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ClinicalTrials.gov public records Last synced Oct 7, 2026, 5:11 AM EDT

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Showing 97–120 of 141 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Pigmentary Retinopathy Clear all
Active, not recruiting Phase 1 Interventional

Clinical Trial of Autologous Intravitreal Bone-marrow CD34+ Stem Cells for Retinopathy

NCT01736059
View directory record Official record
Conditions
Non-exudative Age-related Macular Degeneration, Diabetic Retinopathy, Retina Vein Occlusion, Retinitis Pigmentosa, Hereditary Macular Degeneration
Interventions
CD34+ bone marrow stem cells intravitreal
Drug
Lead sponsor
University of California, Davis
Other
Eligibility
18 Years to 100 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2026
U.S. locations
1
States / cities
Sacramento, California
Source: ClinicalTrials.gov public record
Updated Jan 29, 2026 · Synced Oct 7, 2026, 5:11 AM EDT
Recruiting Phase 1 Interventional

The Effects of Disulfiram (Antabuse®) on Visual Acuity in Patients With Retinal Degeneration

NCT06319872
View directory record Official record
Conditions
Alcohol Use Disorder, Retinal Dystrophies, Age-Related Macular Degeneration, Retinitis Pigmentosa, Stargardt Disease
Interventions
Oral disulfiram
Drug
Lead sponsor
University of Rochester
Other
Eligibility
18 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Sep 16, 2026 · Synced Oct 7, 2026, 5:11 AM 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 7, 2026, 5:11 AM EDT
Recruiting Phase 1Phase 2 Interventional

A Study to Investigate the Safety of DSP-3077 After a Unilateral Eye Injection in Male and Female Participants 18 Years of Age or Older With Retinitis Pigmentosa

NCT06891885
View directory record Official record
Conditions
Retinitis Pigmentosa
Interventions
DSP-3077 Retinal Sheet Cohort 1, DSP-3077 Retinal Sheet Cohort 2, DSP-3077 Retinal Sheet Cohort 3
Drug
Lead sponsor
Sumitomo Pharma America, Inc.
Industry
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
2
States / cities
Boston, Massachusetts • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 17, 2026 · Synced Oct 7, 2026, 5:11 AM EDT
Completed No phase listed Observational

A Patient Registry Study for Patients Treated With Voretigene Neparvovec in US

NCT03597399
View directory record Official record
Conditions
Confirmed Biallelic RPE65 Mutation-associated Retinal Dystrophy
Interventions
AAV2-hRPE65v2,voretigene neparvovec-rzyl
Biological
Lead sponsor
Spark Therapeutics, Inc.
Industry
Eligibility
12 Months and older
Enrollment
87 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
10
States / cities
Los Angeles, California • Miami, Florida • Iowa City, Iowa + 6 more
Source: ClinicalTrials.gov public record
Updated Sep 22, 2025 · Synced Oct 7, 2026, 5:11 AM 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 7, 2026, 5:11 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Retinal Imaging in Patients With Inherited Retinal Degenerations

NCT00254605
View directory record Official record
Conditions
Retinitis Pigmentosa
Interventions
Not listed
Lead sponsor
University of California, San Francisco
Other
Eligibility
13 Years and older
Enrollment
400 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2005 – 2028
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Jun 5, 2026 · Synced Oct 7, 2026, 5:11 AM 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 7, 2026, 5:11 AM EDT
Completed Phase 3 Interventional

Long-term Safety and Efficacy Follow-up of BIIB111 for the Treatment of Choroideremia and BIIB112 for the Treatment of X-Linked Retinitis Pigmentosa

NCT03584165
View directory record Official record
Conditions
Choroideremia, X-Linked Retinitis Pigmentosa
Interventions
BIIB111, BIIB112
Genetic
Lead sponsor
NightstaRx Ltd, a Biogen Company
Other
Eligibility
18 Years and older · Male only
Enrollment
277 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
10
States / cities
Los Angeles, California • Gainesville, Florida • Miami, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Jul 6, 2026 · Synced Oct 7, 2026, 5:11 AM EDT
Completed Phase 1 Interventional

Acupuncture for the Treatment of Vision Loss Due to Retinitis Pigmentosa

NCT01604356
View directory record Official record
Conditions
Retinitis Pigmentosa
Interventions
Electro-acupuncture
Procedure
Lead sponsor
Johns Hopkins University
Other
Eligibility
10 Years and older
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2014 · Synced Oct 7, 2026, 5:11 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 7, 2026, 5:11 AM EDT
Completed Phase 3 Interventional Results available

Clinical Trial of Lutein for Patients With Retinitis Pigmentosa Receiving Vitamin A

NCT00346333
View directory record Official record
Conditions
Retinitis Pigmentosa
Interventions
Lutein, Cornstarch control
Drug · Dietary Supplement
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
18 Years to 60 Years
Enrollment
240 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2008
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jan 31, 2014 · Synced Oct 7, 2026, 5:11 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 7, 2026, 5:11 AM EDT
Active, not recruiting Phase 3 Interventional

A Phase 3 Study Of OCU400 Gene Therapy for the Treatment Of Retinitis Pigmentosa

NCT06388200
View directory record Official record
Conditions
Retinitis Pigmentosa
Interventions
Sub-Retinal Administration of OCU400-301
Genetic
Lead sponsor
Ocugen
Industry
Eligibility
3 Years and older
Enrollment
140 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
12
States / cities
Phoenix, Arizona • La Jolla, California • Los Angeles, California + 9 more
Source: ClinicalTrials.gov public record
Updated Jul 31, 2026 · Synced Oct 7, 2026, 5:11 AM EDT
Completed No phase listed Observational

Natural History Study in Inherited Retinal Disease Subjects Caused by Mutations in RPE65 or LRAT

NCT02575430
View directory record Official record
Conditions
Leber Congenital Amaurosis (LCA), Retinitis Pigmentosa (RP)
Interventions
No treatment: retrospective chart review
Other
Lead sponsor
QLT Inc.
Industry
Eligibility
8 Years and older
Enrollment
59 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
2
States / cities
Baltimore, Maryland • Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Apr 29, 2016 · Synced Oct 7, 2026, 5:11 AM EDT
Recruiting No phase listed Observational

A Natural History Study Seeks to Understand the Clinical, Genomic, Pharmacological, Laboratory, and Dietary Determinates of Pyrimidine and Purine Metabolism Disorders

NCT06092346
View directory record Official record
Conditions
AMPD3, OMIM*102772, AMP Deaminase Deficiency, AK1, OMIM *103000, Adenylate Kinase Deficiency, AMPD1, OMIM *102770, Myopathy Due to Myoadenylate Deaminase Deficiency, TPMT, OMIM *187680, Thoipurines, Poor Metabolism of, IMPDH1, OMIM *146690, Retinitis Pigmentosa Type 10, Leber Congenital Amauriosis Type 11, APRT, OMIM *102600, Adenine Phosphoribosyltransferase Deficiency, HPRT1, OMIM *308000 Lesch-Nyhan Disease, XDH, OMIM *607633, Xanthinuria Type 1, SLC2A9, OMIM *606142 Hypouricemia, SLC22A12, OMIM *607096 Hypouricemia, PRPS1 Def, OMIM *311850, Arts Syndrome; Charcot-Marie-Tooth Disease, PRPS1 SA, OMIM *311850 Gout, PRPS-related Phosphoribosylpyrophosphate Synthetase Superactivity, AMPD2, OMIM *102771, Spastic Paraplegia 63; Pontocerebellar Hypoplasia, ITPA, OMIM *147520, Inosine Triphosphatase Deficiency; Developmental and Epileptic Encephalopathy 35, ADSL, OMIM *608222, Adenylosuccinate Lyase Deficiency, PNP, OMIM *164050, Nucleoside Phosphorylase Deficiency, ADA2, OMIM *607575,Sneddon Syndrome; VAIHS, CAD, *1140120, Developmental and Epileptic Encephalopathy, UPB1, OMIM *606673, Beta-ureidopropionase Deficiency, DPYS, OMIM *613326, Dihydropyrimidinase Deficiency, DPYD, OMIM *274270, Dihydropyrimidine Dehydrogenase Deficiency, DHODH, OMIM *126064, Miller Syndrome (Postaxial Acrofacial Dysostosis), UMPS, OMIM *613891, Orotic Aciduria, NT5C3A<TAB>, OMIM *606224, Anemia, Hemolytic, Due to UMPH1 Deficiency, UNG, OMIM *191525, Hyper-IgM Syndrome 5, AICDA, OMIM *605257, Immunodeficiency With Hyper-IgM, Type 2; HIGM2, Purine-Pyrimidine Metabolism, Metabolic Disease
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
1 Month to 100 Years
Enrollment
999 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2099
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 19, 2026 · Synced Oct 7, 2026, 5:11 AM EDT
Completed Phase 1 Interventional

Evaluation of Safety of Ciliary Neurotrophic Factor Implants in the Eye

NCT00063765
View directory record Official record
Conditions
Retinitis Pigmentosa
Interventions
Ciliary Neurotrophic Factor Implant NT-501
Drug
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
Not listed
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2006
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 7, 2026, 5:11 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Study of Oral Minocycline in Treating Bilateral Cystoid Macular Edema Associated With Retinitis Pigmentosa

NCT02140164
View directory record Official record
Conditions
Retinitis Pigmentosa
Interventions
Minocycline
Drug
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
12 Years and older
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 17, 2024 · Synced Oct 7, 2026, 5:11 AM EDT
Recruiting No phase listed Observational

Study of BEST1 Vitelliform Macular Dystrophy

NCT05809635
View directory record Official record
Conditions
Best Vitelliform Macular Dystrophy, Retinitis Pigmentosa
Interventions
Natural History Study
Other
Lead sponsor
Columbia University
Other
Eligibility
Not listed
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 30, 2025 · Synced Oct 7, 2026, 5:11 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 7, 2026, 5:11 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 7, 2026, 5:11 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 7, 2026, 5:11 AM EDT
Enrolling by invitation Phase 2 Interventional

A Study Comparing Two Doses of AGTC-501 in Male Participants With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (DAWN)

NCT06275620
View directory record Official record
Conditions
X-Linked Retinitis Pigmentosa
Interventions
AGTC-501 (high dose and standard corticosteroid regimen), AGTC-501 (low dose and standard corticosteroid regimen), AGTC-501 (high dose and modified corticosteroid regimen)
Biological
Lead sponsor
Beacon Therapeutics
Industry
Eligibility
12 Years and older · Male only
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
7
States / cities
Jacksonville, Florida • Miami, Florida • Boston, Massachusetts + 4 more
Source: ClinicalTrials.gov public record
Updated Oct 30, 2024 · Synced Oct 7, 2026, 5:11 AM EDT
Completed Phase 3 Interventional Results available

Phase 3 Crossover Trial of Two Formulations of Setmelanotide in Participants With Specific Gene Defects in the MC4R Pathway

NCT05194124
View directory record Official record
Conditions
Bardet-Biedl Syndrome, POMC Deficiency
Interventions
Setmelanotide 2 mg, Setmelanotide 2.5 mg, Setmelanotide 3 mg, Setmelanotide 20 mg, Setmelanotide 25 mg, Setmelanotide 30 mg
Drug
Lead sponsor
Rhythm Pharmaceuticals, Inc.
Industry
Eligibility
6 Years and older
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
2
States / cities
Scottsdale, Arizona • Marshfield, Wisconsin
Source: ClinicalTrials.gov public record
Updated Nov 26, 2024 · Synced Oct 7, 2026, 5:11 AM EDT