Clinical trial NCT07002398: Safety and Preliminary Efficacy of VG801 in Patients With ABCA4 Mutation-associated Retinal Dystrophy (Stargardt Disease)
Official title: 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)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
PHASE2
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
Lead sponsor: ViGeneron GmbH
Sponsor class: INDUSTRY
First posted: 2025-06-03
Enrollment: 15
Sex: ALL
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

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.


Eligibility criteria: 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.).

Primary outcomes:
measure: Adverse Events (AEs) and Serious Adverse Events (SAEs)
timeFrame: Baseline to Month 12
Secondary outcomes:
measure: Best Corrected Visual Acuity (BCVA)
timeFrame: Screening to Month 12
measure: Optical coherence tomography (OCT)
timeFrame: Baseline to Month 12
measure: Fundus autofluorescence
timeFrame: Baseline to Month 12
measure: Microperimetry
timeFrame: Baseline to Month 12
measure: Novel Virtual Reality Mobility Test (Exploratory)
timeFrame: Baseline to Month 12
Other outcomes:
Locations:
city: Shanghai
country: China
facility: Shanghai General Hospital
state: Shanghai
Study officials:
affiliation: Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
name: Xiaodong Sun, MD
role: PRINCIPAL_INVESTIGATOR
References: