AAVB-039 gene therapy for Stargardt disease
Official title: An Open-label, Multicenter, Two Part, Ascending Dose Followed by a Controlled Trial to Assess the Safety and Efficacy of a Subretinal Administration of AAVB-039 in Participants With Stargardt Disease (STGD1) (CELESTE)
Run by AAVantgarde Bio Srl · NCT07161544 · Data updated 2026-11-12
This study will test a single subretinal injection of AAVB-039 gene therapy in people with Stargardt disease caused by two ABCA4 mutations to see if it is safe and shows early benefit.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Ages | 8 Years to 55 Years |
| Sex | All |
| Enrollment | 75 (estimated) |
| Sponsor | AAVantgarde Bio Srl Industry-sponsored |
| Locations | 11 sites (Phoenix, Arizona and 10 other locations) |
| Status | Recruiting |
| Started | September 29, 2025 |
| Main results expected | July 2032 |
| Study ends | July 2032 |
| Registry record updated | September 30, 2026 |
| Trial ID | NCT07161544 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
People with a confirmed molecular diagnosis of Stargardt disease caused by biallelic ABCA4 mutations who can undergo subretinal surgery and follow study procedures are ideal candidates.
✕Probably not a fit
Patients with other retinal diseases, significant ocular or systemic comorbidities, prior gene or cell therapy, or inability to undergo subretinal surgery are unlikely to receive benefit from this intervention.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
AAVB-039 is an AAV-based gene therapy given as a single subretinal injection in a Phase 1/2 protocol for participants with Stargardt disease due to biallelic ABCA4 mutations. The trial's primary focus is on safety and tolerability after one administration, with initial efficacy measured by visual function tests and retinal imaging. Eligible participants must have a confirmed molecular diagnosis and be suitable candidates for subretinal surgery, while those with complicating ocular or systemic disease or prior gene/cell therapy are excluded. The study is being run at multiple U.S. retinal centers to provide specialized surgical care and close follow-up.
Why it matters
Potential benefit: If successful, AAVB-039 could slow or stabilize vision loss in people with ABCA4-related Stargardt disease.
How similar studies have gone: Previous gene therapy attempts targeting ABCA4 have been challenging because of the gene's large size and have shown mixed early-phase results, so this approach remains largely experimental.
Who can join: the full rules
Show full inclusion / exclusion criteria
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
Where it's running
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Associated Retina Consultants
Phoenix, Arizona, United States Recruiting -
Children's Hospital Los Angeles
Los Angeles, California, United States Recruiting -
Retina Vitreous Associates Medical Group
Los Angeles, California, United States Recruiting -
University of Florida Health Science Center
Jacksonville, Florida, United States Recruiting -
Mayo Clinic
Rochester, Minnesota, United States Recruiting -
Retina Consultants of Texas
Bellaire, Texas, United States Recruiting
Show 5 more locations
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Retina Foundation of the Southwest
Dallas, Texas, United States Recruiting -
University of Utah
Salt Lake City, Utah, United States Recruiting -
Oslo University Hospital
Oslo, Kirkeveien 166, Norway Recruiting -
Moorfields Eye Hospital NHS Foundation Trust
London, England, United Kingdom Recruiting -
The Retina Clinic
London, England, United Kingdom Recruiting
Study contacts
- Study coordinator: AAVantgarde Clinical Operations Lead
- Email: clinicaltrials@aavantgarde.com
- Phone: +448000465680
Similar studies
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This will test whether a single injection of RTx-021 into one eye is safe and may help people aged 16 and older with Stargardt disease.
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How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.