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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Optic Nerve Atrophy Clear all
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 12, 2013 · Synced Jul 22, 2026, 7:35 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Identifying Biomarkers of Parkinson's Disease Using Magnetic Resonance Imaging (MRI)

NCT01888185
View directory record Official record
Conditions
Parkinson's Disease (PD), Parkinsonism, Progressive Supranuclear Palsy (PSP), Multiple System Atrophy (MSA)
Interventions
Not listed
Lead sponsor
Milton S. Hershey Medical Center
Other
Eligibility
21 Years to 90 Years
Enrollment
290 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2019
U.S. locations
1
States / cities
Hershey, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 2, 2020 · Synced Jul 22, 2026, 7:35 PM 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 15, 2026 · Synced Jul 22, 2026, 7:35 PM 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 1, 2026 · Synced Jul 22, 2026, 7:35 PM 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 26, 2024 · Synced Jul 22, 2026, 7:35 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Facilitating Diagnostics and Prognostics of Parkinsonian Syndromes Using Neuroimaging

NCT03872102
View directory record Official record
Conditions
Parkinson Disease, Multiple System Atrophy, Progressive Supranuclear Palsy
Interventions
Not listed
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
Not listed
Enrollment
90 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2026
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jan 26, 2026 · Synced Jul 22, 2026, 7:35 PM EDT
Recruiting No phase listed Observational

NYSCF Scientific Discovery Biobank

NCT06203106
View directory record Official record
Conditions
ALS, Amyotrophic Lateral Sclerosis, Alzheimer Disease, Alzheimer Disease, Early Onset, Alzheimer Disease, Late Onset, Batten Disease, Corticobasal Degeneration, Dementia, Frontotemporal Dementia, Huntington Disease, Lewy Body Disease, Multiple Sclerosis, Multiple System Atrophy, Parkinson Disease, Parkinson's Disease and Parkinsonism, Progressive Supranuclear Palsy, INAD, Diabetes, Diabetes Mellitus, Diabetes Mellitus, Type 2, Diabetes Mellitus, Type 1, Macular Degeneration, Ovarian Cancer, Cervical Cancer, Uterine Cancer, Vaginal Cancer, Vulvar Cancer, PTSD, Post Traumatic Stress Disorder
Interventions
Biological Sample Collection
Other
Lead sponsor
New York Stem Cell Foundation Research Institute
Other
Eligibility
30 Days and older
Enrollment
10,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2045
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 2, 2025 · Synced Jul 22, 2026, 7:35 PM 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 4, 2025 · Synced Jul 22, 2026, 7:35 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Biomarkers in Neural Disorders

NCT02761707
View directory record Official record
Conditions
Parkinson's Disease, Alzheimer's Disease, Progressive Supranuclear Palsy, Essential Tremor, Multiple System Atrophy, Drug Induced Parkinson's Disease, Diffuse Lewy Body Disease, Myasthenia Gravis, Spinal Cord Injuries
Interventions
Electrical Brainstem Responses, Olfactory Tests, Visual Deprivation, Peripheral Nerve Stimulation
Other
Lead sponsor
University of Pennsylvania
Other
Eligibility
18 Years to 80 Years
Enrollment
54 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2019
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 17, 2019 · Synced Jul 22, 2026, 7:35 PM EDT
Recruiting Not applicable Interventional

Neurologic Stem Cell Treatment Study

NCT02795052
View directory record Official record
Conditions
Neurologic Disorders, Nervous System Diseases, Neurodegenerative Diseases, Neurological Disorders, Stroke, Traumatic Brain Injury, Cadasil, Chronic Traumatic Encephalopathy, Cerebral Infarction, Cerebral Ischemia, Cerebral Stroke, Cerebral Hemorrhage, Parkinson, Multi-System Degeneration, MSA - Multiple System Atrophy, Progressive Supranuclear Palsy, ALS, Amyotrophic Lateral Sclerosis, Neuropathy, Diabetic Neuropathies, Alzheimer Disease, Dementia, Frontotemporal Dementia, Lewy Body Disease, Cognitive Impairment, Lewy Body Variant of Alzheimer Disease
Interventions
Intravenous and Intranasal BMSC
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 25, 2026 · Synced Jul 22, 2026, 7:35 PM EDT
Completed No phase listed Observational

Defining Phenotypes of Movement Disorders :Parkinson's Plus Disorders (PD), Essential Tremor (ET), Cortical Basal Degeneration (CBD), Multiple Systems Atrophy (MSA), Magnetoencephalography.

NCT02132052
View directory record Official record
Conditions
Essential Tremor, Multiple System Atrophy, Corticobasal Degeneration, Supranuclear Palsy, Progressive, Parkinson Disease
Interventions
Not listed
Lead sponsor
University of Colorado, Denver
Other
Eligibility
40 Years and older
Enrollment
81 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Jun 24, 2021 · Synced Jul 22, 2026, 7:35 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Misfolded Proteins in the Skin of People With Parkinson's Disease and Other Parkinsonism

NCT04518059
View directory record Official record
Conditions
Parkinson Disease, Parkinsonism, Dementia With Lewy Bodies, Multiple System Atrophy, Progressive Supranuclear Palsy, Corticobasal Degeneration
Interventions
punch skin biopsy
Procedure
Lead sponsor
University Hospitals Cleveland Medical Center
Other
Eligibility
21 Years to 89 Years
Enrollment
184 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2025
U.S. locations
2
States / cities
Cleveland, Ohio • South Euclid, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 30, 2025 · Synced Jul 22, 2026, 7:35 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Inherited Retinal Degenerative Disease Registry

NCT02435940
View directory record Official record
Conditions
Eye Diseases Hereditary, Retinal Disease, Achromatopsia, Bardet-Biedl Syndrome, Bassen-Kornzweig Syndrome, Batten Disease, Best Disease, Choroidal Dystrophy, Choroideremia, Cone Dystrophy, Cone-Rod Dystrophy, Congenital Stationary Night Blindness, Enhanced S-Cone Syndrome, Fundus Albipunctatus, Goldmann-Favre Syndrome, Gyrate Atrophy, Juvenile Macular Degeneration, Kearns-Sayre Syndrome, Leber Congenital Amaurosis, Refsum Syndrome, Retinitis Pigmentosa, Retinitis Punctata Albescens, Retinoschisis, Rod-Cone Dystrophy, Rod Dystrophy, Rod Monochromacy, Stargardt Disease, Usher Syndrome
Interventions
Not listed
Lead sponsor
Foundation Fighting Blindness
Other
Eligibility
Not listed
Enrollment
20,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2037
U.S. locations
1
States / cities
Columbia, Maryland
Source: ClinicalTrials.gov public record
Updated May 18, 2026 · Synced Jul 22, 2026, 7:35 PM 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 14, 2024 · Synced Jul 22, 2026, 7:35 PM 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 Nov 23, 2025 · Synced Jul 22, 2026, 7:35 PM 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 28, 2026 · Synced Jul 22, 2026, 7:35 PM EDT
Active, not recruiting No phase listed Observational

Automated Imaging Differentiation of Parkinsonism

NCT05913687
View directory record Official record
Conditions
Parkinson Disease, Multiple System Atrophy, Parkinson Variant, Progressive Supranuclear Palsy
Interventions
AIDP
Diagnostic Test
Lead sponsor
University of Florida
Other
Eligibility
40 Years to 80 Years
Enrollment
315 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
19
States / cities
Birmingham, Alabama • La Jolla, California • San Francisco, California + 15 more
Source: ClinicalTrials.gov public record
Updated Jun 25, 2026 · Synced Jul 22, 2026, 7:35 PM EDT
Active, not recruiting Phase 2 Interventional

AMX0035 in Adult Patients With Wolfram Syndrome

NCT05676034
View directory record Official record
Conditions
Wolfram Syndrome
Interventions
AMX0035
Drug
Lead sponsor
Amylyx Pharmaceuticals Inc.
Industry
Eligibility
17 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Apr 13, 2026 · Synced Jul 22, 2026, 7:35 PM 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 26, 2024 · Synced Jul 22, 2026, 7:35 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Diagnostic and Prognostic Biomarkers in Parkinson Disease

NCT00653783
View directory record Official record
Conditions
Parkinson Disease, Multiple System Atrophy, Progressive Supranuclear Palsy
Interventions
Not listed
Lead sponsor
The Parkinson Study Group
Network
Eligibility
18 Years and older
Enrollment
200 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2007
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Dec 30, 2008 · Synced Jul 22, 2026, 7:35 PM EDT
Completed No phase listed Observational

I-Tracking Neurodegeneration in Early Wolfram Syndrome

NCT03951298
View directory record Official record
Conditions
Wolfram Syndrome
Interventions
Not listed
Lead sponsor
Washington University School of Medicine
Other
Eligibility
1 Day and older
Enrollment
127 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Aug 5, 2024 · Synced Jul 22, 2026, 7:35 PM 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 3, 2024 · Synced Jul 22, 2026, 7:35 PM 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 3, 2026 · Synced Jul 22, 2026, 7:35 PM EDT
Completed Phase 4 Interventional

Trial of Parkinson's And Zoledronic Acid

NCT03924414
View directory record Official record
Conditions
Parkinson Disease, Osteoporosis, Parkinsonism, Parkinson's Disease and Parkinsonism, Atypical Parkinsonism, Progressive Supranuclear Palsy, Multiple System Atrophy, Vascular Parkinsonism, Dementia With Lewy Bodies
Interventions
Zoledronic Acid 5Mg/Bag 100Ml Inj, Placebo
Drug · Other
Lead sponsor
California Pacific Medical Center Research Institute
Other
Eligibility
60 Years and older
Enrollment
2,721 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
77
States / cities
Scottsdale, Arizona • Fayetteville, Arkansas • Little Rock, Arkansas + 58 more
Source: ClinicalTrials.gov public record
Updated Jun 24, 2026 · Synced Jul 22, 2026, 7:35 PM EDT