Recruiting

Phase 1

Phase 2

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

Sponsor:

AAVantgarde Bio Srl

Code:

NCT07161544

Conditions

Stargardt Disease

Eligibility Criteria

Sex: All

Age: 8 - 55

Healthy Volunteers: Not accepted

Interventions

AAVB-039

Study Details

Brief 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.

Conditions

Stargardt Disease

Study ID

NCT07161544

Start date

Sep 29, 2025

Status verified date

Jul, 2026

Completion date

Jul, 2032

Anticipated

Primary completion date

Jul, 2032

Anticipated

Eligibility Criteria

Sex: All

Age: 8 - 55

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

75 participants

Anticipated

Allocation

Non randomized

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Cohort 1

AAVB-039 dose level 1 (low dose)

experimental: Cohort 2

AAVB-039 dose level 2 (mid dose)

experimental: Cohort 3

AAVB-039 dose level 3 (high dose)

experimental: Cohorts 4-5

AAVB-039 dose level 1, 2 or 3

Interventions

AAVB-039

Single subretinal administration

Primary outcome measure

To measure the number and severity of treatment related adverse events following treatment with AAVB-039 [ Time Frame: 0-60 months ]

Central Contacts and Locations

Central contacts

AAVantgarde Clinical Operations Lead

+448000465680 clinicaltrials@aavantgarde.com

Locations

Associated Retina Consultants

Recruiting

Phoenix, Arizona, United States, 85020

Contacts

Study Team

hello@doctrials.com

Principal Investigator:

Dr. Benjamin Bakall

Retina Vitreous Associates Medical Group

Recruiting

Los Angeles, California, United States, 90211

Contacts

Kristina Passoian, Trials Director

Kpassoian@laretina.com

Study Referrals

Studyreferrals@laretina.com

Principal Investigator:

Dr. David Liao

University of Florida Health Science Center

Recruiting

Jacksonville, Florida, United States, 32209

Contacts

G. Shabbir Hamdani

Shabbir.hamdani@ufhealth.org

Principal Investigator:

Dr. Sandeep Grover

Mayo Clinic

Recruiting

Rochester, Minnesota, United States, 55905

Contacts

Suzy Wernimont

507-538-8119 Wernimont.Suzanne@mayo.edu

Principal Investigator:

Dr. Brittni Scruggs

Retina Consultants of Texas

Recruiting

Bellaire, Texas, United States, 77401

Principal Investigator:

Dr. Kenneth Fan

Retina Foundation of the Southwest

Recruiting

Dallas, Texas, United States, 75231

Contacts

Senior Clinical Research Coordinator

jclark@retinafoundation.org

Principal Investigator:

Dr. Karl Csaky

University of Utah

Recruiting

Salt Lake City, Utah, United States, 84112

Contacts

Kimberley Wegner Lead Coordinator

Kimberley.wegner@hsc.utah.edu

Principal Investigator:

Dr. Paul Bernstein

More Information

Sponsor

AAVantgarde Bio Srl

Last update posted

Jul 30, 2026

Last verified

Jul, 2026

Keywords

Stargardt Disease
Biallelic Mutation
Gene Mutation
ABCA4

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by AAVantgarde Bio Srl on 2026-07-30.