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 人开始时间: 2011年5月20日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 5
研究概览
简要总结
2017 results in: https://www.ncbi.nlm.nih.gov/pubmed/28716862
研究设计
- 研究类型
- Interventional
入排标准
- 性别
- Male
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
研究者
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