RecruitingPhase 1Phase 2NCT07002398

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

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)

Sponsor

VeonGen Therapeutics GmbH

Enrollment

15 participants

Start Date

Dec 23, 2024

Study Type

INTERVENTIONAL

Conditions

Retinal Dystrophy Due to Biallelic ABCA4 Mutations Stargardt Disease 1

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.

Eligibility

Min Age: 6 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called VG801 for people with retinal dystrophy due to biallelic abca4 mutations and stargardt disease 1. The study is currently recruiting participants at 1 location. People eligible for this study include aged 6 Years and older.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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