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临床试验/2024-511411-25-00
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

Janssen Cilag International8 个研究点 分布在 6 个国家目标入组 31 人开始时间: 2024年6月21日最近更新:
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

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

申办方类型
Pharmaceutical company
责任方
Principal Investigator
主要研究者

CTIS Point of Contact

Scientific

Janssen Cilag International

研究点 (8)

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