Actively Recruiting
Safety and Preliminary Efficacy of VG801 in Patients With ABCA4 Mutation-associated Retinal Dystrophy (Stargardt Disease)
Led by VeonGen Therapeutics GmbH · Updated on 2025-12-11
15
Participants Needed
1
Research Sites
70 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Safety and Preliminary Efficacy of VG801 in Patients With ABCA4 Mutation-associated Retinal Dystrophy (Stargardt Disease)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Written informed consent is given.
- Participants are aged 6 years or older.
- Clinical diagnosis of a macular lesion consistent with Stargardt disease or recessive hereditary macular dystrophy.
- Confirmed molecular diagnosis of ABCA4 mutations (homozygotes or compound heterozygotes).
- Poor vision in the study eye.
You will not qualify if you...
- Presence of eye conditions like uveitis, glaucoma, diabetic retinopathy, or implanted medical devices in the vitreous or subretinal space.
- Systemic diseases preventing surgery or affecting study results.
- History of intraocular surgery within the last 6 months.
- Previous participation in a gene therapy trial.
- Participation in another clinical trial involving investigational drugs or devices within the last 6 months.
- Other eye diseases that could impact study outcomes, such as ocular opacities, advanced cataracts, or amblyopia.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Shanghai General Hospital
Shanghai, Shanghai Municipality, China, 200080
Actively Recruiting
Research Team
R
Research Coordinator
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
1
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