Safety and Preliminary Efficacy of VG801 in Patients With ABCA4 Mutation-associated Retinal Dystrophy (Stargardt Disease)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: VG801.
- Who it may be relevant to
- Registry conditions: Retinal Dystrophy Due to Biallelic ABCA4 Mutations, Stargardt Disease 1. Basic parameters: from 6 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- China
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
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)
Overview
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.
Interventions
- Drug VG801
Administered as specified in the single treatment arm. Study Cohort: Low dose, medium dose and high dose cohort
Primary outcome measures
- Adverse Events (AEs) and Serious Adverse Events (SAEs) [Time frame: Baseline to Month 12]
Secondary outcome measures (5)
- Best Corrected Visual Acuity (BCVA) [Time frame: Screening to Month 12]
- Optical coherence tomography (OCT) [Time frame: Baseline to Month 12]
- Fundus autofluorescence [Time frame: Baseline to Month 12]
- Microperimetry [Time frame: Baseline to Month 12]
- Novel Virtual Reality Visual Test (Exploratory) [Time frame: Baseline to Month 12]
Eligibility criteria
Inclusion criteria
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.
Exclusion criteria
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.).
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Sequential
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
China · 1 center
- Shanghai General Hospital — Shanghai
Identifiers
NCT: NCT07002398 · VG801-2022A