Overview
This is a single-arm, open-label, non-randomized, single dose-escalation, first-in-human (FIH) clinical trial to evaluate the safety and preliminary efficacy of VG801 for treatment of patients with retinal dystrophy (Stargardt disease) due to biallelic ABCA4 mutations.
Eligibility
Inclusion Criteria:
To be eligible for study entry, subjects must satisfy all the following criteria:
- Written informed consent.
- Subjects aged ≥ 6 years.
- Clinical diagnosis of a macular lesion phenotypically consistent with a recessive hereditary macular dystrophy (Stargardt disease).
- Confirmed molecular diagnosis of ABCA4 mutations (homozygotes or compound heterozygotes).
- 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:
- Pre-existing eye conditions such as uveitis, glaucoma, or diabetic retinopathy or implantation of a medical device in the vitreous cavity or subretinal space.
- Systemic diseases that would preclude the planned surgery or interfere with the interpretation of study results.
- History of intraocular surgery within the previous 6 months.
- Previous participation in a gene therapy trial.
- Participation in a clinical trial (investigational drug or medical device) within the previous 6 months.
- Any other eye disease that may affect the outcome of the study (e.g., ocular opacities, advanced cataracts, amblyopia, etc.).