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Safety and Preliminary Efficacy of VG801 in Patients With ABCA4 Mutation-associated Retinal Dystrophy (Stargardt Disease)
Sponsor: VeonGen Therapeutics GmbH
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
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