NCT07002398RecruitingPhase 1/2
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)
Source: ClinicalTrials.gov · Last updated 2 Sept 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Recruiting
- Phase (registry)
- Phase 1/2
- Study type
- Interventional
- Enrollment
- 18 (estimated)
- First submitted
- 23 May 2025
- Start date
- 23 Dec 2024
- Primary completion date
- Dec 2027
- Lead sponsor
- VeonGen Therapeutics GmbH
- NCT number
- NCT07002398
What you commit to
- Study type: Interventional · Phase 1/2
- Planned participants: 18
- From 23 Dec 2024 to the primary completion date, Dec 2027
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- 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.
Other illness
- 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.
- Any other eye disease that may affect the outcome of the study (e.g., ocular opacities, advanced cataracts, amblyopia, etc.).
Other
- Subjects will be excluded from the study if one or more of the following statements are applicable to either eye:
Who the study is looking for
- 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.
Age in the registry: 6 years to - · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Save your city in your profile to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Shanghai General Hospital Coordinator of this site? Confirm its status | Shanghai | China | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.