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-081 (biological)
- How long the study runs
- Study runs about 60 months (dates as stated)
- About the drug or intervention
- AAVB-081 — biological: Single subretinal administration
- Patient visit burden
- Not specified by the sponsor
In plain English
This study is testing a treatment called AAVB-081, given as an injection under the retina (the light-sensitive layer at the back of the eye), in people with Usher syndrome type 1B — a condition caused by changes in a gene called MYO7A. Usher syndrome type 1B can cause retinitis pigmentosa, an eye condition that gradually damages vision. The study is run by AAVantgarde Bio Srl.
Who can take part
- A genetic test confirming Usher syndrome type 1B caused by a change (mutation) in the MYO7A gene
- Being willing to follow the study plan, as agreed when giving consent
Who may not be able to
- Not being willing or able to meet the study's requirements
- Taking part in a clinical study of an investigational product in the past 6 months
- Having previously taken part in another gene therapy trial
- Having any condition that would make surgery under the retina unsafe or impossible
- Having other eye or general health conditions that could complicate the study
What taking part involves
- • A one-off injection of AAVB-081 under the retina, which requires eye surgery. Not stated — ask the trial team about any other parts of the treatment.
Time commitment: Not stated — ask the trial team about how many visits are needed, how long the study lasts, and what taking part involves.
Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.
- Type of study
- Testing a treatment
- Ages
- 18 Years to 50 Years
- Who
- All
- Number of participants
- 15
- Started
- 2024-07-02
- Last checked
- 2025-05
Plain English Summary
What is this study?
- • Testing a new treatment for usher syndrome, type 1b
- • Phase1/Phase2 - 15 participants
- • The purpose of the 081-101 study is to evaluate the safety and tolerability of a single subretinal injection of AAVB-081 in USH1B patients with retinitis pigmentosa due to a mutation in the MYO7A gene
Who can take part?
- • Ages 18 Years to 50 Years
- • Diagnosed with usher syndrome, type 1b
Where?
- • London - Moorfields Eye Hospital
- • London - Retina Clinic London
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
The purpose of the 081-101 study is to evaluate the safety and tolerability of a single subretinal injection of AAVB-081 in USH1B patients with retinitis pigmentosa due to a mutation in the MYO7A gene. The study will also assess the initial efficacy following AAVB-081 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: 18 Years - 50 Years
- Who can join: All genders
What the study is looking for
- ✓Molecular diagnosis of USB1B due to MYO7A 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 an Investigation Product in the past 6 months
- ✗Previous participation in another Gene 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
London, United Kingdom
Research Portfolio Managers- Daniela Narvaez, Anika Kadchha, Abi Chandrakumar, Xin Liu
moorfields.resadmin@nhs.net0207 566 2036Retina Clinic London
London, United Kingdom
How to Get in Touch
Clinical Operations Manager
Sponsor contactCONTACT