A Study of AAVB-039 in Participants With Stargardt Disease (STGD1)
Recruiting
Phase 1
Phase 2
Interventional Study
Stargardt Disease
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 8 – 55
- Sex
- Any
- Trial phase
- Phase 1/2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 75 (estimated)
- Sponsor
- AAVantgarde Bio Srl · Industry
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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.
Trial Locations
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Eligibility Criteria
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
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Contacts