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ClinicalTrials.gov public records Last synced Sep 12, 2026, 3:25 AM EDT

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Showing 1–17 of 17 matching trials from the live ClinicalTrials.gov search.
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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 – 2026
U.S. locations
8
States / cities
Phoenix, Arizona • Los Angeles, California • Aurora, Colorado + 5 more
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
Not listed Not applicable Interventional

Stem Cell Ophthalmology Treatment Study

NCT01920867
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
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
Conditions
Retinitis Pigmentosa, Choroideremia, Stargardt Macular Dystrophy, Stargardt Disease, Geographic Atrophy from Age-related Macular Degeneration, X-lined Retinoschisis, Retinal Dystrophies
Interventions
Not listed
Lead sponsor
Ray Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Irvine, California
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
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
Conditions
Geographic Atrophy, Stargardt Disease, RPE-mediated Maculopathy, Age Related Macular Degeneration
Interventions
Injection
Biological
Lead sponsor
Cellio Therapeutics Inc
Industry
Eligibility
18 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
3
States / cities
Beverly Hills, California • Palo Alto, California • Sacramento, California
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
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
Conditions
Stargardt Disease
Interventions
Not listed
Lead sponsor
AAVantgarde Bio Srl
Industry
Eligibility
8 Years to 50 Years
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
1
States / cities
Dallas, Texas
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
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
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
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
Conditions
Stargardt Disease, Diabetic Retinopathy, Macular Degeneration (Age Related)
Interventions
Anterior Chamber (AC) Tap
Other
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years to 100 Years
Enrollment
77 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
1
States / cities
Baltimore, Maryland
Conditions
Stargardt Disease, Stargardt Macular Dystrophy, Stargardt-like Macular Dystrophy
Interventions
ASP2020
Drug
Lead sponsor
Astellas Institute for Regenerative Medicine
Industry
Eligibility
6 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
1
States / cities
Phoenix, Arizona