X-Linked retinitis pigmentosa (XLRP) MedDRA version: 20.0 Level: PT Classification code 10038914 Term: Retinitis pigmentosa System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: AAV8-RPGR
Product Code: AAV8-RPGR
Pharmaceutical Form: Suspension for injection
CAS Number: 2246424-95-1
Current Sponsor code: AAV8-RPGR
Other descriptive name: AAV8-RPGR
Concentration t
Sponsors
NightstaRx Limited
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: Subjects are eligible for study participation if they meet all of the following inclusion criteria: Part I 1. Subject is willing and able to give informed consent for participation in the study 2. Are male, =18 years of age, and able to comply and adequately perform all study assessments 3. Have a genetically confirmed diagnosis of XLRP (with RPGR mutation) Part II 1. Subject / parent (if applicable) is willing and able to provide informed consent for participation in the study 2. Are male, =10 years of age, and able to comply and adequately perform all study assessments 3. Documentation of a pathogenic mutation in the RPGR gene Are the trial subjects under 18? yes Number of subjects for this age range: 20 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 43 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Parts 1 and II: Subjects are not eligible for study participation if they meet any of the following exclusion criteria. 1. Have a history of amblyopia in either eye 2. Are unwilling to use barrier contraception methods (if applicable), or abstain from sexual intercourse, for a period of 3 months following treatment with AAV8-RPGR 3. Have participated in another research study involving an investigational product in the past 12 weeks or received a gene/cell-based therapy at any time previously
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the safety, tolerability and efficacy of a single sub-retinal injection of AAV8-RPGR in subjects with XLRP;Secondary Objective: Not applicable;Primary end point(s): Part I: The primary safety endpoints are the incidence of dose-limiting toxicities (DLTs), and treatment-emergent adverse events (TEAEs) over a 24-month period. Part II: The primary efficacy endpoint is improvement from Baseline in microperimetry.;Timepoint(s) of evaluation of this end point: Part I: Incidence of dose-limiting toxicities (DLTs), and treatment-emergent adverse events (TEAEs) over a 24-month period. Part II: Microperimetry will be assessed at regular intervals throughout the study. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Part I: Secondary and Exploratory Endpoints: •Change from baseline in microperimetry at regular intervals throughout the study •Change from baseline in best-corrected visual acuity (BCVA) at regular intervals throughout the study Part II: Secondary Endpoints: • The safety endpoint is incidence of TEAEs over a 12-month period. • Change from baseline in microperimetry at regular intervals throughout the study. • Change from baseline in best-corrected visual acuity (BCVA) at regular intervals throughout the year. • Other secondary and exploratory ophthalmology endpoints are assessed at regular intervals throughout the study. ;Timepoint(s) of evaluation of this end point: Part I - Secondary and Exploratory Endpoints: •Microperimetry at regular intervals throughout the study •BCVA at regular intervals throughout the study •Other secondary and exploratory ophthalmology endpoints are assessed at regular intervals throughout the study. Part II - Secondary and Exploratory Endpoints: •Microperimetry at regular intervals throughout the study •BCVA at regular intervals throughout the study •Other secondary and exploratory ophthalmology endpoints are assessed at regular intervals throughout the study. | — |
Countries
United Kingdom, United States
Contacts
Public ContactClinical Operations Department
NightstaRx Limited
Outcome results
None listed