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RecruitingNCT07002398Updated Sep 2, 2026

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

A Phase 1/2 interventional study of VG801 in Retinal Dystrophy Due to Biallelic ABCA4 Mutations and Stargardt Disease 1, sponsored by VeonGen Therapeutics GmbH. Recruiting at 1 site in China. Open to participants aged 6 Years and older. Per ClinicalTrials.gov, last updated 2026-09-02.

Sponsored by VeonGen Therapeutics GmbH · Phase 1/2, Interventional, and Treatment

From the registry’s dates

  • Registered 5 months after the study started (first participant enrolled Dec 2024, registered May 2025).
  • Started Dec 2024; still recruiting 1 year 9 months later.
Phase
Phase 1/2
Study type
Interventional
Enrollment
18
Allocation
Not applicable
Ages
6 Years and older
Sex
All
01

Study 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.

02

Conditions studied

  • Retinal Dystrophy Due to Biallelic ABCA4 Mutations
  • Stargardt Disease 1

Keywords

  • ABCA4 mutation
  • Gene therapy
  • Eye disease
  • Stargardt disease
  • Retinal disease
  • Retinal degeneration
  • VG801
03

In context

Stargardt Disease

67 studies on the registry are indexed under Stargardt Disease; 26 are open to participants now.

This study's planned enrollment of 18 is below the median of 28 across 44 interventional studies indexed under Stargardt Disease.

Browse Stargardt Disease studies →

Lead sponsor

This is the only study on the registry with VeonGen Therapeutics GmbH as lead sponsor.

Counted across the registry records on this site, refreshed daily.

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Who can participate

Ages eligible
6 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

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

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.).
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Study design

Phase
Phase 1 / Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Sequential assignment
Masking
None (open label)
Enrollment
18 participants (estimated)

Study arms

  • Experimental
    VG801

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

    Drug: VG801

Interventions

  • DrugVG801

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

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What researchers measure

Primary outcomes

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

    Abnormal examination results will be recorded.

    Time frame: Baseline to Month 12

Secondary outcomes

  1. Best Corrected Visual Acuity (BCVA)

    Abnormal examination results will be recorded.

    Time frame: Screening to Month 12

  2. Optical coherence tomography (OCT)

    Abnormal examination results will be recorded.

    Time frame: Baseline to Month 12

  3. Fundus autofluorescence

    Abnormal examination results will be recorded.

    Time frame: Baseline to Month 12

  4. Microperimetry

    Abnormal examination results will be recorded.

    Time frame: Baseline to Month 12

  5. Novel Virtual Reality Visual Test (Exploratory)

    Time frame: Baseline to Month 12

07

Study locations

1 of 1 sites recruiting
  • Shanghai General Hospital
    Shanghai, Shanghai Municipality 200080, China
    • Shanghai General Hospital · Contact · +86-021-36123569
    • Xiaodong Sun, MD · Principal investigator
    Recruiting
08

References and documents

Individual participant data

Plan to share: Undecided

No publications or documents are linked to this record.

09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Sep 2, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
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Registry details

Key details

Study ID
NCT07002398
Lead sponsor
VeonGen Therapeutics GmbH
Responsible party
Sponsor
First posted
Jun 3, 2025
Start date
Dec 23, 2024
Primary completion
Dec 2027 (estimated)
Completion
Dec 2027 (estimated)
Last update
Sep 2, 2026

Study contacts

Research Coordinator
Contact
shiyilunli@sina.com
+86-021-36123569
Xiaodong Sun, MD
principal investigator · Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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