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

NCT07002398 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: VG801-2022A
• Study Type: interventional
• Target: 18
• Locations: 1 country
• Sites: 1

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.

Conditions

Stargardt Disease 1; Retinal Dystrophy Due to Biallelic ABCA4 Mutations

Keywords

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

Outcome Measures

Primary Outcomes (1)

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

  Abnormal examination results will be recorded.

  Baseline to Month 12

Secondary Outcomes (5)

• Best Corrected Visual Acuity (BCVA)

  Screening to Month 12

• Optical coherence tomography (OCT)

  Baseline to Month 12

• Fundus autofluorescence

  Baseline to Month 12

• Microperimetry

  Baseline to Month 12

• Novel Virtual Reality Visual Test (Exploratory)

  Baseline to Month 12

Study Arms (1)

VG801

EXPERIMENTAL

Participants will receive a single dose of subretinal injection of VG801at Day 0.

Drug: VG801

Interventions

VG801 DRUG

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

VG801

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.).

Sponsors & Collaborators

• VeonGen Therapeutics GmbH: lead

Study Sites (1)

Shanghai General Hospital

Shanghai, Shanghai Municipality, 200080, China

RECRUITING

MeSH Terms

Conditions

Stargardt Disease; Eye Diseases; Retinal Diseases; Retinal Degeneration

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Macular Degeneration; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Xiaodong Sun, MD

  Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Research Coordinator

CONTACT

+86-021-36123569; shiyilunli@sina.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 23, 2025
• First Posted: June 3, 2025
• Study Start: December 23, 2024
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): December 1, 2027
• Last Updated: September 2, 2026

Record last verified: 2026-08

Locations

CN (1)