2024-511411-25-00招募中3 期
Phase 3 Follow-up Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 31
- 试验地点
- 8
- 主要终点
- Efficacy: Change from baseline in binocular vision-guided mobility assessment (VMA) after bilateral subretinal delivery of AAV5-hRKp.RPGR Adverse events Laboratory Assessment
研究概览
简要总结
To assess the long-term safety and tolerability of AAV5-hRKp.RPGR in individuals with RPGR-XLRP
To assess the long-term efficacy of treatment with AAV5-hRKp.RPGR in individuals with RPGR-XLRP based on assessments of functional vision as measured by vision-guided mobility assessment (VMA).
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 0 years 至 64 years（0-17 Years, 18-64 Years）
- 接受健康志愿者
- 是
入选标准
- •1.Previously completed participation in Study MGT-RPGR-
- •2.Must reconfirm that he or she understands the purpose of, and procedures required for, the study and is willing to participate in the study.
排除标准
- •There are no specific exclusion criteria.
结局指标
主要结局
Efficacy: Change from baseline in binocular vision-guided mobility assessment (VMA) after bilateral subretinal delivery of AAV5-hRKp.RPGR Adverse events Laboratory Assessment
Efficacy: Change from baseline in binocular vision-guided mobility assessment (VMA) after bilateral subretinal delivery of AAV5-hRKp.RPGR Adverse events Laboratory Assessment
次要结局
未报告次要终点
研究者
CTIS Point of Contact
Scientific
Janssen Cilag International
研究点 (8)
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