RecruitingPhase 1/2

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

China15 participantsStarted 2024-12-23

Plain-language 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 retinal dystrophy (Stargardt disease) due to biallelic ABCA4 mutations.

Who can participate

Age range6 Years

SexALL

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Inclusion 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

✕. 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.).

What they're measuring

Adverse Events (AEs) and Serious Adverse Events (SAEs)

Timeframe: Baseline to Month 12

Trial details

NCT IDNCT07002398

SponsorVeonGen Therapeutics GmbH

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2026-05

Contact for this trial

Research Coordinator

+86-021-36123569 shiyilunli@sina.com

View on ClinicalTrials.gov More Retinal Dystrophy Due to Biallelic ABCA4 Mutations trials