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ClinicalTrials.gov public records Last synced Oct 26, 2026, 4:51 AM EDT

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Showing 1–24 of 172 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Retinal Dystrophy Clear all
Completed No phase listed Observational

A Genetic Analysis of Usher Syndrome in Ashkenazi Jews

NCT00016471
View directory record Official record
Conditions
Usher Syndrome, Retinitis Pigmentosa, Congenital Hearing Impairment
Interventions
Audiogram, Electroretinogram
Procedure
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2002
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 26, 2026, 4:51 AM EDT
Active, not recruiting Phase 2 Interventional

Phase 2 Tolerability and Effects of ALK-001 on Stargardt Disease

NCT02402660
View directory record Official record
Conditions
Stargardt Disease, Stargardt Macular Degeneration, Stargardt Macular Dystrophy, Autosomal Recessive Stargardt Disease 1 (ABCA4-related)
Interventions
ALK-001, Placebo
Drug
Lead sponsor
Alkeus Pharmaceuticals, Inc.
Industry
Eligibility
8 Years to 70 Years
Enrollment
160 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2028
U.S. locations
14
States / cities
Phoenix, Arizona • Los Angeles, California • Aurora, Colorado + 11 more
Source: ClinicalTrials.gov public record
Updated Sep 21, 2026 · Synced Oct 26, 2026, 4:51 AM EDT
Completed Phase 1 Interventional

SAD of IVT VP-001 in PRPF31 Mutation-Associated Retinal Dystrophy Subjects

NCT05902962
View directory record Official record
Conditions
Retinal Dystrophy, PRPF31 Mutationassociated Retinal Dystrophy, RP11
Interventions
VP-001
Drug
Lead sponsor
PYC Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
6
States / cities
Jacksonville, Florida • Miami, Florida • Ann Arbor, Michigan + 3 more
Source: ClinicalTrials.gov public record
Updated May 29, 2026 · Synced Oct 26, 2026, 4:51 AM EDT
Completed No phase listed Observational

Long-Term Follow-Up Gene Therapy Study for Leber Congenital Amaurosis OPTIRPE65 (Retinal Dystrophy Associated With Defects in RPE65)

NCT02946879
View directory record Official record
Conditions
Leber Congenital Amaurosis (LCA), Eye Diseases, Eye Diseases, Hereditary, Retinal Diseases
Interventions
AAV OPTIRPE65
Biological
Lead sponsor
MeiraGTx UK II Ltd
Industry
Eligibility
3 Years to 100 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2023
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Apr 11, 2024 · Synced Oct 26, 2026, 4:51 AM EDT
Completed Not applicable Interventional

Clinical Trial of a Serious Game for Individuals With SCI/D

NCT02341950
View directory record Official record
Conditions
Spinal Cord Injury, Spinal Cord Involvement, Spina Bifida, Transverse Myelitis, Polio and Post-polio Syndrome, Syringomyelia, Spinal Stenosis, Spinal Neoplasms, Spinal Cord Diseases
Interventions
SCI Hard
Behavioral
Lead sponsor
University of Michigan
Other
Eligibility
13 Years to 29 Years
Enrollment
184 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Feb 22, 2019 · Synced Oct 26, 2026, 4:51 AM EDT
Active, not recruiting Not applicable Interventional

Activating Spinal Circuits to Improve Walking, Balance, Strength, and Reduce Spasticity

NCT05429736
View directory record Official record
Conditions
Incomplete Spinal Cord Injury, Spasticity, Muscle
Interventions
Motor Skill Training (MST), Transcutaneous Spinal Stimulation (TSS)
Behavioral · Device
Lead sponsor
Shepherd Center, Atlanta GA
Other
Eligibility
18 Years to 70 Years
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Sep 1, 2026 · Synced Oct 26, 2026, 4:51 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 26, 2026, 4:51 AM EDT
Terminated No phase listed Observational

Descemet Membrane Endothelial Keratoplasty vs. Descemet's Stripping With Endothelial Keratoplasty vs. Descemet Stripping Only

NCT04527523
View directory record Official record
Conditions
Cystoid Macular Edema, Fuchs Dystrophy
Interventions
Optical Coherence Tomography
Diagnostic Test
Lead sponsor
Wake Forest University Health Sciences
Other
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
1
States / cities
Winston-Salem, North Carolina
Source: ClinicalTrials.gov public record
Updated Jan 13, 2023 · Synced Oct 26, 2026, 4:51 AM EDT
Completed Not applicable Interventional

Auditory Neural Function in Implanted Patients With Usher Syndrome

NCT04906135
View directory record Official record
Conditions
Usher Syndrome, Cochlear Implantation
Interventions
Experimental manipulation of stimulation parameters
Other
Lead sponsor
Ohio State University
Other
Eligibility
1 Year to 85 Years
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
4
States / cities
Boston, Massachusetts • Columbus, Ohio • Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Jul 29, 2025 · Synced Oct 26, 2026, 4:51 AM EDT
Active, not recruiting Phase 2 Interventional

Open-Label Extension: Tolerability and Effects of ALK-001 on Stargardt Disease (TEASE)

NCT04239625
View directory record Official record
Conditions
Stargardt Disease, Stargardt Macular Degeneration, Stargardt Macular Dystrophy, Autosomal Recessive Stargardt Disease 1 (ABCA4-related)
Interventions
ALK-001
Drug
Lead sponsor
Alkeus Pharmaceuticals, Inc.
Industry
Eligibility
8 Years to 70 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2029
U.S. locations
8
States / cities
Phoenix, Arizona • Los Angeles, California • Aurora, Colorado + 5 more
Source: ClinicalTrials.gov public record
Updated Sep 21, 2026 · Synced Oct 26, 2026, 4:51 AM EDT
Completed Not applicable Interventional Results available

Feasibility Study of a Chronic Retinal Stimulator in Retinitis Pigmentosa

NCT00279500
View directory record Official record
Conditions
Retinitis Pigmentosa
Interventions
Argus 16 Retinal Stimulation System
Device
Lead sponsor
Second Sight Medical Products
Industry
Eligibility
18 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2014
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Apr 12, 2023 · Synced Oct 26, 2026, 4:51 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)

NCT02781480
View directory record Official record
Conditions
Leber Congenital Amaurosis
Interventions
AAV RPE65
Biological
Lead sponsor
MeiraGTx UK II Ltd
Industry
Eligibility
3 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Jul 12, 2021 · Synced Oct 26, 2026, 4:51 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety of a Single, Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Retinitis Pigmentosa

NCT02320812
View directory record Official record
Conditions
Retinitis Pigmentosa (RP)
Interventions
human retinal progenitor cells
Biological
Lead sponsor
jCyte, Inc
Industry
Eligibility
18 Years and older
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
2
States / cities
Irvine, California • Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Mar 5, 2019 · Synced Oct 26, 2026, 4:51 AM EDT
Withdrawn Not applicable Interventional

Transcorneal Electrical Stimulation for the Treatment of Retinitis Pigmentosa - a Safety and Efficacy Multicenter Study

NCT02548572
View directory record Official record
Conditions
Retinitis Pigmentosa
Interventions
Transcorneal Electrical Stimulation using Okustim device
Device
Lead sponsor
Wills Eye
Other
Eligibility
22 Years to 80 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 1, 2019 · Synced Oct 26, 2026, 4:51 AM EDT
Completed Not applicable Interventional Results available

Computer-Assisted Rehabilitation Environment Training After Argus Retinal Prosthesis

NCT03444961
View directory record Official record
Conditions
Retinitis Pigmentosa
Interventions
CAREN system training
Device
Lead sponsor
The Cleveland Clinic
Other
Eligibility
25 Years to 100 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 14, 2021 · Synced Oct 26, 2026, 4:51 AM EDT
Completed No phase listed Observational

Repository for Inherited Eye Diseases

NCT00378742
View directory record Official record
Conditions
Retinitis Pigmentosa, Inherited Ophthalmic Diseases
Interventions
Not listed
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
1 Day to 100 Years
Enrollment
6,618 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2015
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 8, 2025 · Synced Oct 26, 2026, 4:51 AM EDT
Completed No phase listed Observational

Study of Usher Syndromes, Type 1 and Type 2

NCT00001347
View directory record Official record
Conditions
Retinitis Pigmentosa
Interventions
Not listed
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
Not listed
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1993 – 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 26, 2026, 4:51 AM EDT
Completed No phase listed Observational

Rate of Progression in EYS Related Retinal Degeneration

NCT04127006
View directory record Official record
Conditions
Retinitis Pigmentosa, Eye Diseases
Interventions
Not listed
Lead sponsor
Jaeb Center for Health Research
Other
Eligibility
18 Years and older
Enrollment
103 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
13
States / cities
San Francisco, California • Denver, Colorado • Gainesville, Florida + 10 more
Source: ClinicalTrials.gov public record
Updated Aug 5, 2026 · Synced Oct 26, 2026, 4:51 AM EDT
Active, not recruiting Phase 3 Interventional Results available

Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis

NCT00999609
View directory record Official record
Conditions
Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis
Interventions
AAV2-hRPE65v2,voretigene neparvovec-rzyl
Biological
Lead sponsor
Spark Therapeutics, Inc.
Industry
Eligibility
3 Years and older
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2030
U.S. locations
2
States / cities
Iowa City, Iowa • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 23, 2025 · Synced Oct 26, 2026, 4:51 AM EDT
Not listed Not applicable Interventional

Stem Cell Ophthalmology Treatment Study

NCT01920867
View directory record Official record
Conditions
Retinal Disease, Macular Degeneration, Hereditary Retinal Dystrophy, Optic Nerve Disease, Glaucoma
Interventions
RB (Retrobulbar), ST (Subtenon), IV (Intravenous), IVIT (Intravitreal), IO (Intraocular)
Procedure
Lead sponsor
MD Stem Cells
Industry
Eligibility
18 Years and older
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2020
U.S. locations
1
States / cities
Westport, Connecticut
Source: ClinicalTrials.gov public record
Updated Oct 23, 2019 · Synced Oct 26, 2026, 4:51 AM EDT
Completed Phase 2 Interventional Results available

Retinal Imaging in CNTF -Releasing Encapsulated Cell Implant Treated Patients for Early-stage Retinitis Pigmentosa

NCT01530659
View directory record Official record
Conditions
Retinitis Pigmentosa, Usher Syndrome Type 2, Usher Syndrome Type 3
Interventions
NT-501, Sham
Drug · Procedure
Lead sponsor
Neurotech Pharmaceuticals
Industry
Eligibility
18 Years to 55 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2019
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Feb 14, 2023 · Synced Oct 26, 2026, 4:51 AM EDT
Recruiting Phase 1Phase 2 Interventional

Disulfiram for Treatment of Retinal Degeneration

NCT05626920
View directory record Official record
Conditions
Inherited Retinal Dystrophy Primarily Involving Sensory Retina
Interventions
Disulfiram 250 mg, Placebo
Drug
Lead sponsor
University of Washington
Other
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated May 5, 2026 · Synced Oct 26, 2026, 4:51 AM EDT
Completed Phase 1 Interventional

Effect of DHA Supplements on Macular Function in Patients With Stargardt Macular Dystrophy and Stargardt-like Macular Dystrophy

NCT00060749
View directory record Official record
Conditions
Macular Degeneration
Interventions
Docosahexaenoic Acid (DHA) Dietary Supplement
Drug
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
Not listed
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2007
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 26, 2026, 4:51 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

High Resolution Retinal Imaging

NCT01866371
View directory record Official record
Conditions
Stargardts, Retinitis Pigmentosa, Age-related Macular Degeneration, Choroideremia, Geographic Atrophy
Interventions
Retinal imaging
Procedure
Lead sponsor
University of Pennsylvania
Other
Eligibility
7 Years and older
Enrollment
600 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2031
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 5, 2026 · Synced Oct 26, 2026, 4:51 AM EDT