NCT00778882撤回1 期
An Open-label, Uncontrolled, Single Center, Phase I/II Trial to Assess the Safety and Efficacy of Autologous Hematopoietic Stem Cells Transduced With MT-gp91 Retroviral Vector in gp91 Defective Chronic Granulomatous Disease Patients
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 撤回
- 入组人数
- 2
- 试验地点
- 1
- 主要终点
- The incidence of adverse events through 1 year
研究概览
简要总结
The purpose of this study is to evaluate the safety and efficacy of administration of autologous hematopoietic stem cells transduced with MT-gp91 retroviral vector for patients with X-linked chronic granulomatous disease.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •gp91 defective male patients with chronic granulomatous disease: confirmed by DHR
- •Weigh greater than or equal to 15 kg
- •History of severe infections: more than 2 times
- •Performance status: ECOG 0-2
- •Patients must be free of significant functional deficits in major organs, but the following eligibility criteria may be modified in individual cases.
- •Heart: a shortening fraction > 28%; QTc interval < 0.44
- •Liver: total bilirubin < 2 × upper limit of normal; ALT < 3 × upper limit of normal; AST < 3 x upper limit of normal
- •Kidney: creatine < 2 x normal
- •Blood: WBC > 2,500/uL; platelet > 100,000/uL; hematocrit > 26%
- •Written informed consent obtained from patient (or guardian if patients age < 19)
排除标准
- •Presence of a HLA-matched sibling for stem cell donation
- •Evidence or history of malignant tumor
- •Presence of a severe infection
- •Presence of an active tuberculosis
- •Uncorrectable electrolyte, Ca, P
- •Unable to comply with the protocol or to cooperate fully with the Investigator or site personnel
研究组 & 干预措施
VM106
Experimental
干预措施: VM106 (Drug)
结局指标
主要结局
The incidence of adverse events through 1 year
时间窗: 1 year
次要结局
- RCR, insertional mutagenesis, immune response against normal gp91 protein(1 year)
- Safety and efficacy of fludarabine/busulfan conditioning(1 year)
- Functional reconstitution of respiratory burst(1 year)
- Presence of vector-positive cells(1 year)
研究者
研究点 (1)
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