Study of Subretinally Injected AAVB-081 in Patients With Usher Syndrome Type IB (USH1B) Retinitis Pigmentosa
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
1 other identifier
interventional
15
2 countries
3
Brief 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_1
Started Jul 2024
Longer than P75 for phase_1
3 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
July 2, 2024
CompletedFirst Submitted
Initial submission to the registry
August 16, 2024
CompletedFirst Posted
Study publicly available on registry
September 19, 2024
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 1, 2025
CompletedStudy Completion
Last participant's last visit for all outcomes
July 1, 2029
ExpectedMay 2, 2025
May 1, 2025
12 months
August 16, 2024
May 1, 2025
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
To measure the number and severity of treatment related adverse events following treatment with AAVB-081
61 months
Secondary Outcomes (2)
Change from baseline in microperimetry following treatment with AAVB-081
61 months
Change from baseline in static perimetry following treatment with AAVB-081
61 months
Study Arms (3)
Cohort 1
EXPERIMENTALAAVB-081 dose level 1
Cohort 2
EXPERIMENTALAAVB-081 dose level 2
Cohort 3
EXPERIMENTALAAVB-081 dose level 3
Interventions
Eligibility Criteria
You may qualify if:
- Molecular diagnosis of USB1B due to MYO7A mutation
- Willingness to adhere to protocol per informed consent
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (3)
University of Campania Luigi Vanvitelli
Naples, Italy
Moorfields Eye Hospital
London, United Kingdom
Retina Clinic London
London, United Kingdom
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SEQUENTIAL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
August 16, 2024
First Posted
September 19, 2024
Study Start
July 2, 2024
Primary Completion
July 1, 2025
Study Completion (Estimated)
July 1, 2029
Last Updated
May 2, 2025
Record last verified: 2025-05
Data Sharing
- IPD Sharing
- Will not share