EUCTR2009-017346-32-IT进行中（未招募）1 期
A PHASE I/II CLINICAL TRIAL OF HAEMATOPOIETIC STEM CELL GENE THERAPY FOR THE WISKOTT-ALDRICH SYNDROME - TIGET-WAS
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 8
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
- •1) Diagnosis of WAS defined by genetic mutation and at least one of the following criteria: -severe WAS mutation -absent WASP expression -severe clinical score (Zhu clinical score >= 3); 2) No HLA-identical sibling donor; and 3.1) Negative search for a matched unrelated donor (10/10) or an adequate unrelated cord blood donor (5-6/6) within 4-6 months; or 3.2) Patients of > 5 years of age who are not candidate to unrelated allogeneic transplant based on clinical conditions and 4) Parental/guardian/patient signed informed consent
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 1
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Patients who meet any of the following criteria will be excluded from study admission: 1) Patients positive for HIV-infection 2) Patients affected by neoplasia 3) Patients with cytogenetic alterations typical of MDS/AML 4) Patients with end-organ functions or any other severe disease which, in the judgement of the investigator, would make the patient inappropriate for entry into this study 5) Patients who underwent an allogeneic haematopoietic stem cell transplantation in the previous 6 months 6) Patients who underwent an allogeneic haematopoietic stem cell transplantation with evidence of residual cells of donor origin
研究者
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