Phase I/II Gene Therapy Study for X-Linked Chronic Granulomatous Disease

Johann Wolfgang Goethe University Hospital2 个研究点分布在 1 个国家目标入组 2 人开始时间: 2004年1月1日最近更新: 2007年11月28日

适应症

试验速览

阶段: 1 期
发起方
入组人数: 2
试验地点: 2
主要终点: safety, toxicity and feasibility

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)patients 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
盲法: None

入排标准

年龄范围: 18 Years 至 —（Adult, Older 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 non-related donor
•therapy resistent life threatening infections/organ dysfunction
•no other treatment options e.g. BMT

排除标准

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

结局指标

主要结局

safety, toxicity and feasibility

时间窗: 2 years

次要结局

Engraftment of gene corrected stem cells, functional reconstitution of respiratory burst, clinical benefit(2 years)

研究者

发起方

Johann Wolfgang Goethe University Hospital

申办方类型

Other

研究点 (2)

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