EUCTR2019-002343-14-NL进行中（未招募）1 期
PHASE I/II CLINICAL TRIAL OF AUTOLOGOUS HEMATOPOIETIC STEM CELL GENE THERAPY FOR RAG1-DEFICIENT SEVERE COMBINED IMMUNODEFICIENCY
eiden University Medical Center0 个研究点目标入组 5 人开始时间: 2019年7月26日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 5
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
- All
入选标准
- •1.RAG1 deficient SCID as confirmed by genetic analysis
- •2.Peripheral blood T cells < 300/µL and/or naïve T cells < 1/µL
- •3.Age < 2 years
- •4.Age at least 8 weeks by the time of busulfan administration
- •5.lack of an available HLA-matched donor (HLA-identical sibling or 10/10 (A, B, C, DR, DQ) allele-matched (un)related donor)
- •6.signed informed consent (parental or guardian)
- •7.able to return to the study centre for follow-up (per protocol) during the 2-year study and the 15-year long-term off study review
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 5
- •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.availability of a HLA-matched donor (HLA-identical sibling or 10/10 (A, B, C, DR, DQ) allele-matched (un)related donor)
- •2.RAG 1 deficiency with peripheral blood T cells > 300/µL and/or naïve T cells > 1/µL
- •3.Omenn syndrome
- •4.Previous allogeneic HSCT
- •5.Significant organ dysfunction/co-morbidity (including but not limited to the ones listed below)
- •a.Mechanical ventilation
- •b.Shortening fraction on echocardiogram <25%
- •c.Renal failure defined as dialysis dependence
- •d.Uncontrolled seizure disorder
- •6.any other medical condition which, in the opinion of the treating physician, would in-terfere with the good conduction of the clinical trial (e.g. contraindications for stem cell harvest or administration of conditioning medication)
- •7.HIV infection or HTLV infection
研究者
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