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 4-6 |
AAVB-039 dose level 1, 2 or 3 |
|
Recruiting Locations
Retina Consultants of Texas
Bellaire 4673353, Texas 4736286 77401
Bellaire 4673353, Texas 4736286 77401
More Details
- Status
- Recruiting
- Sponsor
- AAVantgarde Bio Srl