Safety and Preliminary Efficacy of VG801 in Patients With ABCA4 Mutation-associated Retinal Dystrophy (Stargardt Disease)

Launched by VEONGEN THERAPEUTICS GMBH · May 23, 2025

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called VG801 for patients with Stargardt disease, a type of inherited eye condition that affects vision. Stargardt disease is linked to specific changes in a gene known as ABCA4, and this trial aims to see if VG801 is safe and if it might help improve vision in people with this condition. The trial is currently looking for participants of all genders, aged 6 years and older, who have been diagnosed with Stargardt disease and have poor vision in at least one eye.

If you or a loved one is considering participating in this trial, you should know that participants will receive a single dose of VG801 and be monitored for any side effects. However, there are certain criteria that need to be met to join. For example, individuals must not have other serious eye conditions or diseases that could interfere with the study results. This trial is important as it could pave the way for new treatments for Stargardt disease, giving hope to those affected by this vision impairment.

Gender

ALL

Eligibility criteria

• 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.).