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

Phase 1

Recruiting

• Conditions: Retinal Dystrophy Due to Biallelic ABCA4 Mutations; Stargardt Disease 1
• Interventions: Drug: VG801

• Registration Number: NCT07002398
• Lead Sponsor: ViGeneron GmbH

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

Detailed Description

Not available

Study Timeline

• Study Start: 2024-12-23
• Status Verified: 2025-01
• Study First Submitted: 2025-05-23
• Study First Submitted QC: 2025-05-23
• Last Update Submitted: 2025-05-23
• Study First Posted: 2025-06-03
• Last Update Posted: 2025-06-03
• Primary Completion: 2026-05
• Study Completion: 2026-05

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 15

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.).

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
VG801	VG801	Participants will receive a single dose of subretinal injection of VG801at Day 0.

Primary Outcome Measures

Name	Time	Method
Adverse Events (AEs) and Serious Adverse Events (SAEs)	Baseline to Month 12	Abnormal examination results will be recorded.

Secondary Outcome Measures

Name	Time	Method
Best Corrected Visual Acuity (BCVA)	Screening to Month 12	Abnormal examination results will be recorded.
Optical coherence tomography (OCT)	Baseline to Month 12	Abnormal examination results will be recorded.
Fundus autofluorescence	Baseline to Month 12	Abnormal examination results will be recorded.
Microperimetry	Baseline to Month 12	Abnormal examination results will be recorded.
Novel Virtual Reality Mobility Test (Exploratory)	Baseline to Month 12

Trial Locations

Locations (1)

Shanghai General Hospital; 🇨🇳 Shanghai, Shanghai, China