Condition

Stargardt Disease clinical trials and research

• 17Clinical trials
• 3NIH research projects
• 8Countries

Newest Stargardt Disease clinical trials

All recruiting trials (17)

• ASP2020 eye injection safety in Stargardt-type macular dystrophy

  A small study will test whether a single ASP2020 eye injection is safe and may help people with Stargardt-type macular dystrophy.

  Recruiting at 4 sites Phase 1 Astellas Pharma Inc · Phoenix, Arizona and 3 other locations · NCT07734064

• C.001 for retinal degeneration: a first-in-human dose-escalation safety test

  A first-in-human study will test the safety and tolerability of a single subretinal injection of C.001 in adults with retinal diseases involving retinal pigment epithelium degeneration.

  Recruiting at 2 sites Phase 1 Cellio Therapeutics Inc · Beverly Hills, California and 2 other locations · NCT07594236

• Functional visual field and navigation testing for moderate-to-profound inherited retinal disease

  This project will test whether functional visual field and navigation tests can measure real-world vision and mobility in people with moderate-to-profound inherited retinal diseases.

  Recruiting Ray Therapeutics, Inc. · Irvine, California · NCT07502664

• Single intravitreal RTx-021 injection for Stargardt disease

  This will test whether a single injection of RTx-021 into one eye is safe and may help people aged 16 and older with Stargardt disease.

  Recruiting at 3 sites Phase 1/2 Ray Therapeutics, Inc. · Bakersfield, California and 2 other locations · NCT07439887

• Tracking how Stargardt-type macular dystrophy progresses in children and adults

  This project will follow children and adults with Stargardt-type macular dystrophies over time to see how their vision and eye findings change.

  Recruiting at 15 sites Astellas Pharma Inc · Phoenix, Arizona and 14 other locations · NCT07425574

• ALK-001 to slow progression of Stargardt disease

  This trial will test whether ALK-001 can slow vision loss in people aged 8 to 45 with ABCA4-related Stargardt disease.

  Recruiting at 12 sites Phase 3 Alkeus Pharmaceuticals, Inc. · Phoenix, Arizona and 13 other locations · NCT07419334

• DC6001 tablets in healthy Chinese adults

  This trial will test single and repeated doses of DC6001 tablets in healthy Chinese adults to see how the drug is tolerated, how the body handles it, and whether food affects absorption.

  Recruiting Phase 1 Heronova Pharmaceuticals · Beijing, Beijing Municipality · NCT07417566

• Restoring central vision with the PRIMA implant for photoreceptor degeneration

  This trial will test whether a small PRIMA implant can restore limited central vision in adults with inherited photoreceptor degeneration such as Stargardt disease or retinitis pigmentosa.

  Recruiting Science Corporation · Sydney, New South Wales · NCT07266584

• AAVB-039 gene therapy for Stargardt disease

  This study will test a single subretinal injection of AAVB-039 gene therapy in people with Stargardt disease caused by two ABCA4 mutations to see if it is safe and shows early benefit.

  Recruiting at 11 sites Phase 1/2 AAVantgarde Bio Srl · Phoenix, Arizona and 10 other locations · NCT07161544

• Evaluating a new gene therapy for Stargardt Disease

  This study is testing a new gene therapy to see if it can safely help people with Stargardt Disease Type 1 slow down or improve their vision loss.

  Recruiting at 5 sites Phase 1/2 Splice Bio · Aurora, Colorado and 4 other locations · NCT06942572

• Assessing a vision test for patients with macular degeneration and Stargardt disease

  This study is testing a new vision test to see how well it works for people with macular degeneration and Stargardt disease compared to standard vision tests.

  Recruiting Nanoscope Therapeutics Inc. · Dallas, Texas · NCT06805474

• Identifying potential participants for Stargardt disease clinical trials

  This study is trying to find people with Stargardt disease who might qualify for upcoming clinical trials by checking if they meet certain criteria, like having specific gene mutations.

  Recruiting at 7 sites Ascidian Therapeutics, Inc · San Francisco, California and 8 other locations · NCT06445322

• Observing the progression of Stargardt Disease Type 1

  This study is tracking how Stargardt Disease Type 1 progresses in about 75 people to better understand the condition and help improve future treatments.

  Recruiting at 20 sites Splice Bio · San Diego, California and 19 other locations · NCT06435000

• Assessing vision measurement methods for patients with retinal degeneration

  This study is testing different ways to measure vision in people with retinal degeneration to see how their eyesight affects daily life and how it changes over time.

  Recruiting Ray Therapeutics, Inc. · Irvine, California · NCT06375239

• Disulfiram (Antabuse) for vision in retinal degeneration

  This study will test whether taking oral disulfiram (Antabuse) improves vision in adults with retinal degeneration who are also being treated for alcohol use disorder.

  Recruiting Phase 1 University of Rochester · Rochester, New York · NCT06319872

• Using stem cells to treat retinal and optic nerve damage

  This study is testing if using stem cells from your own bone marrow can help improve vision in people with serious eye damage from conditions like age-related macular degeneration and optic nerve problems.

  Recruiting at 4 sites MD Stem Cells · Westport, Connecticut and 3 other locations · NCT03011541

• Registry for inherited retinal degenerative diseases

  This study is creating a registry for people with rare inherited eye diseases to help track their health and experiences over time, making it easier to understand these conditions and support research.

  Recruiting Foundation Fighting Blindness · Columbia, Maryland · NCT02435940

NIH-funded research (3)

Early-stage science funded by the US National Institutes of Health. Most of these projects don't enroll patients, but they show what is being worked on.

• Combination drug approach to slow vision loss in Stargardt disease

  This project tests a combo-drug strategy to lower toxic eye pigments and help protect vision for people with Stargardt disease.

  NIH research Columbia University Health Sciences · New York, United States · NIH-11303329

• New treatment approach for Stargardt disease

  This study is exploring a new way to help people with Stargardt disease by testing a treatment that could protect their eyesight by reducing harmful substances in the eye.

  NIH research Columbia University Health Sciences · New York, United States · NIH-11044985

• Investigating a new oral treatment for Stargardt disease to slow vision loss.

  This study is testing a new pill called ALK-001 that aims to help people with Stargardt disease by slowing down vision loss caused by a problem with a specific gene.

  NIH research Alkeus Pharmaceuticals, INC. · Somerville, United States · NIH-10433821

Related topics

Quick answers

How many Stargardt Disease clinical trials are recruiting right now?

17, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.

Where are these trials running?

In 8 countries. The most sites are in United States (15 trials), United Kingdom (2 trials), Norway (1 trial), China (1 trial), Australia (1 trial). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 1: 4, Phase 1/2: 3, Phase 3: 1. Trials with no phase listed are usually device, behavioural or observational studies.

How do I join one?

Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.

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