Usher Syndrome, Type 1BPhase 1/2NCT06591793
Study of Subretinally Injected AAVB-081 in Patients With Usher Syndrome Type IB (USH1B) Retinitis Pigmentosa
A Phase 1/2 study testing AAVB-081 in people with Usher Syndrome, Type 1B, run by AAVantgarde Bio Srl at 3 sites in 2 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 50 years
- Conditions the study is for
- Usher Syndrome, Type 1B
- What is being tested?
- AAVB-081
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 3 sitesin 2 countries
- Study dates
- 2 Jul 2024to Jul 2029
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 50 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Molecular diagnosis of USB1B due to MYO7A mutation
- Willingness to adhere to protocol per informed consent
Who cannot join
- 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
Trial locations and contacts
3 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
3 sites in 2 countries
United Kingdom2 sites, 2 recruiting
- Recruiting
Moorfields Eye Hospital
London
Michel Michaelides
- Recruiting
Retina Clinic London
London
Paulo E Stanga
Italy1 site, 1 recruiting
- Recruiting
University of Campania Luigi Vanvitelli
Naples
Francesca Simonelli
About this study
Official title: A Phase 1/2 Multicenter, Open-label, Dose Escalation, Safety and Efficacy Study of Subretinal Administration of Dual AAV8.MYO7A, AAVB-081 in Subjects With Usher Syndrome Type IB (USH1B) Retinitis Pigmentosa
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.
- Conditions
- Usher Syndrome, Type 1B
- Being tested
- AAVB-081
- Keywords
- Usher Syndrome, MYO7A, USH1B, Retinitis Pigmentosa
- Medical subject headings
- Usher syndrome, type 1B, Usher Syndromes, Retinitis Pigmentosa
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AAVB-081Biological
Single subretinal administration
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | AAVB-081 dose level 1 Receives: Biological: AAVB-081 |
| Cohort 2 | Experimental | AAVB-081 dose level 2 Receives: Biological: AAVB-081 |
| Cohort 3 | Experimental | AAVB-081 dose level 3 Receives: Biological: AAVB-081 |
What is being measured
The primary outcome is the main question the study is designed to answer.
To measure the number and severity of treatment related adverse events following treatment with AAVB-081
Measured: 61 months
Secondary outcomes (2)
Change from baseline in microperimetry following treatment with AAVB-081
Measured: 61 months
Change from baseline in static perimetry following treatment with AAVB-081
Measured: 61 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Sep 2024
- Study start2 Jul 2024
- Primary completionJul 2025 (estimated)
- Study completionJul 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Usher Syndrome, Type 1BStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- AAVantgarde Bio Srl
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06591793
- Sponsor's protocol ID
- 081-101
- Status last verified
- May 2025
- First submitted
- 16 Aug 2024
- Last updated
- 2 May 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2 Multicenter, Open-label, Dose Escalation, Safety and Efficacy Study of Subretinal Administration of Dual AAV8.MYO7A, AAVB-081 in Subjects With Usher Syndrome Type IB (USH1B) Retinitis Pigmentosa
- ClinicalTrials.gov ID
- NCT06591793
- Acronym / sponsor ID
- 081-101
- Conditions
- Usher Syndrome, Type 1B
- Keywords
- Usher Syndrome, MYO7A, USH1B, Retinitis Pigmentosa
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- AAVantgarde Bio SrlIndustry (pharma, biotech, devices)
- Study start
- 2 Jul 2024 (actual)
- Primary completion
- Jul 2025 (estimated)
- Study completion
- Jul 2029 (estimated)
- First posted
- 19 Sep 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Biological: AAVB-081AAVB-081 dose level 1 |
| Cohort 2 | Experimental | Biological: AAVB-081AAVB-081 dose level 2 |
| Cohort 3 | Experimental | Biological: AAVB-081AAVB-081 dose level 3 |
Who may qualify
- Ages
- 18 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (2 listed)
- Molecular diagnosis of USB1B due to MYO7A mutation
- Willingness to adhere to protocol per informed consent
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AAVB-081Single subretinal administration | Biological | Cohort 1, Cohort 2, Cohort 3 |
Primary outcome
To measure the number and severity of treatment related adverse events following treatment with AAVB-081
Time frame: 61 months
2 secondary outcomes
Change from baseline in microperimetry following treatment with AAVB-081
Time frame: 61 months
Change from baseline in static perimetry following treatment with AAVB-081
Time frame: 61 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 May 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
3 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing AAVB-081 in people with Usher Syndrome, Type 1B, run by AAVantgarde Bio Srl at 3 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 2 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United Kingdom, Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Clinical Operations Manager (+448000465680, clinicaltrials@aavantgarde.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To measure the number and severity of treatment related adverse events following treatment with AAVB-081 (time frame: 61 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 May 2025.