Phase 3 Follow-up Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 97
- 试验地点
- 38
- 主要终点
- Number of Participants With Abnormalities in Laboratory Assessments
研究概览
简要总结
A clinical trial of AAV5-hRKp.RPGR vector for participants with X-linked retinitis pigmentosa (XLRP)
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Single (Outcomes Assessor)
入排标准
- 年龄范围
- 3 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female, 3 years of age or older, has XLRP confirmed by a retinal specialist and has a predicted disease-causing sequence variant in RPGR confirmed by an accredited laboratory.
排除标准
- 未提供
研究组 & 干预措施
Already Treated in MGT-RPGR-021
Already treated
干预措施: Genetic: AAV5-hRKp.RPGR Low Dose (Biological)
Deferred Treatment From MGT-RPGR-021 Low Dose
Deferred treatment
干预措施: Genetic: AAV5-hRKp.RPGR Low Dose (Biological)
Deferred Treatment From MGT-RPGR-021 of Intermediate Dose
Deferred treatment
干预措施: Genetic: AAV5-hRKp.RPGR Intermediate Dose (Biological)
Already Treated in MGT-RPGR-021
Already treated
干预措施: Genetic: AAV5-hRKp.RPGR Intermediate Dose (Biological)
结局指标
主要结局
Number of Participants With Abnormalities in Laboratory Assessments
时间窗: Day 1 - Month 60
Number of participants with abnormalities in laboratory assessments will be assessed.
Number of Participants with Ocular and Non-ocular Adverse Events
时间窗: Day 1 - Month 60
Number of participants with ocular and non-ocular adverse events will be assessed.
Number of Participants With Abnormalities in Laboratory Assessments
时间窗: Day 1 - Month 60
Number of participants with abnormalities in laboratory assessments will be assessed.
Change From Baseline in Low Luminance Visual Acuity by ETDRS Chart Letter Score in Monocular Assessment
时间窗: From Baseline- Month 60
Change from baseline in low luminance visual acuity by ETDRS chart letter score in monocular assessment will be assessed.
Change From Baseline in Best Corrected Visual Acuity (BCVA) by Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score in Monocular Assessment
时间窗: From Baseline - Month 60
Change from baseline in BCVA by ETDRS chart letter score in monocular assessment will be assessed.
次要结局
未报告次要终点