Status:
RECRUITING
Safety and Preliminary Efficacy of VG801 in Patients With ABCA4 Mutation-associated Retinal Dystrophy (Stargardt Disease)
Lead Sponsor:
VeonGen Therapeutics GmbH
Conditions:
Retinal Dystrophy Due to Biallelic ABCA4 Mutations
Stargardt Disease 1
Eligibility:
All Genders
6+ years
Phase:
PHASE1
PHASE2
Brief Summary
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 re...
Eligibility Criteria
Inclusion
- 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
- 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.).
Key Trial Info
Start Date :
December 23 2024
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
May 1 2026
Estimated Enrollment :
15 Patients enrolled
Trial Details
Trial ID
NCT07002398
Start Date
December 23 2024
End Date
May 1 2026
Last Update
December 11 2025
Active Locations (1)
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1
Shanghai General Hospital
Shanghai, Shanghai Municipality, China, 200080