Safety and Preliminary Efficacy of VG801 in Patients With ABCA4 Mutation-associated Retinal Dystr… (NCT07002398) | Clinical Trial Compass
RecruitingPhase 1/2
Safety and Preliminary Efficacy of VG801 in Patients With ABCA4 Mutation-associated Retinal Dystrophy (Stargardt Disease)
China15 participantsStarted 2024-12-23
Plain-language 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.
Who can participate
Age range
6 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion 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
. Pre-existing eye conditions such as uveitis, glaucoma, or diabetic retinopathy or implantation of a medical device in the vitreous cavity or subretinal space.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Adverse Events (AEs) and Serious Adverse Events (SAEs)