interventional

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

NCT06891443
View directory record Official record
Conditions
Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Sensation Disorders, Vision Disorder, Neurological Manifestations, Eye Diseases, Hereditary, Eye Diseases, Eye Disorders Congenital, Retinal Disease
Interventions
sepofarsen, Placebo IVT
Drug · Other
Lead sponsor
Laboratoires Thea
Industry
Eligibility
6 Years and older
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
5
States / cities
San Francisco, California • Miami, Florida • Iowa City, Iowa + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 25, 2026 · Synced Oct 2, 2026, 5:18 AM EDT
Terminated Phase 2 Interventional Results available

A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease

NCT02473445
View directory record Official record
Conditions
Mitochondrial Diseases
Interventions
Cysteamine Bitartrate
Drug
Lead sponsor
Amgen
Industry
Eligibility
6 Years to 17 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
5
States / cities
San Diego, California • Stanford, California • Akron, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 27, 2024 · Synced Oct 2, 2026, 5:18 AM EDT
Completed No phase listed Observational

REALITY LHON Registry

NCT03295071
View directory record Official record
Conditions
Leber Hereditary Optic Neuropathy
Interventions
Patient-reported outcomes (PROs)
Other
Lead sponsor
GenSight Biologics
Industry
Eligibility
Not listed
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
5
States / cities
Pasadena, California • Atlanta, Georgia • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated Mar 2, 2026 · Synced Oct 2, 2026, 5:18 AM EDT
Not yet recruiting Phase 1Phase 2 Interventional

A Basket Clinical Study to Assess Glycerol Tributyrate in Patients With Mitochondrial Encephalopathy, Lactic Acidosis, Stroke-like Episodes (MELAS) or Leber's Hereditary Optic Neuropathy-Plus (LHON-Plus)

NCT06792500
View directory record Official record
Conditions
MELAS Syndrome, Lebers Hereditory Optic Neuropathy With Extra Ocular Symptoms (LHON-Plus)
Interventions
Glycerol Tributyrate
Drug
Lead sponsor
George Washington University
Other
Eligibility
18 Years to 65 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Jun 4, 2026 · Synced Oct 2, 2026, 5:18 AM EDT
Active, not recruiting Phase 2Phase 3 Interventional

A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)

NCT03913143
View directory record Official record
Conditions
Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders, Sensation Disorders, Neurologic Manifestations, Eye Diseases, Eye Diseases, Hereditary, Eye Disorders Congenital, Retinal Disease
Interventions
sepofarsen, Sham
Drug · Other
Lead sponsor
ProQR Therapeutics
Industry
Eligibility
8 Years and older
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
1
States / cities
Iowa City, Iowa
Source: ClinicalTrials.gov public record
Updated Mar 17, 2022 · Synced Oct 2, 2026, 5:18 AM EDT
Completed Phase 4 Interventional Results available

Study to Assess the Efficacy and Safety of Raxone in LHON Patients

NCT02774005
View directory record Official record
Conditions
Leber's Hereditary Optic Neuropathy (LHON)
Interventions
Idebenone
Drug
Lead sponsor
Santhera Pharmaceuticals
Industry
Eligibility
12 Years and older
Enrollment
199 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
9
States / cities
Phoenix, Arizona • Palo Alto, California • Stanford, California + 6 more
Source: ClinicalTrials.gov public record
Updated Apr 21, 2023 · Synced Oct 2, 2026, 5:18 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

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

NCT01694940
View directory record Official record
Conditions
Mitochondrial Disorders, Mitochondrial Genetic Disorders, Mitochondrial Diseases, Disorder of Mitochondrial Respiratory Chain Complexes, Deletion and Duplication of Mitochondrial DNA
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2026
U.S. locations
16
States / cities
San Diego, California • Stanford, California • Aurora, Colorado + 12 more
Source: ClinicalTrials.gov public record
Updated Feb 4, 2026 · Synced Oct 2, 2026, 5:18 AM EDT
Terminated Phase 1Phase 2 Interventional

Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND1 Mutations

NCT05820152
View directory record Official record
Conditions
Leber Hereditary Optic Neuropathy (LHON)
Interventions
NFS-02 Injection
Drug
Lead sponsor
Neurophth Therapeutics Inc
Other
Eligibility
18 Years to 75 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Sep 20, 2024 · Synced Oct 2, 2026, 5:18 AM EDT
Completed Phase 3 Interventional Results available

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

NCT03293524
View directory record Official record
Conditions
Leber Hereditary Optic Neuropathy
Interventions
GS010, Placebo
Genetic · Drug
Lead sponsor
GenSight Biologics
Industry
Eligibility
15 Years and older
Enrollment
98 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
7
States / cities
Pasadena, California • Aurora, Colorado • Atlanta, Georgia + 4 more
Source: ClinicalTrials.gov public record
Updated Apr 16, 2026 · Synced Oct 2, 2026, 5:18 AM EDT
Completed Phase 3 Interventional Results available

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)

NCT02652780
View directory record Official record
Conditions
Optic, Atrophy, Hereditary, Leber
Interventions
GS010, Sham Intravitreal Injection
Biological · Device
Lead sponsor
GenSight Biologics
Industry
Eligibility
15 Years and older
Enrollment
37 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
3
States / cities
Los Angeles, California • Atlanta, Georgia • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 2, 2026 · Synced Oct 2, 2026, 5:18 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Extension Study to Study PQ-110-001 (NCT03140969)

NCT03913130
View directory record Official record
Conditions
Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders, Sensation Disorders, Neurologic Manifestations, Eye Diseases, Eye Diseases, Hereditary, Eye Disorders Congenital, Retinal Disease
Interventions
QR-110
Drug
Lead sponsor
Laboratoires Thea
Industry
Eligibility
6 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
2
States / cities
Iowa City, Iowa • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 15, 2024 · Synced Oct 2, 2026, 5:18 AM EDT
Completed Phase 3 Interventional Results available

RESCUE and REVERSE Long-term Follow-up

NCT03406104
View directory record Official record
Conditions
Leber Hereditary Optic Neuropathy
Interventions
GS010, Sham Intravitreal Injection
Genetic · Other
Lead sponsor
GenSight Biologics
Industry
Eligibility
15 Years and older
Enrollment
62 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
3
States / cities
Pasadena, California • Atlanta, Georgia • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 2, 2026 · Synced Oct 2, 2026, 5:18 AM EDT
Completed Phase 2 Interventional Results available

A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for Treatment of Leber's Hereditary Optic Neuropathy

NCT02693119
View directory record Official record
Conditions
Leber's Hereditary Optic Neuropathy
Interventions
elamipretide (MTP-131) 1% topical ophthalmic solution, Vehicle topical ophthalmic solution
Drug
Lead sponsor
Stealth BioTherapeutics Inc.
Industry
Eligibility
18 Years to 50 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
Pasadena, California
Source: ClinicalTrials.gov public record
Updated Nov 30, 2021 · Synced Oct 2, 2026, 5:18 AM EDT
Recruiting Not applicable Interventional

Stem Cell Ophthalmology Treatment Study II

NCT03011541
View directory record Official record
Conditions
Retinal Disease, Age-Related Macular Degeneration, Retinitis Pigmentosa, Stargardt Disease, Optic Neuropathy, Nonarteritic Ischemic Optic Neuropathy, Optic Atrophy, Optic Nerve Disease, Glaucoma, Leber Hereditary Optic Neuropathy, Blindness, Vision Loss Night, Vision Loss Partial, Vision, Low, Retinopathy, Maculopathy, Macular Degeneration, Retina Atrophy
Interventions
Arm 1
Procedure
Lead sponsor
MD Stem Cells
Industry
Eligibility
18 Years and older
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2028
U.S. locations
2
States / cities
Westport, Connecticut • Coral Springs, Florida
Source: ClinicalTrials.gov public record
Updated Jun 29, 2026 · Synced Oct 2, 2026, 5:18 AM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

New Non-invasive Modalities for Assessing Retinal Structure and Function

NCT03475173
View directory record Official record
Conditions
Ischemic Optic Neuropathy, Branch Retinal Artery Occlusion, Hemianopia, Leber Hereditary Optic Neuropathy, Acute Zonal Occult Outer Retinopathy
Interventions
LSFG-NAVI
Device
Lead sponsor
Randy Kardon
Other
Eligibility
18 Years to 99 Years
Enrollment
500 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2028
U.S. locations
1
States / cities
Iowa City, Iowa
Source: ClinicalTrials.gov public record
Updated Sep 8, 2026 · Synced Oct 2, 2026, 5:18 AM EDT
Terminated Phase 1Phase 2 Interventional Accepts healthy volunteers Results available

Near-infrared Light-emitting Diode (NIR-LED) Therapy for Leber's Hereditary Optic Neuropathy (LHON)

NCT01389817
View directory record Official record
Conditions
Leber's Hereditary Optic Neuropathy (LHON)
Interventions
Near-infrared light-emitting diode (NIR-LED) therapy (Med Light 630 PRO (Medical Devices Inc.))
Device
Lead sponsor
Medical College of Wisconsin
Other
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2013
U.S. locations
3
States / cities
Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Sep 29, 2014 · Synced Oct 2, 2026, 5:18 AM EDT