A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa using AAV8

Phase 1

• Conditions: X-Linked retinitis pigmentosa (XLRP); MedDRA version: 20.0Level: PTClassification code 10038914Term: Retinitis pigmentosaSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Eye Diseases [C11]

• Registration Number: EUCTR2016-003852-60-GB
• Lead Sponsor: ightstaRx Limited

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2016-11-09
• Last Update Posted: 3 November 2020

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: Male
• Target Recruitment: 63

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

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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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: <br>The primary safety endpoints are the incidence of dose-limiting toxicities (DLTs), and treatment-emergent adverse events (TEAEs) over a 24-month period. <br><br>Part II:<br>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. <br>Part II:<br>Microperimetry will be assessed at regular intervals throughout the study.