Actively Recruiting
Assessing Safety and Efficacy of Subretinal AAVB-039 in Stargardt Disease (STGD1)
Led by AAVantgarde Bio Srl · Updated on 2026-05-29
75
Participants Needed
7
Research Sites
N/A
Total Duration
On this page
Sponsors
A
AAVantgarde Bio Srl
Lead Sponsor
A
AAVantgarde Bio UK Ltd
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and initial effectiveness of a single subretinal injection of AAVB-039 in people with Stargardt disease caused by mutations in the ABCA4 gene. This study is an open-label trial conducted in multiple centers, focusing on participants aged 8 to 55 years. It aims to understand how well this gene therapy is tolerated and whether it can impact disease progression. Participants will receive one of three dose levels of AAVB-039 administered directly under the retina in a single procedure. The study includes different cohorts receiving low, mid, or high doses of the gene therapy. This approach allows careful monitoring of safety and response to increasing doses over time. Throughout the trial, participants will be closely monitored for treatment-related side effects over a period of up to 60 months. Researchers will assess changes in retinal health using imaging techniques like short wavelength fundus autofluorescence, microperimetry to measure macular sensitivity, and visual acuity tests. The study will also track changes in the area of retinal atrophy and ellipsoid zone loss to evaluate the therapy's impact on Stargardt disease progression.
CONDITIONS
Brief Title
A Study of AAVB-039 in Participants With Stargardt Disease (STGD1)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Molecular diagnosis of Stargardt disease due to ABCA4 mutation
- Willingness to adhere to protocol per informed consent
You will not qualify if you...
- 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
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 60 months
Participants receive a single subretinal administration of AAVB-039 at one of several dose levels depending on cohort assignment.
Regular visits for safety and efficacy assessments over 60 months
Trial Site Locations
Total: 7 locations
1
Associated Retina Consultants
Phoenix, Arizona, United States, 85020
Actively Recruiting
2
Retina Vitreous Associates Medical Group
Los Angeles, California, United States, 90211
Actively Recruiting
3
Mayo Clinic
Rochester, Minnesota, United States, 55905
Actively Recruiting
4
Retina Consultants of Texas
Bellaire, Texas, United States, 77401
Actively Recruiting
5
Retina Foundation of the Southwest
Dallas, Texas, United States, 75231
Actively Recruiting
6
Moorfields Eye Hospital NHS Foundation Trust
London, England, United Kingdom, EC1V 2PD
Actively Recruiting
7
The Retina Clinic
London, England, United Kingdom, W1G 9AX
Actively Recruiting
Research Team
A
AAVantgarde Clinical Operations Lead
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
4
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