At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- AAVB-039 (biological)
- How long the study runs
- Study runs about 82 months (dates as stated)
- About the drug or intervention
- AAVB-039 — biological: Single subretinal administration
- Patient visit burden
- Not specified by the sponsor
- Type of study
- Testing a treatment
- Ages
- 8 Years to 55 Years
- Who
- All
- Number of participants
- 75
- Started
- 2025-09-29
- Last checked
- 2026-09
Plain English Summary
What is this study?
- • Testing a new treatment for stargardt disease
- • Phase1/Phase2 - 75 participants
- • 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
Who can take part?
- • Ages 8 Years to 55 Years
- • Diagnosed with stargardt disease
Where?
- • London - Moorfields Eye Hospital NHS Foundation Trust
- • London - The Retina Clinic
This is a simplified summary. Always discuss with your doctor before making any decisions.
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.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 8 Years - 55 Years
- Who can join: All genders
What the study is looking for
- ✓Molecular diagnosis of Stargardt disease due to ABCA4 mutation
- ✓Willingness to adhere to protocol per agreement to take part
Who cannot take part
- ✗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
See the full criteria
Where Is This Study? (2 UK sites)
Moorfields Eye Hospital NHS Foundation Trust
London EC1V 2PD, United Kingdom
The Retina Clinic
London W1G 9AY, United Kingdom
How to Get in Touch
AAVantgarde Clinical Operations Lead
Sponsor contactCONTACT