NCT04671433已完成3 期
Phase 3 Randomized, Controlled Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 105
- 试验地点
- 51
- 主要终点
- Change From Baseline to Week 52 in Vision-guided Mobility Assessment (VMA) as Measured by the Ability of the Participant to Navigate Through a VMA Maze
研究概览
简要总结
A clinical trial of AAV5-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)
盲法说明
No intervention - deferred treatment group (Bilateral, subretinal administration of AAV5-RPGR to be administered in the follow-up study)
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
- •Has had ocular surgery within 3 months prior to screening or is anticipated to require ocular surgery within 6 months after the study intervention administration
- •Any investigational ocular treatment or any other ocular treatment that could confound the interpretation of the efficacy results or affect participant compliance with the visit schedule
- •Has undergone prior retinal surgery involving the macula, macular laser photocoagulation, external-beam radiation therapy, transpupillary thermotherapy, glaucoma filtration surgery or corneal surgery
研究组 & 干预措施
Experimental Immediate Treatment
Experimental
Low dose.
干预措施: Genetic: AAV5-hRKp.RPGR (Biological)
Experimental - Immediate Treatment
Experimental
Intermediate dose.
干预措施: Genetic: AAV5-hRKp.RPGR (Biological)
Deferred Treatment
Other
Deferred Treatment
干预措施: Genetic: AAV5-hRKp.RPGR (Biological)
结局指标
主要结局
Change From Baseline to Week 52 in Vision-guided Mobility Assessment (VMA) as Measured by the Ability of the Participant to Navigate Through a VMA Maze
时间窗: From Baseline to 52 Weeks
Change from baseline to Week 52 in VMA as measured by the ability of the participant to navigate through a VMA maze.
次要结局
- Change in Retinal Function as Assessed by Pointwise Response in Full Visual Field at Week 52(From Baseline to Week 52)
- Change From Baseline in the Modified Low Luminance Questionnaire (mLLQ) Extreme Lighting Domain score at Week 52(From Baseline to Week 52)
- Change From Baseline in Mean Retinal Sensitivity of Worse-seeing Eye Within the Central 10 Degrees Excluding Scotoma in Static Perimetry (MRS10) at Week 52(From Baseline to Week 52)
- Change From Baseline in Mean Retinal Sensitivity Within the Central 10 Degrees Excluding Scotoma (Mean Retinal Sensitivity Within the Central 10 Degree Excluding Scotoma in Static Perimetry [MRS10]) in Static Perimetry at Week 52(From Baseline to Week 52)
- Change in Retinal Function as Assessed by Pointwise Response in Worse-seeing Eye in the Central 30 Degrees Visual Field at Week 52(From Baseline to Week 52)
- Change From Baseline in Visual Function as Assessed by Low Luminance Visual Acuity Using the ETDRS Chart Letter Score in Worse-seeing Eye at Week 52(From Baseline to Week 52)
- Change in Retinal Function as Assessed by Pointwise Response in Worse-seeing Eye in Full Visual Field at Week 52(From Baseline to Week 52)
- Change From Baseline in Retinal Function as Assessed by Mean Retinal Sensitivity Within the Full Visual Field (MRS90) in Static Perimetry at Week 52(From Baseline to Week 52)
- Change in Functional Vision by Using Vision-guided Mobility Assessment (VMA) Response in the "Worse-seeing Eye" at Week 52(From Baseline to Week 52)
- Number of Participants With Abnormalities in Laboratory Assessments(Day 1 - 52 Weeks)
- Change in Retinal Function as Assessed by Pointwise Response in the Central 30 Degrees Visual Field at Week 52(From Baseline to Week 52)
- Change From Baseline in Visual Function as Assessed by monocular Best Corrected Visual Acuity (BCVA) Using the ETDRS Chart Letter Score at Week 52(From Baseline to Week 52)
- Change From Baseline in Visual Function as Assessed by Monocular Low Luminance Visual Acuity Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter score at Week 52(From Baseline to Week 52)
- Number of Participants with Ocular and Non-ocular Adverse Events(Day 1 - Week 52)
研究者
研究点 (51)
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