Usher Syndrome, Type 1B
Conditions
Keywords
Usher Syndrome, MYO7A, USH1B, Retinitis Pigmentosa
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.
Interventions
BIOLOGICALAAVB-081
Single subretinal administration
Sponsors
AAVantgarde Bio Srl
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 50 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To measure the number and severity of treatment related adverse events following treatment with AAVB-081 | 61 months |
Secondary
| Measure | 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 |
Countries
Italy, United Kingdom
Contacts
Primary ContactClinical Operations Manager
Outcome results
None listed