Long-term Follow-up Study of Participants Following an Open Label, Multi-Centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector (AAV5- hRKp.RPGR) for Gene Therapy of Adults and Children With X-linked Retinitis Pigmentosa Owing to Defects in Retinitis Pigmentosa GTPase Regulator (RPGR)

Janssen Research & Development, LLC5 个研究点分布在 2 个国家目标入组 42 人2017年7月31日

适应症X-Linked Retinitis Pigmentosa

干预措施AAV5-hRKp.RPGR

概览

阶段: 不适用
干预措施: AAV5-hRKp.RPGR
疾病 / 适应症: X-Linked Retinitis Pigmentosa
发起方: Janssen Research & Development, LLC
入组人数: 42
试验地点: 5
主要终点: Assess the Longer-term Safety of AAV5-hRKp.RPGR Administered to Participants
状态: 已完成
最后更新: 3个月前

概览研究者入排标准研究组 & 干预措施结局指标研究点相似试验

概览

简要总结

This is a long-term follow-up study assessing safety of patients for up to 60 months following advanced therapy investigational medicinal product (ATIMP) AAV5-hRKp.RPGR vector in participants with XLRP caused by mutations in RPGR.

注册库

clinicaltrials.gov

开始日期

2017年7月31日

结束日期

2025年9月17日

最后更新

3个月前

研究类型

Observational

性别

Male

研究者

发起方

Janssen Research & Development, LLC

责任方

Sponsor

入排标准

入选标准

•Received AAV5-hRKp.RPGR in the MGT009 Study

排除标准

•Unwilling or unable to meet with the requirements of the study

研究组 & 干预措施

Follow up cohort

no intervention follow up study

干预措施: AAV5-hRKp.RPGR

结局指标

主要结局

Assess the Longer-term Safety of AAV5-hRKp.RPGR Administered to Participants

时间窗: 60 Months

Longer-term safety of subretinal AAV5-hRKp.RPGR administered to participants will be reported.

次要结局

Change in Functional Vision of Low Luminance Questionnaire (LLQ) Domain Scores in Patient Reported Outcome (PRO)(Up to 60 Months)
Change in Best Corrected Visual Acuity (BCVA) Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score(Up to 60 Months)
Change in Low Luminance Visual Acuity (LLVA) as Assessed by ETDRS Chart Under Low Luminance Conditions(Up to 60 Months)
Change in Functional Vision of Walk Time in Vision-guided Mobility Assessment (VMA)(Up to 60 Months)
Change in Mean Retinal Sensitivity Within the Central 10 Degree Visual Field Excluding Scotoma (MRS10) in Static Perimetry(Up to 60 months)
Change In Retinal Sensitivity by Pointwise Comparison of Data in Static Perimetry Within the Full Visual Field(Up to 60 months)
Change In Retinal Sensitivity by Pointwise Comparison of Data in Static Perimetry Within the Central 30 Degrees Visual Field(Up to 60 months)
Change in Mean Retinal Sensitivity Within the Full Visual Field Excluding Scotoma (MRS90) in Static Perimetry(Up to 60 months)