A Study of AAVB-039 in Participants With Stargardt Disease (STGD1)
Purpose
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.
Condition
- Stargardt Disease
Eligibility
- Eligible Ages
- Between 8 Years and 55 Years
- Eligible Sex
- All
- Accepts Healthy Volunteers
- No
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
- Phase
- Phase 1/Phase 2
- Study Type
- Interventional
- Allocation
- Non-Randomized
- Intervention Model
- Sequential Assignment
- Primary Purpose
- Treatment
- Masking
- None (Open Label)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
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 |
|
Recruiting Locations
Retina Vitreous Associates Medical Group
Los Angeles, California 90211
Los Angeles, California 90211
Mayo Clinic
Rochester, Minnesota 55905
Rochester, Minnesota 55905
Retina Consultants of Texas
Bellaire, Texas 77401
Bellaire, Texas 77401
Retina Foundation of the Southwest
Dallas, Texas 75231
Dallas, Texas 75231
More Details
- Status
- Recruiting
- Sponsor
- AAVantgarde Bio Srl