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临床试验/NCT00927134
NCT00927134已完成1 期

Phase I/II Gene Therapy Study for X-linked Chronic Granulomatous Disease in Children

University of Zurich2 个研究点 分布在 1 个国家目标入组 2 人开始时间: 2004年6月1日最近更新:
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适应症

试验速览

阶段
1 期
状态
已完成
入组人数
2
试验地点
2
主要终点
eradication of pre-existing therapy refractory bacterial and/or fungal infections
研究概览研究设计入排标准结局指标研究者研究点记录与标识符相似试验

研究概览

简要总结

The aim of the study is to evaluate the side effects and risks after infusion of retroviral gene corrected autologous CD34+ cells of the peripheral blood of chemotherapy conditioned (busulphan) children with chronic granulomatous disease (CGD). Also gene corrected and functional active granulocytes in the peripheral blood and the engraftment in the bone marrow of the patients will be monitored an documented.

研究设计

研究类型
Interventional
分配方式
Non Randomized
干预模型
Single Group
主要目的
Treatment
盲法
None

入排标准

年龄范围
1 Year 至 18 Years（Child, Adult）
性别
Male
接受健康志愿者
否

入选标准

  • •x-linked Chronic Granulomatous Disease
  • •history of life-threatening severe infections
  • •no HLA-matched related or unrelated donor
  • •therapy resistent life threatening infections/organ dysfunction
  • •no other treatment options e.g. HSCT

排除标准

  • •> 18 years of age
  • •HIV infection
  • •life expectancy > 2 years
  • •infections treatable by conventional therapy (antibiotics, antimycotics, allogeneic granulocytes)

结局指标

主要结局

eradication of pre-existing therapy refractory bacterial and/or fungal infections

时间窗: 6 months

次要结局

  • Reconstitution of ROS production by peripheral blood cells(1 month)

研究者

申办方类型
Other
责任方
Sponsor

研究点 (2)

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