STARGARDT DISEASE

This study tests whether injecting a person's own bone marrow stem cells into or near the eye can help treat various retinal and optic nerve diseases, including age-related macular degeneration, retinitis pigmentosa, and glaucoma. Participants receive stem cell injections via dif…

This study tests a gene therapy called AAVB-039 for Stargardt disease, an inherited condition that causes vision loss. About 75 adults with a specific gene mutation will receive a single injection under the retina. The main goal is to check safety, but researchers will also measu…

This early-stage study tests a new treatment called C.001 for people with retinal diseases that cause vision loss, such as geographic atrophy and Stargardt disease. The treatment is given as a single injection into the eye. The main goal is to check if it is safe, and researchers…

This Phase 3 trial tests an oral drug called gildeuretinol acetate (ALK 001) in 230 people aged 8 to 45 with Stargardt disease, a genetic condition that causes progressive vision loss. Participants take the drug or a placebo daily for 24 months. The goal is to see if the drug can…

This early-stage trial tests a single injection of a gene therapy called RTx-021 in the eye of people with Stargardt disease, a genetic condition that causes vision loss. Up to 18 participants will receive different doses to check safety and tolerability. Researchers will monitor…

This study tests a device called PRIMA, which is implanted under the retina to help people with inherited retinal diseases like Stargardt disease and retinitis pigmentosa see better. The trial will enroll 5 adults with severe central vision loss and measure improvements in visual…

This early study tests a new gene therapy called ACDN-01 for people with Stargardt disease, a genetic condition that leads to progressive vision loss. The therapy is given as a one-time injection under the retina. The main goals are to check safety and find the right dose, with a…

This study looks at whether disulfiram (Antabuse), a drug used for alcohol use disorder, can improve vision in people with retinal degeneration (like macular degeneration or retinitis pigmentosa). Researchers believe it may calm overactive cells in the retina. The study involves …

This study follows 90 children and adults with Stargardt disease or similar inherited eye conditions to see how their vision changes over 2 years. Participants will have regular eye exams and imaging every 6 months. No treatment is given—the goal is to better understand the natur…

This study uses a special camera called an Adaptive Optics Scanning Laser Ophthalmoscope (AOSLO) to take very detailed pictures of the retina in people with inherited retinal diseases like retinitis pigmentosa. Researchers will measure how the light-sensitive cells in the eye cha…

This registry collects information from people with inherited retinal diseases, like retinitis pigmentosa and Stargardt disease. Participants share their symptoms, family history, and genetic test results online. The goal is to help researchers understand these rare diseases and …

This study enrolls 25 people with severe retinal diseases like retinitis pigmentosa and Stargardt disease to test new ways of measuring visual function. Researchers will evaluate how well participants can perform various vision tests, such as navigating obstacles or detecting lig…

This prescreening study aims to identify up to 50 people with ABCA4-related retinopathy, including Stargardt disease, for possible participation in future ACDN-01 clinical trials. Participants will undergo genetic testing and various eye exams to confirm their condition. This is …

This early-phase trial tests the safety of an oral drug called DC6001 in 76 healthy adults. Researchers will give single or multiple doses to see how the body handles the drug and if it causes side effects. This study does not involve people with Stargardt disease and is not desi…

This observational study will follow 75 people aged 12 to 65 with Stargardt disease type 1, a genetic eye condition that causes vision loss. Researchers will use eye exams and imaging to track how the disease changes over time. The goal is to better understand the disease's progr…