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ClinicalTrials.gov public records Last synced Oct 21, 2026, 1:03 AM EDT

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Showing 97–120 of 164 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Hereditary Retinal Dystrophy Clear all
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 21, 2026, 1:03 AM EDT
Not listed Phase 1Phase 2 Interventional

Single Ascending Dose Study in Participants With LCA10

NCT03872479
View directory record Official record
Conditions
Leber Congenital Amaurosis 10, Inherited Retinal Dystrophies, Eye Diseases, Hereditary, Retinal Disease, Retinal Degeneration, Vision Disorders, Eye Disorders Congenital
Interventions
EDIT-101
Drug
Lead sponsor
Editas Medicine, Inc.
Industry
Eligibility
3 Years and older
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
5
States / cities
Miami, Florida • Boston, Massachusetts • Ann Arbor, Michigan + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 5, 2022 · Synced Oct 21, 2026, 1:03 AM EDT
Terminated No phase listed Observational

New Enrollment Post-Approval Study of the Argus® II Retinal Prosthesis System

NCT01860092
View directory record Official record
Conditions
Retinitis Pigmentosa
Interventions
Not listed
Lead sponsor
Second Sight Medical Products
Industry
Eligibility
25 Years and older
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2020
U.S. locations
17
States / cities
Los Angeles, California • Denver, Colorado • Miami, Florida + 13 more
Source: ClinicalTrials.gov public record
Updated Dec 23, 2021 · Synced Oct 21, 2026, 1:03 AM EDT
Terminated No phase listed Observational

Observational Natural History Study of Autosomal Dominant Retinitis Pigmentosa (adRP)

NCT02926092
View directory record Official record
Conditions
Retinitis Pigmentosa
Interventions
Observation
Diagnostic Test
Lead sponsor
Shire
Industry
Eligibility
Not listed
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Mar 17, 2021 · Synced Oct 21, 2026, 1:03 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional Results available

Study of Subretinally Injected ATSN-101 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D

NCT03920007
View directory record Official record
Conditions
Leber Congenital Amaurosis, LCA, LCA1
Interventions
ATSN-101, ATSN-101 Diluent Solution, Prednisone, Triamcinalone Acetonide, 1% Prednisolone, Trimethoprim/polymyxin B
Drug
Lead sponsor
Atsena Therapeutics Inc.
Industry
Eligibility
6 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2027
U.S. locations
2
States / cities
Portland, Oregon • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 3, 2026 · Synced Oct 21, 2026, 1:03 AM EDT
Recruiting No phase listed Observational

Development and Evaluation of Functional Visual Field and Navigation Endpoints in Moderate to Profound Inherited Retinal Disease (DEFINE-IRD)

NCT07502664
View directory record Official record
Conditions
Retinitis Pigmentosa, Stargardt Macular Dystrophy, Stargardt Disease, Geographic Atrophy From Age-related Macular Degeneration, X-linked Retinoschisis, Retinal Dystrophies
Interventions
Not listed
Lead sponsor
Ray Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Irvine, California
Source: ClinicalTrials.gov public record
Updated Mar 31, 2026 · Synced Oct 21, 2026, 1:03 AM 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 21, 2026, 1:03 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 21, 2026, 1:03 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 21, 2026, 1:03 AM EDT
Completed Phase 2 Interventional

Safety Study in Retinal Transplantation for Retinitis Pigmentosa.

NCT00345917
View directory record Official record
Conditions
Retinitis Pigmentosa.
Interventions
Retinal transplantation instrument, Fetal tissue.
Device
Lead sponsor
Radtke, Norman D., M.D.
Individual
Eligibility
21 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2012
U.S. locations
1
States / cities
Louisville, Kentucky
Source: ClinicalTrials.gov public record
Updated Apr 11, 2012 · Synced Oct 21, 2026, 1:03 AM EDT
Completed Not applicable Interventional

Exercise and RP (AVAMC and Emory)

NCT03381235
View directory record Official record
Conditions
Retinitis Pigmentosa
Interventions
Spin exercise
Other
Lead sponsor
Emory University
Other
Eligibility
18 Years to 60 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Dec 26, 2018 · Synced Oct 21, 2026, 1:03 AM EDT
Active, not recruiting Not applicable Interventional

Electro-acupuncture and Transcorneal Electrical Stimulation (TES) for Retinitis Pigmentosa

NCT02086890
View directory record Official record
Conditions
Retinitis Pigmentosa
Interventions
Electro-acupuncture, Laser Acupuncture, Transcorneal Electrical Stimulation, Sham Electro-acupuncture, Sham laser acupuncture, Sham transcorneal electrical stimulation
Device
Lead sponsor
Nova Southeastern University
Other
Eligibility
18 Years and older
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2027
U.S. locations
1
States / cities
Fort Lauderdale, Florida
Source: ClinicalTrials.gov public record
Updated Feb 27, 2026 · Synced Oct 21, 2026, 1:03 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Study of SAR421869 in Participants With Retinitis Pigmentosa Associated With Usher Syndrome Type 1B

NCT01505062
View directory record Official record
Conditions
Usher Syndrome, Retinitis Pigmentosa
Interventions
SAR421869
Drug
Lead sponsor
Sanofi
Industry
Eligibility
6 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2019
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Apr 28, 2022 · Synced Oct 21, 2026, 1:03 AM 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 21, 2026, 1:03 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Familial Mediterranean Fever and Related Disorders: Genetics and Disease Characteristics

NCT00001373
View directory record Official record
Conditions
Familial Mediterranean Fever (FMF), Autoinflammation, Periodic Fever, Fever, Genetic Diseases, ROSAH, ALPK1
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
2 Months to 115 Years
Enrollment
5,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 1994
U.S. locations
5
States / cities
Washington D.C., District of Columbia • Baltimore, Maryland • Bethesda, Maryland + 1 more
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 21, 2026, 1:03 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

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

NCT05203939
View directory record Official record
Conditions
Retinitis Pigmentosa, Leber Congenital Amaurosis
Interventions
OCU400 Low Dose, OCU400 Med Dose, OCU400 High Dose, OCU400 Second Eye Dosing
Drug
Lead sponsor
Ocugen
Industry
Eligibility
6 Years and older
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
7
States / cities
Phoenix, Arizona • La Jolla, California • Miami, Florida + 4 more
Source: ClinicalTrials.gov public record
Updated Jul 31, 2026 · Synced Oct 21, 2026, 1:03 AM EDT
Completed No phase listed Observational

Long Term Follow Up of Sub-retinal Transplantation of hESC Derived RPE Cells in Stargardt Macular Dystrophy Patients

NCT02445612
View directory record Official record
Conditions
Stargardt's Macular Dystrophy
Interventions
MA09-hRPE
Biological
Lead sponsor
Astellas Institute for Regenerative Medicine
Industry
Eligibility
18 Years and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2019
U.S. locations
3
States / cities
Los Angeles, California • Miami, Florida • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 31, 2024 · Synced Oct 21, 2026, 1:03 AM EDT
Recruiting Phase 1Phase 2 Interventional

BS01 in Patients With Retinitis Pigmentosa

NCT04278131
View directory record Official record
Conditions
Retinitis Pigmentosa
Interventions
BS01
Drug
Lead sponsor
Bionic Sight LLC
Industry
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2029
U.S. locations
1
States / cities
Teaneck, New Jersey
Source: ClinicalTrials.gov public record
Updated Jan 23, 2026 · Synced Oct 21, 2026, 1:03 AM EDT
Terminated Phase 1Phase 2 Interventional

Dose Escalation Study to Evaluate the Safety/Tolerability and Efficacy of EA-2353 in Subjects With Retinitis Pigmentosa

NCT05392751
View directory record Official record
Conditions
Retinitis Pigmentosa, Retinitis Pigmentosa Syndrome
Interventions
EA-2353
Drug
Lead sponsor
Endogena Therapeutics, Inc
Industry
Eligibility
18 Years and older
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
5
States / cities
Miami, Florida • Ann Arbor, Michigan • Portland, Oregon + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 15, 2024 · Synced Oct 21, 2026, 1:03 AM EDT
Completed Phase 1 Interventional

Repeated Treatments of QLT091001 in Subjects With Leber Congenital Amaurosis or Retinitis Pigmentosa (Extension of Study RET IRD 01)

NCT01521793
View directory record Official record
Conditions
LCA (Leber Congenital Amaurosis), RP (Retinitis Pigmentosa)
Interventions
QLT091001
Drug
Lead sponsor
QLT Inc.
Industry
Eligibility
5 Years to 65 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
2
States / cities
Chicago, Illinois • Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 28, 2014 · Synced Oct 21, 2026, 1:03 AM EDT
Active, not recruiting Phase 1 Interventional

FIGHT-RP 1 Extension Study

NCT03999021
View directory record Official record
Conditions
Retinitis Pigmentosa
Interventions
NAC effervescent tablets
Drug
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2030
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 7, 2026 · Synced Oct 21, 2026, 1:03 AM 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 21, 2026, 1:03 AM EDT
Completed Phase 1 Interventional

Safety/Proof of Concept Study of Oral QLT091001 in Subjects With Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT) Mutations

NCT01014052
View directory record Official record
Conditions
LCA (Leber Congenital Amaurosis), RP (Retinitis Pigmentosa)
Interventions
QLT091001
Drug
Lead sponsor
QLT Inc.
Industry
Eligibility
5 Years to 65 Years
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
3
States / cities
Chicago, Illinois • Baltimore, Maryland • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 14, 2013 · Synced Oct 21, 2026, 1:03 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Cell Collection to Study Eye Diseases

NCT01432847
View directory record Official record
Conditions
Retinal Disease, AMD, Retinal Degeneration, Retinitis Pigmentosa
Interventions
Not listed
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
1 Day to 120 Years
Enrollment
930 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2011
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 22, 2026 · Synced Oct 21, 2026, 1:03 AM EDT