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RECRUITING
NCT07002398
PHASE1/PHASE2

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

Sponsor: VeonGen Therapeutics GmbH

View on ClinicalTrials.gov

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.

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)

Key Details

Gender

All

Age Range

6 Years - Any

Study Type

INTERVENTIONAL

Enrollment

15

Start Date

2024-12-23

Completion Date

2026-05

Last Updated

2025-12-11

Healthy Volunteers

No

Interventions

DRUG

VG801

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

Locations (1)

Shanghai General Hospital

Shanghai, Shanghai Municipality, China