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ClinicalTrials.gov public records Last synced Sep 26, 2026, 4:16 PM EDT

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Showing 25–44 of 20 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: ABCA4 Retinopathy Clear all
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 26, 2026, 4:16 PM EDT
Recruiting Phase 1Phase 2 Interventional

Study to Evaluate ACDN-01 in ABCA4-related Stargardt Retinopathy (STELLAR)

NCT06467344
View directory record Official record
Conditions
Stargardt Disease, Cone Rod Dystrophy, Juvenile Macular Degeneration, Stargardt Disease 1
Interventions
ACDN-01
Drug
Lead sponsor
Ascidian Therapeutics, Inc
Industry
Eligibility
12 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
10
States / cities
San Francisco, California • Gainesville, Florida • Miami, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Dec 1, 2025 · Synced Sep 26, 2026, 4:16 PM 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 22, 2019 · Synced Sep 26, 2026, 4:16 PM 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 26, 2026, 4:16 PM 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 26, 2026, 4:16 PM EDT
Active, not recruiting No phase listed Observational

Natural History of Eye Diseases Related to ABCA4 Mutations

NCT01736293
View directory record Official record
Conditions
Retinal Degeneration, ABCA4-Related Retinopathies
Interventions
Not listed
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
12 Years to 100 Years
Enrollment
68 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2012
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 31, 2026 · Synced Sep 26, 2026, 4:16 PM 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 20, 2026 · Synced Sep 26, 2026, 4:16 PM 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 15, 2026 · Synced Sep 26, 2026, 4:16 PM 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 26, 2026, 4:16 PM 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 26, 2026, 4:16 PM 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 26, 2026, 4:16 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Oral Metformin for Treatment of ABCA4 Retinopathy

NCT04545736
View directory record Official record
Conditions
ABCA4 Retinopathy, Stargardt Disease, Retinal Dystrophy, Retinal Degeneration
Interventions
Metformin hydrochloride
Drug
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
12 Years to 100 Years
Enrollment
55 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2029
U.S. locations
2
States / cities
Bethesda, Maryland • Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Sep 23, 2026 · Synced Sep 26, 2026, 4:16 PM EDT
Terminated No phase listed Observational

Novel Quantification Methods for Fluorescence to Detect Progression in Stargardt Disease

NCT01676766
View directory record Official record
Conditions
Stargardt Disease
Interventions
Not listed
Lead sponsor
University of Michigan
Other
Eligibility
5 Years to 18 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Nov 7, 2016 · Synced Sep 26, 2026, 4:16 PM 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 26, 2026, 4:16 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 2, 2018 · Synced Sep 26, 2026, 4:16 PM 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 20, 2026 · Synced Sep 26, 2026, 4:16 PM EDT
Active, not recruiting Phase 2Phase 3 Interventional

A Phase 2/3 Trial to Assess the Efficacy and Safety of OCU410ST for Stargardt Disease

NCT05956626
View directory record Official record
Conditions
Stargardt Disease
Interventions
OCU410ST
Drug
Lead sponsor
Ocugen
Industry
Eligibility
5 Years and older
Enrollment
51 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
14
States / cities
Phoenix, Arizona • Sacramento, California • Gainesville, Florida + 11 more
Source: ClinicalTrials.gov public record
Updated Jul 30, 2026 · Synced Sep 26, 2026, 4:16 PM EDT
Completed Phase 2 Interventional

Safety and Effects of a Single Intravitreal Injection of vMCO-010 Optogenetic Therapy in Subjects With Stargardt Disease

NCT05417126
View directory record Official record
Conditions
Stargardt Disease
Interventions
Gene Therapy-vMCO-010
Biological
Lead sponsor
Nanoscope Therapeutics Inc.
Industry
Eligibility
16 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
2
States / cities
Miami, Florida • McAllen, Texas
Source: ClinicalTrials.gov public record
Updated Nov 8, 2023 · Synced Sep 26, 2026, 4:16 PM 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 26, 2026, 4:16 PM EDT
Completed Phase 3 Interventional Results available

Safety and Efficacy of Emixustat in Stargardt Disease

NCT03772665
View directory record Official record
Conditions
Stargardt Disease
Interventions
Emixustat, Placebo
Drug
Lead sponsor
Kubota Vision Inc.
Industry
Eligibility
16 Years and older
Enrollment
194 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
11
States / cities
Beverly Hills, California • San Francisco, California • Atlanta, Georgia + 8 more
Source: ClinicalTrials.gov public record
Updated May 29, 2024 · Synced Sep 26, 2026, 4:16 PM EDT