Study of Subretinally Injected AAVB-081 in Patients With... | NCT06591793

Studies
Study of Subretinally Injected AAVB-081 in Patients With Usher Syndrome Type IB (USH1B) Retinitis Pigmentosa

NCT06591793

Sponsor

AAVantgarde Bio Srl

Status

Recruiting

Last Update Posted

May 2, 2025Actual

Enrollment

15Estimated

Phase

Phase 1Phase 2

Conditions

Usher Syndrome, Type 1B

Interventions

AAVB-081

Countries

Italy
United Kingdom

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

May 2025

Overall Recruitment Status or Expanded Access Status

Recruiting

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jul 2, 2024Actual

Primary Completion Date

Jul 2025Estimated

Completion Date

Jul 2029Estimated

First Submitted Date

Aug 16, 2024

First Submission Date that Met QC Criteria

Sep 9, 2024

First Posted Date

Sep 19, 2024Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

May 1, 2025

Last Update Posted Date

May 2, 2025Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Yes

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

FDAAA801 Violation

Not provided

Description Module

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.

Detailed Description

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 1Phase 2

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

15Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Cohort 1	Experimental	AAVB-081 dose level 1	Biological: AAVB-081
Cohort 2	Experimental	AAVB-081 dose level 2	Biological: AAVB-081
Cohort 3	Experimental	AAVB-081 dose level 3	Biological: AAVB-081

Interventions

Name	Type	Description	Arm Group Labels	Other Names
AAVB-081	Biological	Single subretinal administration	Cohort 1 Cohort 2 Cohort 3

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
To measure the number and severity of treatment related adverse events following treatment with AAVB-081		61 months

Secondary Outcomes

Measure	Description	Time Frame
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

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Molecular diagnosis of USB1B due to MYO7A 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 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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

50 Years

Standard Ages

Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Name	Role	Phone	Extension	Email
Clinical Operations Manager	Contact	+448000465680		clinicaltrials@aavantgarde.com

Overall Officials

Not provided

Locations

Facility	Status	City	State	ZIP	Country	Contacts
University of Campania Luigi Vanvitelli	Recruiting	Naples			Italy

IPD Sharing Statement Module

Plan to Share IPD

Description

Not provided

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided

Condition Browse Module

MeSH Terms

ID	Term
C536485	Usher syndrome, type 1B
D052245	Usher Syndromes
D012174	Retinitis Pigmentosa

Ancestor Terms

ID	Term
D054062	Deaf-Blind Disorders
D003638	Deafness
D034381	Hearing Loss
D006311	Hearing Disorders

Browse Leaves

Not provided

Browse Branches

Not provided