A Study of AAVB-039 in Participants With Stargardt Disease (STGD1)

NCT07161544 · tracked via the Priya Life Science UK tracker

Sponsor

AAVantgarde Bio Srl

Phase

Phase 1/2

Started

2025-09-29

Last updated

2026-07-30

Condition(s) studied

Stargardt Disease

Investigational drug(s) / intervention(s)

AAVB-039

AAVB-039: Single subretinal administration

Study summary

The purpose of the 039-101 study is to evaluate the safety and tolerability of a single subretinal injection of AAVB-039 in participants with Stargardt disease secondary to a biallelic mutation of the ABCA4 gene. The study will also assess initial efficacy following AAVB-039 administration.

Eligibility

Sex

ALL

Min age

8 Years

Max age

55 Years

Healthy volunteers

Inclusion Criteria: * Molecular diagnosis of Stargardt disease due to ABCA4 mutation * Willingness to adhere to protocol per informed consent Exclusion Criteria: * Unwillingness to meet the requirements of the study * Participation in a clinical study with another Investigation Medicinal Product * Previous participation in another gene or cell therapy trial * Any condition that would preclude subretinal surgery * Complicating ocular and/or systemic diseases

Primary outcome measure(s)

To measure the number and severity of treatment related adverse events following treatment with AAVB-039 — 0-60 months

Trial sites (9)

Facility	City	Region	Status
Associated Retina Consultants	Phoenix	Arizona	Recruiting
Retina Vitreous Associates Medical Group	Los Angeles	California	Recruiting
University of Florida Health Science Center	Jacksonville	Florida	Recruiting
Mayo Clinic	Rochester	Minnesota	Recruiting
Retina Consultants of Texas	Bellaire	Texas	Recruiting
Retina Foundation of the Southwest	Dallas	Texas	Recruiting
University of Utah	Salt Lake City	Utah	Recruiting
Moorfields Eye Hospital NHS Foundation Trust	London	England	Recruiting
The Retina Clinic	London	England	Recruiting

More AAVantgarde Bio Srl trials in the UK

Study of Subretinally Injected AAVB-081 in Patients With Usher Syndrome Type IB (USH1B) Retinitis Pigmentosa Recruiting

Other trials for the same condition

An Observational Study in Subjects to Follow the Progression of Stargardt Disease Type 1 (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ABCA4 Gene Recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07161544 on ClinicalTrials.gov ↗ ← All trials in the UK