Hereditary macular dystrophy

This early-stage trial is testing whether a gene therapy called NGGT001, injected under the retina, is safe and tolerable for people with Bietti's crystalline dystrophy, a rare inherited condition that causes progressive vision loss. The study enrolls about 11 adults with a confi…

This trial tests an experimental gene therapy called NGGT001 for people with Bietti Crystalline Dystrophy, a rare inherited condition that causes progressive vision loss. The therapy is delivered as a single injection under the retina, aiming to replace a faulty gene with a worki…

Researchers are testing whether a daily pill called ALK-001 can slow the progression of Stargardt disease, a rare inherited condition that causes vision loss in children and young adults. The trial enrolls people aged 8 and older who have a clinical diagnosis of Stargardt disease…

Researchers are testing a daily oral drug called ALK-001 in people aged 8 to 70 with Stargardt disease, an inherited condition that causes progressive vision loss. The trial compares ALK-001 to a placebo over 24 months to see if the drug is safe and can slow damage to the retina.…

This study follows patients with Stargardt's macular degeneration, an inherited eye disease that causes progressive vision loss, who previously received an experimental gene therapy called SAR422459. The goal is to check the long-term safety and tolerability of the treatment and …

This study looks at a rare disease called KSHV-associated multicentric Castleman's disease (MCD), caused by a virus. Researchers are testing several drug combinations, including chemotherapy and antivirals, to see if they can control the disease. The study also tracks how the dis…

This phase 2 study tests the drug sirolimus (rapamycin) in 7 people with idiopathic multicentric Castleman disease who have not improved with standard therapy. The goal is to see if sirolimus can improve symptoms like fatigue, fever, and night sweats, and shrink enlarged lymph no…

This study tests whether metformin, a widely used diabetes drug, can safely slow vision loss in people with ABCA4 retinopathy (Stargardt disease). Fifty-five participants aged 12 and older will take metformin by mouth for 24 months, with regular eye exams to track changes. The go…

This study tests a new gene therapy called OCU410ST for people with Stargardt disease, an inherited eye condition that causes vision loss. Early results show the treatment slowed damage to the retina by nearly half and improved or stabilized eyesight in all treated patients. The …

This trial investigates whether implanting a fresh corneal lenticule — a thin layer of donor cornea taken from myopic patients — can improve transparency and vision in people with macular corneal dystrophy, a condition that clouds the cornea. Using a femtosecond laser, surgeons r…

This study tests a single injection of ZVS101e, a gene therapy that delivers a working copy of the CYP4V2 gene directly into the retina. It is for adults with Bietti's crystalline dystrophy, a rare inherited condition that leads to progressive vision loss. The trial will check wh…

This study tests a single injection of ZVS101e gene therapy for people with Bietti's crystalline dystrophy, a rare inherited condition that leads to progressive vision loss. The therapy delivers a working copy of the CYP4V2 gene directly to the retina. Researchers will measure wh…

This study provides ZVS101e, a gene therapy, to 32 people with Bietti crystalline corneoretinal dystrophy (BCD) who were in the control group of a previous Phase III trial. The therapy uses a harmless virus to deliver a working gene to the eye, aiming to slow vision loss. The mai…

This study tests a drug called tinlarebant in 60 people with Stargardt disease, a genetic condition that causes vision loss. The goal is to see if the drug can slow damage to the retina and preserve eyesight. Participants take the drug or a placebo, and researchers measure change…

This early-stage trial is testing whether it is safe to inject a person's own bone marrow stem cells into their eye to treat vision loss from conditions like macular degeneration and diabetic retinopathy. Fifteen adults with severe vision loss will receive the injection and be mo…

This Phase 3 trial tests a gene therapy called VGR-R01 for Bietti crystalline dystrophy, a rare inherited condition that leads to progressive vision loss. The study will enroll 45 adults who receive either the gene therapy injection under the retina or a control treatment. Resear…

This early-stage trial tests a gene therapy called VGR-R01 for Bietti Crystalline Dystrophy, a rare inherited eye disease that leads to progressive vision loss. The study involves 12 adults who receive the therapy via injection into the eye. The main goal is to check safety and t…

This study follows 68 people aged 12 and older who have ABCA4 gene mutations, which can cause vision loss from diseases like Stargardt disease. Over 10 years, researchers will collect blood and skin samples and perform detailed eye exams to understand how the disease progresses. …

This study follows 80 people aged 8 to 50 with Stargardt disease, a genetic eye condition that causes vision loss. Researchers will monitor how the disease changes over time using eye imaging. The goal is to better understand the disease and prepare for future clinical trials.

This study aims to understand how diet affects the eyes and skin by measuring natural pigments called carotenoids. Researchers will use non-invasive methods to check levels in the eye and skin, and also test blood in some participants. Anyone age 7 or older having a routine eye e…

This study looks at how common Charles Bonnet Syndrome (CBS) is in people with vision loss from conditions like macular degeneration or glaucoma. CBS causes complex visual hallucinations without mental illness. Researchers will survey 640 participants to measure how often it happ…