VG801 gene therapy for Stargardt disease from ABCA4 mutations

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a single-arm, open-label, dose-escalation first-in-human trial administering a single dose of VG801 to patients with biallelic ABCA4-related Stargardt disease to test safety and preliminary signs of benefit. Participants must have a confirmed molecular diagnosis and poor vision in the study eye and will be enrolled into escalating dose cohorts. Key exclusions include other active ocular disease, recent intraocular surgery, systemic conditions that preclude surgery, or prior gene therapy. Patients will undergo the planned ocular procedure and scheduled follow-up visits to monitor safety and visual outcomes over the study period.

Why it matters

Who can join: the full rules

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.).

Where it's running

• Shanghai General Hospital
  Shanghai, Shanghai Municipality, China Recruiting

Study contacts

• Principal investigator: Xiaodong Sun, MD — Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
• Study coordinator: Research Coordinator
• Email: shiyilunli@sina.com
• Phone: +86-021-36123569

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Identifying potential participants for Stargardt disease clinical trials

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• Gene therapy for Retinitis Pigmentosa caused by CNGA1 mutations

  This study is testing a new gene therapy called VG901 to see if it is safe and can help people with Retinitis Pigmentosa caused by CNGA1 gene mutations.

  Recruiting Phase 1 VeonGen Therapeutics GmbH · Tübingen · NCT06291935

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.