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

A Single Arm, Ph1/2, Open-label, Multicenter Trial With Dose-exploration Via Subretinal Injection to Evaluate the Safety and Preliminary Efficacy of VG801 for Treatment of ABCA4 Mutation-associated Recessive Hereditary Retinal Dystrophy (Stargardt Disease)

Status

Recruiting

Phases

Phase 1Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT07002398

Enrollment

Registered

2025-06-03

Start date

2024-12-23

Completion date

2027-12-01

Last updated

2026-09-02

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Retinal Dystrophy Due to Biallelic ABCA4 Mutations, Stargardt Disease 1

Keywords

ABCA4 mutation, Gene therapy, Eye disease, Stargardt disease, Retinal disease, Retinal degeneration, VG801

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.

Interventions

DRUGVG801

Administered as specified in the single treatment arm. Study Cohort: Low dose, medium dose and high dose cohort

Study design

Allocation

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

6 Years to No maximum

Healthy volunteers

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.).

Design outcomes

Primary

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

Secondary

Measure	Time frame	Description
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 Visual Test (Exploratory)	Baseline to Month 12	—

Countries

China

Contacts

CONTACTResearch Coordinator

shiyilunli@sina.com+86-021-36123569

PRINCIPAL_INVESTIGATORXiaodong Sun, MD

Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Sep 3, 2026