Safety and Preliminary Efficacy of VG801 in Patients With ABCA4 Mutation-associated Retinal Dystrophy (Stargardt Disease)

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

15 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-12-23

Study Completion Date

2026-05-31

Brief Summary

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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.

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Detailed Description

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Conditions

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Retinal Dystrophy Due to Biallelic ABCA4 Mutations Stargardt Disease 1

Study Design

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Allocation Method

NA

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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VG801

Participants will receive a single dose of subretinal injection of VG801at Day 0.

Group Type EXPERIMENTAL

VG801

Intervention Type DRUG

Administered as specified in the single treatment arm. Study Cohort: Low dose, medium dose and high dose cohort

Interventions

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VG801

Administered as specified in the single treatment arm. Study Cohort: Low dose, medium dose and high dose cohort

Intervention Type DRUG

Eligibility Criteria

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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.).

Minimum Eligible Age

6 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No