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Showing 1–24 of 44 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: ABCA4 Retinopathy Clear all
Recruiting Phase 1Phase 2 Interventional

Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety of a Single Intravitreal Injection of RTx-021 in Patients With Stargardt Disease

NCT07439887
View directory record Official record
Conditions
Stargardt Disease
Interventions
RTx-021
Genetic
Lead sponsor
Ray Therapeutics, Inc.
Industry
Eligibility
16 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
3
States / cities
Bakersfield, California • Pittsburgh, Pennsylvania • Bellaire, Texas
Source: ClinicalTrials.gov public record
Updated Apr 14, 2026 · Synced Sep 9, 2026, 9:06 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 30, 2024 · Synced Sep 9, 2026, 9:06 AM EDT
Completed Phase 1Phase 2 Interventional

Sub-retinal Transplantation of hESC Derived RPE(MA09-hRPE)Cells in Patients With Stargardt's Macular Dystrophy

NCT01345006
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
2011 – 2015
U.S. locations
3
States / cities
Los Angeles, California • Miami, Florida • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 30, 2024 · Synced Sep 9, 2026, 9:06 AM EDT
Completed Phase 2 Interventional Results available

Zimura Compared to Sham in Patients With Autosomal Recessive Stargardt Disease (STGD1)

NCT03364153
View directory record Official record
Conditions
Stargardt's Macular Dystrophy
Interventions
avacincaptad pegol, Sham
Drug
Lead sponsor
Astellas Pharma Global Development, Inc.
Industry
Eligibility
18 Years to 60 Years
Enrollment
121 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
17
States / cities
Phoenix, Arizona • Los Angeles, California • Gainesville, Florida + 14 more
Source: ClinicalTrials.gov public record
Updated Apr 14, 2026 · Synced Sep 9, 2026, 9:06 AM 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 2, 2018 · Synced Sep 9, 2026, 9:06 AM EDT
Enrolling by invitation 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 – 2026
U.S. locations
14
States / cities
Phoenix, Arizona • Los Angeles, California • Aurora, Colorado + 11 more
Source: ClinicalTrials.gov public record
Updated Apr 26, 2025 · Synced Sep 9, 2026, 9:06 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 18, 2026 · Synced Sep 9, 2026, 9:06 AM EDT
Recruiting Phase 1Phase 2 Interventional

A Phase 1/2, First-in-Human Dose Escalation/Expansion Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of a Subretinal Injection of SB-007 in Subjects With Stargardt Disease (STGD1)

NCT06942572
View directory record Official record
Conditions
Stargardt Disease, Stargardt Macular Degeneration, Stargardt Macular Dystrophy
Interventions
SB-007
Genetic
Lead sponsor
Splice Bio
Industry
Eligibility
12 Years to 65 Years
Enrollment
57 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
5
States / cities
Aurora, Colorado • Miami, Florida • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated Apr 28, 2025 · Synced Sep 9, 2026, 9:06 AM EDT
Completed Phase 3 Interventional

Phase 3, Randomized, Placebo-Controlled Study of Tinlarebant to Explore Safety and Efficacy in Adolescent Stargardt Disease

NCT05244304
View directory record Official record
Conditions
Stargardt Disease 1
Interventions
Tinlarebant, Placebo
Drug
Lead sponsor
Belite Bio, Inc
Industry
Eligibility
12 Years to 20 Years
Enrollment
104 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
3
States / cities
Palo Alto, California • Minneapolis, Minnesota • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Jul 22, 2026 · Synced Sep 9, 2026, 9:06 AM EDT
Active, not recruiting Phase 2 Interventional

Phase I/II Follow-up Study of SAR422459 in Patients With Stargardt's Macular Degeneration

NCT01736592
View directory record Official record
Conditions
Stargardt's Disease
Interventions
Long term follow up in all patients who received SAR422459 in previous study TDU13583
Drug
Lead sponsor
Sanofi
Industry
Eligibility
6 Years and older
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2033
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Aug 25, 2026 · Synced Sep 9, 2026, 9:06 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Phase 1 Pilot Study of 4-MP to Treat Stargardt Macular Dystrophy

NCT00346853
View directory record Official record
Conditions
Macular Dystrophy, Corneal
Interventions
4-Methylpyrazole, saline
Drug · Other
Lead sponsor
University of Utah
Other
Eligibility
18 Years to 65 Years
Enrollment
10 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2005 – 2006
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Aug 4, 2020 · Synced Sep 9, 2026, 9:06 AM EDT
Recruiting No phase listed Observational

An Observational Study in Subjects to Follow the Progression of Stargardt Disease Type 1 (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ABCA4 Gene

NCT06435000
View directory record Official record
Conditions
Stargardt, Stargardt's Disease, Stargardt Disease, STGD1
Interventions
Not listed
Lead sponsor
Splice Bio
Industry
Eligibility
12 Years to 65 Years
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
14
States / cities
San Diego, California • Aurora, Colorado • Gainesville, Florida + 10 more
Source: ClinicalTrials.gov public record
Updated Sep 18, 2025 · Synced Sep 9, 2026, 9:06 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Phase I/IIA Study of SAR422459 in Participants With Stargardt's Macular Degeneration

NCT01367444
View directory record Official record
Conditions
Stargardt's Disease
Interventions
SAR422459
Drug
Lead sponsor
Sanofi
Industry
Eligibility
6 Years and older
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2019
U.S. locations
4
States / cities
Miami, Florida • Iowa City, Iowa • Portland, Oregon + 1 more
Source: ClinicalTrials.gov public record
Updated Apr 13, 2022 · Synced Sep 9, 2026, 9:06 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Microcurrent Stimulation to Treat Macular Degeneration

NCT01790958
View directory record Official record
Conditions
Retinal Diseases, Stargardt's Disease, Retinitis Pigmentosa
Interventions
Not listed
Lead sponsor
Retina Institute of Hawaii
Other
Eligibility
50 Years and older
Enrollment
50 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012
U.S. locations
4
States / cities
Hilo, Hawaii • Honolulu, Hawaii • Kahului, Hawaii + 1 more
Source: ClinicalTrials.gov public record
Updated Feb 12, 2013 · Synced Sep 9, 2026, 9:06 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 28, 2026 · Synced Sep 9, 2026, 9:06 AM EDT
Active, not recruiting No phase listed Observational

Non-interventional Long Term Follow-up Study of Participants Previously Enrolled in the STARLIGHT Study

NCT06048185
View directory record Official record
Conditions
Stargardt Disease
Interventions
Gene Therapy Product-MCO-010
Biological
Lead sponsor
Nanoscope Therapeutics Inc.
Industry
Eligibility
16 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
2
States / cities
Miami, Florida • McAllen, Texas
Source: ClinicalTrials.gov public record
Updated Mar 23, 2025 · Synced Sep 9, 2026, 9:06 AM EDT
Recruiting Phase 3 Interventional

Study of ALK-001 on the Progression of Stargardt Disease

NCT07419334
View directory record Official record
Conditions
Stargardt Disease
Interventions
ALK-001, Placebo
Drug
Lead sponsor
Alkeus Pharmaceuticals, Inc.
Industry
Eligibility
8 Years to 45 Years
Enrollment
230 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
10
States / cities
Phoenix, Arizona • Aurora, Colorado • Gainesville, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Aug 25, 2026 · Synced Sep 9, 2026, 9:06 AM EDT
Recruiting No phase listed Observational

Prescreening Study to Identify Potential Stargardt Participants for ACDN-01 Clinical Trials (STARPATH)

NCT06445322
View directory record Official record
Conditions
Stargardt Disease, Stargardt Disease 1, Cone Rod Dystrophy, Juvenile Macular Degeneration
Interventions
Prescreening Assessments
Diagnostic Test
Lead sponsor
Ascidian Therapeutics, Inc
Industry
Eligibility
5 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
8
States / cities
San Francisco, California • Gainesville, Florida • Baltimore, Maryland + 5 more
Source: ClinicalTrials.gov public record
Updated Mar 10, 2026 · Synced Sep 9, 2026, 9:06 AM EDT
Active, not recruiting Phase 2Phase 3 Interventional

A Phase 2/3 Study to Evaluate the Efficacy and Safety of Tinlarebant in Subjects With Stargardt Disease

NCT06388083
View directory record Official record
Conditions
STGD1, Stargardt Disease 1
Interventions
Tinlarebant, Placebo
Drug
Lead sponsor
Belite Bio, Inc
Industry
Eligibility
12 Years to 20 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
14
States / cities
Phoenix, Arizona • La Jolla, California • San Francisco, California + 11 more
Source: ClinicalTrials.gov public record
Updated Mar 11, 2026 · Synced Sep 9, 2026, 9:06 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 1, 2017 · Synced Sep 9, 2026, 9:06 AM EDT
Recruiting Phase 1Phase 2 Interventional

A Study of AAVB-039 in Participants With Stargardt Disease (STGD1)

NCT07161544
View directory record Official record
Conditions
Stargardt Disease
Interventions
AAVB-039
Biological
Lead sponsor
AAVantgarde Bio Srl
Industry
Eligibility
8 Years to 55 Years
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
7
States / cities
Phoenix, Arizona • Los Angeles, California • Jacksonville, Florida + 4 more
Source: ClinicalTrials.gov public record
Updated Jul 29, 2026 · Synced Sep 9, 2026, 9:06 AM EDT
Completed Phase 2 Interventional

Study of STG-001 in Subjects With Stargardt Disease

NCT04489511
View directory record Official record
Conditions
Stargardt Disease-1
Interventions
STG-001
Drug
Lead sponsor
Stargazer Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 55 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
5
States / cities
Phoenix, Arizona • Gainesville, Florida • Portland, Oregon + 2 more
Source: ClinicalTrials.gov public record
Updated Apr 26, 2021 · Synced Sep 9, 2026, 9:06 AM EDT
Recruiting No phase listed Observational

A Study to Learn How Stargardt-type Eye Conditions Progress in Children and Adults

NCT07425574
View directory record Official record
Conditions
Stargardt Disease, Stargardt Macular Dystrophy, Stargardt-like Macular Dystrophy
Interventions
No Intervention
Other
Lead sponsor
Astellas Pharma Global Development, Inc.
Industry
Eligibility
6 Years and older
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
15
States / cities
Phoenix, Arizona • Palo Alto, California • Sacramento, California + 12 more
Source: ClinicalTrials.gov public record
Updated Aug 24, 2026 · Synced Sep 9, 2026, 9:06 AM EDT
Recruiting No phase listed Observational

Genotype-Phenotype Study of Patients With Plaquenil -Induced Retinal Toxicity, With Evaluation of the ABCA4 Gene

NCT01145196
View directory record Official record
Conditions
Genotype, Retinal Disease
Interventions
Not listed
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
18 Years to 120 Years
Enrollment
320 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2010
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 31, 2026 · Synced Sep 9, 2026, 9:06 AM EDT