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

VeonGen Therapeutics GmbH

Status and phase: Enrolling; Phase 2; Phase 1

Conditions: Retinal Dystrophy Due to Biallelic ABCA4 Mutations; Stargardt Disease 1

Treatments: Drug: VG801

Study type: Interventional

Funder types: Industry

Identifiers

NCT07002398

VG801-2022A

Details and patient eligibility

About

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.

Enrollment: 15 estimated patients

Sex: All

Ages: 6+ years old

Volunteers: No Healthy Volunteers

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.).

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Sequential Assignment

Masking: None (Open label)

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15 participants in 1 patient group

VG801

Experimental group

Description:

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

Treatment:

Drug: VG801