STARGARDT DISEASE

This early-stage study is testing the safety and initial effects of a single gene therapy injection, called RTx-021, for people with Stargardt disease, a rare genetic condition that causes vision loss. Up to 18 patients aged 16 and older will receive the injection in one eye and …

This is the first human trial of an experimental gene therapy called ACDN-01 for people with ABCA4-related eye diseases like Stargardt disease. The study will test the safety and early signs of effectiveness of a single injection into the eye. Fifteen adult participants will be f…

This study is testing if the oral diabetes medication metformin is safe and can slow down the progression of ABCA4 retinopathy, a genetic eye disease that causes vision loss. It will involve about 56 participants aged 12 and older who have the disease. Participants will take metf…

This is the first study in humans to test the safety of an oral tablet called DC6001, which is being developed for Stargardt disease—a genetic eye condition that causes vision loss. The trial will enroll 76 healthy Chinese adults to see how their bodies process different doses of…

This study is testing whether disulfiram, a medication used to treat alcohol use disorder, can improve vision in people with retinal degeneration diseases like macular degeneration and retinitis pigmentosa. The trial will enroll 15 adults who have both a retinal condition and alc…

This study is testing a one-time gene therapy injection called OCU410ST for Stargardt disease, a genetic eye disorder that causes vision loss in children and young adults. The trial will enroll 51 participants, with about two-thirds receiving the injection and the rest being in a…

This study is testing a new gene therapy, AAVB-039, for people with Stargardt disease, a genetic condition that causes vision loss. The main goal is to see if a single injection into the back of the eye is safe. Researchers will also check if the treatment can slow or stop the pr…

This study aims to better understand how Stargardt disease, a genetic eye condition that causes vision loss, progresses over time. Researchers will follow about 75 people with the disease, using eye scans and vision tests to track changes. The information gathered will help scien…

This study aims to identify people with specific inherited eye diseases, like Stargardt disease, who might be eligible for future treatment trials. It will recruit 50 participants to perform tests that check their genetic profile and eye health. The goal is to create a pool of pr…