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

NCT07002398 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 15

Last updated 2025-12-11

No results posted yet for this study

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.

Conditions

  • Retinal Dystrophy Due to Biallelic ABCA4 Mutations
  • Stargardt Disease 1

Interventions

DRUG

VG801

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

Sponsors & Collaborators

  • VeonGen Therapeutics GmbH

    lead INDUSTRY

Principal Investigators

  • Xiaodong Sun, MD · Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
6 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-12-23
Primary Completion
2026-05-31
Completion
2026-05-31
FDA Drug
Yes

Countries

  • China

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07002398 on ClinicalTrials.gov