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ClinicalTrials.gov public records Last synced Oct 11, 2026, 11:38 PM EDT

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Showing 145–168 of 170 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Retinal Dystrophies Clear all
Completed No phase listed Observational

Stem Cell Models of Best Disease and Other Retinal Degenerative Diseases.

NCT02162953
View directory record Official record
Conditions
Retinal Disease, Bestrophinopathy, Best Vitelliform Macular Dystrophy, Adult Onset Vitelliform Macular Dystrophy, Autosomal Dominant Vitreoretinalchoroidopathy
Interventions
Not listed
Lead sponsor
Mayo Clinic
Other
Eligibility
5 Years and older
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2022
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Jan 9, 2023 · Synced Oct 11, 2026, 11:38 PM 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Withdrawn Not applicable Interventional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 11, 2026, 11:38 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 16, 2026 · Synced Oct 11, 2026, 11:38 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

PIONEER: Intraoperative and Perioperative OCT Study

NCT02423161
View directory record Official record
Conditions
Retinal Detachment, Epiretinal Membrane, Macular Hole, Fuchs Endothelial Dystrophy
Interventions
Not listed
Lead sponsor
The Cleveland Clinic
Other
Eligibility
18 Years and older
Enrollment
750 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2017
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Aug 20, 2024 · Synced Oct 11, 2026, 11:38 PM EDT
Terminated No phase listed Observational

The Natural History of Mucolipidosis Type IV

NCT01067742
View directory record Official record
Conditions
Mucolipidosis Type IV
Interventions
Not listed
Lead sponsor
Baylor Research Institute
Other
Eligibility
1 Year to 64 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2021
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jan 26, 2026 · Synced Oct 11, 2026, 11:38 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Promising ROd-cone DYstrophy Gene therapY

NCT05748873
View directory record Official record
Conditions
Retinitis Pigmentosa
Interventions
SPVN06
Drug
Lead sponsor
SparingVision
Industry
Eligibility
18 Years and older
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2031
U.S. locations
5
States / cities
Miami, Florida • Boston, Massachusetts • Portland, Oregon + 2 more
Source: ClinicalTrials.gov public record
Updated May 15, 2026 · Synced Oct 11, 2026, 11:38 PM EDT
Completed Phase 2 Interventional Results available

Sildenafil for Treatment of Choroidal Ischemia

NCT04356716
View directory record Official record
Conditions
Choroidal Ischemia, Vitelliform Macular Dystrophy, Age-related Macular Degeneration, Central Serous Retinopathy, Retinitis Pigmentosa
Interventions
Sildenafil, Standard of Care Sildenafil, Ocular Coherence Tomography-Angiography (OCT-A), Visual Acuity (VA)
Drug · Other · Diagnostic Test
Lead sponsor
Columbia University
Other
Eligibility
18 Years and older
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2020
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 4, 2025 · Synced Oct 11, 2026, 11:38 PM EDT
Completed No phase listed Observational Accepts healthy volunteers Results available

Rod Sensitivity in Age-related Macular Degeneration (AMD) and Retinitis Pigmentosa (RP)

NCT03322930
View directory record Official record
Conditions
Retinitis Pigmentosa, AMD
Interventions
Rod sensitivity
Diagnostic Test
Lead sponsor
Retina Foundation of the Southwest
Other
Eligibility
10 Years to 85 Years
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2019
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Dec 23, 2019 · Synced Oct 11, 2026, 11:38 PM EDT
Completed No phase listed Observational

Natural History of the Progression of Choroideremia Study

NCT03359551
View directory record Official record
Conditions
Choroideremia
Interventions
Not listed
Lead sponsor
NightstaRx Ltd, a Biogen Company
Other
Eligibility
18 Years and older · Male only
Enrollment
319 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2020
U.S. locations
7
States / cities
Los Angeles, California • Miami, Florida • Baltimore, Maryland + 4 more
Source: ClinicalTrials.gov public record
Updated Feb 10, 2023 · Synced Oct 11, 2026, 11:38 PM EDT
Completed Not applicable Interventional

Argus II/ORCAM Device Study

NCT03248388
View directory record Official record
Conditions
Retinitis Pigmentosa
Interventions
Argus II Eyeglasses, ORCAM
Device
Lead sponsor
Mayo Clinic
Other
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Jan 13, 2022 · Synced Oct 11, 2026, 11:38 PM EDT
Completed No phase listed Observational

The Natural History of the Progression of Atrophy Secondary to Stargardt Disease Type 4: PROM1-Related Macular Dystrophy

NCT02410122
View directory record Official record
Conditions
Stargardt Disease
Interventions
Not listed
Lead sponsor
Johns Hopkins University
Other
Eligibility
6 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
2
States / cities
Baltimore, Maryland • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Aug 3, 2018 · Synced Oct 11, 2026, 11:38 PM 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 11, 2026, 11:38 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 19, 2026 · Synced Oct 11, 2026, 11:38 PM 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 11, 2026, 11:38 PM EDT
Completed No phase listed Observational

Genomic Profiling of Mitochondrial Disease - Imaging Analysis for Precise Mitochondrial Medicine

NCT05012358
View directory record Official record
Conditions
Mitochondrial Myopathies, Mitochondrial DNA Mutation, Mitochondrial Diseases, Chronic Progressive External Ophthalmoplegia With Myopathy, Kearns-Sayre Syndrome
Interventions
Not listed
Lead sponsor
Mayo Clinic
Other
Eligibility
15 Years to 80 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 12, 2024 · Synced Oct 11, 2026, 11:38 PM EDT
Completed Phase 2 Interventional Results available

DHA and X-Linked Retinitis Pigmentosa

NCT00100230
View directory record Official record
Conditions
Retinitis Pigmentosa, X-linked Genetic Diseases
Interventions
docosahexaenoic acid OR corn/soy oil placebo
Drug
Lead sponsor
Retina Foundation of the Southwest
Other
Eligibility
7 Years to 32 Years · Male only
Enrollment
78 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2014
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Mar 17, 2015 · Synced Oct 11, 2026, 11:38 PM EDT
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 11, 2026, 11:38 PM EDT
Not listed Phase 2 Interventional

Cholesterol and Antioxidant Treatment in Patients With Smith-Lemli-Opitz Syndrome (SLOS)

NCT01773278
View directory record Official record
Conditions
Smith-Lemli-Opitz Syndrome, Cone-Rod Dystrophy, Hearing Loss
Interventions
Antioxidants, Cholesterol
Drug
Lead sponsor
University of Colorado, Denver
Other
Eligibility
Up to 65 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2025
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated May 8, 2024 · Synced Oct 11, 2026, 11:38 PM 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 11, 2026, 11:38 PM EDT
Withdrawn Not applicable Interventional

RETINA IMPLANT Alpha AMS in Blind Patients With Retinitis Pigmentosa

NCT03629899
View directory record Official record
Conditions
Retinitis Pigmentosa
Interventions
RETINA IMPLANT Alpha AMS
Device
Lead sponsor
Wills Eye
Other
Eligibility
50 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 10, 2019 · Synced Oct 11, 2026, 11:38 PM 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 11, 2026, 11:38 PM EDT
Suspended No phase listed Observational

CNGB1 and Allied Disorders

NCT04639635
View directory record Official record
Conditions
Retinitis Pigmentosa Associated With CNGB1 Mutations
Interventions
No intervention, this is a natural history progression study
Other
Lead sponsor
Columbia University
Other
Eligibility
Not listed
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2027
U.S. locations
2
States / cities
New York, New York • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 13, 2026 · Synced Oct 11, 2026, 11:38 PM 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 11, 2026, 11:38 PM EDT
Completed Phase 1 Interventional

Investigating the Effect of Vitamin A Supplementation on Retinitis Pigmentosa

NCT00065455
View directory record Official record
Conditions
Retinitis Pigmentosa
Interventions
Vitamin A
Drug
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
18 Years and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2009
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 11, 2026, 11:38 PM EDT