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)
Status: Recruiting
Location: See all (4) locations...
Intervention Type: Biological
Study Type: Interventional
Study Phase: Phase 1/Phase 2
SUMMARY
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.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 8
Maximum Age: 55
Healthy Volunteers: f
View:
• Molecular diagnosis of Stargardt disease due to ABCA4 mutation
• Willingness to adhere to protocol per informed consent
Locations
United States
Texas
Retina Consultants of Texas
RECRUITING
Bellaire
Retina Foundation of the Southwest
RECRUITING
Dallas
Other Locations
United Kingdom
Moorfields Eye Hospital NHS Foundation Trust
RECRUITING
London
The Retina Clinic
RECRUITING
London
Contact Information
Primary
AAVantgarde Clinical Operations Lead
clinicaltrials@aavantgarde.com
+448000465680
Time Frame
Start Date: 2025-09-29
Estimated Completion Date: 2032-07
Participants
Target number of participants: 75
Treatments
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
Related Therapeutic Areas
Sponsors
Leads: AAVantgarde Bio Srl
Collaborators: AAVantgarde Bio UK Ltd