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This study investigates the safety and early effectiveness of an experimental treatment called VG801 in patients with retinal dystrophy (Stargardt disease, an inherited eye condition) caused by mutations in the ABCA4 gene. It can include people aged 6 and over who have a molecularly confirmed ABCA4 mutation and poor vision in the study eye. The study is in Phase 1/Phase 2 and is currently recruiting participants.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: To be eligible for study entry, subjects must satisfy all the following criteria: 1. Written informed consent. 2. Subjects aged ≥ 6 years. 3. Clinical diagnosis of a macular lesion phenotypically consistent with a recessive hereditary macular dystrophy (Stargardt disease). 4. Confirmed molecular diagnosis of ABCA4 mutations (homozygotes or compound heterozygotes). 5. Poor vision in the study eye. Exclusion Criteria: Subjects will be excluded from the study if one or more of the following statements are applicable to either eye: 1. Pre-existing eye conditions such as uveitis, glaucoma, or diabetic retinopathy or implantation of a medical device in the vitreous cavity or subretinal space. 2. Systemic diseases that would preclude the planned surgery or interfere with the interpretation of study results. 3. History of intraocular surgery within the previous 6 months. 4. Previous participation in a gene therapy trial. 5. Participation in a clinical trial (investigational drug or medical device) within the previous 6 months. 6. Any other eye disease that may affect the outcome of the study (e.g., ocular opacities, advanced cataracts, amblyopia, etc.).