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

AAVantgarde Bio Srl

Status and phase: Enrolling; Phase 2; Phase 1

Conditions: Usher Syndrome, Type 1B

Treatments: Biological: AAVB-081

Study type: Interventional

Funder types: Industry

Identifiers

NCT06591793

081-101

Details and patient eligibility

About

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.

Enrollment: 15 estimated patients

Sex: All

Ages: 18 to 50 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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15 participants in 3 patient groups

Cohort 1

Experimental group

Description:

AAVB-081 dose level 1

Treatment:

Biological: AAVB-081

Cohort 2

Experimental group

Description:

AAVB-081 dose level 2

Treatment:

Biological: AAVB-081

Cohort 3

Experimental group

Description:

AAVB-081 dose level 3

Treatment:

Biological: AAVB-081