EUCTR2007-004308-11-GB进行中（未招募）1 期
PHASE I/II CLINICAL TRIAL OF HAEMATOPOIETIC STEM CELL GENE THERAPYFOR THE WISKOTT-ALDRICH SYNDROME - Gene Therapy for WAS , version 5.0
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- Genethon
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
- •a. Males of all ages
- •b. Severe WAS (clinical score 3 – 5) or absence of WAS protein in peripheral blood mononuclear cells determined by Western blotting and flow cytometry
- •c. Molecular confirmation by WAS gene DNA sequencing
- •2. Lack of HLA-genotypically identical bone marrow OR of a 10/10 antigen HLA-matched unrelated donor or cord blood after 3 month search
- •3.Parental, guardian, patient signed informed consent/assessment
- •4.Willing to return for follow-up during the 2 year study
- •5.Only for patients who have received previous allogenic haematopoietic stem cell transplant:
- •a.Failed allogenic haematopoietic stem cell transplant
- •b.Contraindication to repeat allogeneic transplantation for example severe graft versus host disease
- •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
- •a. Males of all ages
- •b. Severe WAS (clinical score 3 – 5) or absence of WAS protein in peripheral blood mononuclear cells determined by Western blotting and flow cytometry
- •c. Molecular confirmation by WAS gene DNA sequencing
- •2. Lack of HLA-genotypically identical bone marrow OR of a 10/10 antigen HLA-matched unrelated donor or cord blood after 3 month search
- •3.Parental, guardian, patient signed informed consent/assessment
- •4.Willing to return for follow-up during the 2 year study
- •5.Only for patients who have received previous allogenic haematopoietic stem cell transplant:
- •a.Failed allogenic haematopoietic stem cell transplant
- •b.Contraindication to repeat allogeneic transplantation for example severe graft versus host disease
- •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
- •a. Males of all ages
- •b. Severe WAS (clinical score 3 – 5) or absence of WAS protein in peripheral blood mononuclear cells determined by Western blotting and flow cytometry
- •c. Molecular confirmation by WAS gene DNA sequencing
- •2. Lack of HLA-genotypically identical bone marrow OR of a 10/10 antigen HLA-matched unrelated donor or cord blood after 3 month search
- •3.Parental, guardian, patient signed informed consent/assessment
- •4.Willing to return for follow-up during the 2 year study
- •5.Only for patients who have received previous allogenic haematopoietic stem cell transplant:
- •a.Failed allogenic haematopoietic stem cell transplant
- •b.Contraindication to repeat allogeneic transplantation for example severe graft versus host disease
- •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
- •a. Patient with HLA-genotypically identical bone marrow
- •b. Patient with 10/10 antigen HLA-matched unrelated donor or cord blood
- •a. Contraindication to leukapheresis
- •i. anaemia (Hb < 8g/dl)
- •ii. cardiovascular instability
- •iii. severe coagulopathy
- •b. Contraindication to bone marrow harvest
- •c. Contraindication to administration of conditioning medication
- •3.HIV positive patient
- •a. Patient with HLA-genotypically identical bone marrow
- •b. Patient with 10/10 antigen HLA-matched unrelated donor or cord blood
- •a. Contraindication to leukapheresis
- •i. anaemia (Hb < 8g/dl)
- •ii. cardiovascular instability
- •iii. severe coagulopathy
- •b. Contraindication to bone marrow harvest
- •c. Contraindication to administration of conditioning medication
- •3.HIV positive patient
- •a. Patient with HLA-genotypically identical bone marrow
- •b. Patient with 10/10 antigen HLA-matched unrelated donor or cord blood
- •a. Contraindication to leukapheresis
- •i. anaemia (Hb < 8g/dl)
- •ii. cardiovascular instability
- •iii. severe coagulopathy
- •b. Contraindication to bone marrow harvest
- •c. Contraindication to administration of conditioning medication
- •3.HIV positive patient
研究者
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