Clinical Trial

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

Recruiting Phase 1/2
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Record status
This record was last updated December 11, 2025 (before its estimated May 2026 completion). Its status may not reflect the trial's current state.
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.
Protocol Amendment History 1 amendment
This ClinicalTrials.gov record has been amended once since 2025-05-23.
Trial Details
NCT Number NCT07002398
Lead Sponsor VeonGen Therapeutics GmbH
Conditions Retinal Dystrophy Due to Biallelic ABCA4 Mutations, Stargardt Disease 1
Enrollment 15 participants
Start Date 2024-12-23
Primary Completion 2026-05 (estimated)
Study Completion 2026-05 (estimated)
Updated on ClinicalTrials.gov 2025-12-11