A Study of AAVB-039 in Participants With Stargardt Disease (STGD1)
ConditionStargardt Disease
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age8-55
SponsorAAVantgarde Bio Srl
About this trial
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 criteria
Qualifiers
Molecular diagnosis of Stargardt disease due to ABCA4 mutation
Willingness to adhere to protocol per informed consent
Disqualifiers
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
Trial design
Treatments tested in this trial
- AAVB-039
Treatment groups
75 Participants
are divided into 4 treatment groupsSponsors and collaborators
AAVantgarde Bio Srl
Lead sponsor
AAVantgarde Bio UK Ltd
Collaborator
Source ClinicalTrials.gov