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)
AAVantgarde Bio Srl
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
- Age range
- 8–55 years
- Sex
- All
- 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
Interventions
- BiologicalAAVB-039
Single subretinal administration
Locations (6)
- Retina Vitreous Associates Medical GroupLos Angeles, California
- Mayo ClinicRochester, Minnesota
- Retina Consultants of TexasBellaire, Texas
- Retina Foundation of the SouthwestDallas, Texas
- Moorfields Eye Hospital NHS Foundation TrustLondon, England
- The Retina ClinicLondon, England