EUCTR2007-000684-16-GB进行中（未招募）1 期
Gene therapy for SCID-X1 using a self-inactivating (SIN) gammaretroviral vector. - Gene therapy for SCID-X1
Great Ormond Street Hospital NHS Trust / University College London - Institute of Child Health0 个研究点目标入组 1 人开始时间: 2009年9月17日最近更新:
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 1
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- Male
入选标准
- •1a)No HLA identical (A,B,C,DR,DQ) family donor
- •b)No HLA identical unrelated donor available within 3 mths of diagnosis or
- •c)Patients whose underlying clinical problems and prognosis would be significantly compromised by chemotherapy conditioning (including persisting pneumonitis, protracted diarrhoea requiring parental nutrition, ongoing visceral viral infection (herpes viruses, HSV,VZV,CMV,EBV or adenovirus), systemic BCG infection, virus-induced lymphoproliferation.
- •2.Diagnosis of classical SCID-X1 based on immunophenotype (absent, or reduced numbers of non-functional T lymphocytes and confirmed by DNA sequencing (clinical genetics laboratory, GOSH)
- •3. Parental/guardian voluntary consent
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1.No available molecular diagnosis confirming SCID-X1
研究者
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Unknown
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