ISRCTN46087965
已完成
1 期
Phase I/II clinical trial of haematopoietic stem cell gene therapy for the Wiskott-Aldrich Syndrome
Genethon (France)0 个研究点目标入组 5 人2011年5月20日
概览
- 阶段
- 1 期
- 干预措施
- 未指定
- 疾病 / 适应症
- Wiskott-Aldrich Syndrome
- 发起方
- Genethon (France)
- 入组人数
- 5
- 状态
- 已完成
- 最后更新
- 8年前
概览
简要总结
2017 results in: https://www.ncbi.nlm.nih.gov/pubmed/28716862
研究者
入排标准
入选标准
- •1\. Males of all ages
- •2\. Severe WAS (clinical score 3 ? 5\) or absence of WAS protein in peripheral blood mononuclear cells determined by Western blotting and flow cytometry
- •3\. Molecular confirmation by WAS gene DNA sequencing
- •4\. Lack of HLA\-genotypically identical bone marrow or of a 10/10 antigen HLA\-matched unrelated donor or cord blood after 3 month search
- •5\. Parental, guardian, patient signed informed consent/assessment
- •6\. Willing to return for follow\-up during the 2 year study and the 3 year long\-term off study review
- •7\. Only for patients who have received previous allogenic haematopoietic stem cell transplant:
- •7\.1\. Failed allogenic haematopoietic stem cell transplant
- •7\.2\. Contraindication to repeat allogeneic transplantation for example severe graft versus host disease
排除标准
- •1\. Patient with HLA\-genotypically identical bone marrow
- •2\. Patient with 10/10 antigen HLA\-matched unrelated donor or cord blood
- •3\. Contraindication to leukapheresis
- •3\.1\. Anaemia (Hb \< 8g/dl)
- •3\.2\. Cardiovascular instability
- •3\.3\. Severe coagulopathy
- •3\.3\.1\. Contraindication to bone marrow harvest
- •3\.3\.2\. Contraindication to administration of conditioning medication
- •3\. Human immunodeficiency virus (HIV) positive patient
结局指标
主要结局
未指定
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