A Dose Escalation (Phase 1), and Dose Expansion (Phase 2/3) Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using an Adeno-Associated Viral Vector (AAV8) Encoding Retinitis Pigmentosa GTPase Regulator (RPGR) - XIRIUS Study

ightstaRx Limited0 个研究点目标入组 63 人开始时间: 2016年11月9日最近更新: 2020年11月3日

适应症

试验速览

暂无简介。

•Subjects are eligible for study participation if they meet all of the following inclusion criteria:
•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)
•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

•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

发起方

ightstaRx Limited