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

ArmDescriptionAssigned Intervention
Experimental
Cohort 1 		AAVB-039 dose level 1 (low dose)
  • Biological: AAVB-039
    Single subretinal administration
Experimental
Cohort 2 		AAVB-039 dose level 2 (mid dose)
  • Biological: AAVB-039
    Single subretinal administration
Experimental
Cohort 3 		AAVB-039 dose level 3 (high dose)
  • Biological: AAVB-039
    Single subretinal administration
Experimental
Cohorts 4-6 		AAVB-039 dose level 1, 2 or 3
  • Biological: AAVB-039
    Single subretinal administration

Recruiting Locations

Retina Consultants of Texas
Bellaire 4673353, Texas 4736286 77401

More Details

Status
Recruiting
Sponsor
AAVantgarde Bio Srl

Study Contact

AAVantgarde Clinical Operations Lead
+448000465680
clinicaltrials@aavantgarde.com