AAVB-039 gene therapy for Stargardt disease
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)
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 |
| Enrollment | 75 (estimated) |
| Ages | 8 Years to 55 Years |
| Sex | All |
| Sponsor | AAVantgarde Bio Srl Industry-sponsored |
| Locations | 6 sites (Los Angeles, California and 5 other locations) |
| Trial ID | NCT07161544 on ClinicalTrials.gov |
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.
Who should consider this trial
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.
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.
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 performed: 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.
Eligibility criteria
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 this trial is running
Los Angeles, California and 5 other locations
- Retina Vitreous Associates Medical Group — Los Angeles, California, United States (Recruiting)
- Mayo Clinic — Rochester, Minnesota, United States (Recruiting)
- Retina Consultants of Texas — Bellaire, Texas, United States (Recruiting)
- Retina Foundation of the Southwest — Dallas, Texas, United States (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
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.