NCT00613561Unknown2 期
Early Diagnosis and Stem Cell Transplantation for Severe Immunodeficiency Diseases
适应症
相关药物
试验速览
- 阶段
- 2 期
- 入组人数
- 25
- 试验地点
- 2
- 主要终点
- To evaluate the efficacy of an optimized reduced intensity conditioning regimen using Fludarabine, Busulfan, and Anti-Thymocyte Globulin in preparing severe immunodeficiency patients for allogeneic hematopoietic progenitor cell transplantation
研究概览
简要总结
The hypothesis of this study is that children with severe primary immunodeficiencies will benefit from early stem cell transplantation utilizing a reduced intensity conditioning regimen. This regimen is associated with a low risk of complications and will lead to correction of the underlying immunological defects.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- — 至 11 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Must show one of the following diseases:
- •Hyper-IgM
- •Wiskott-Aldrich Syndrome
- •Chediak-Higashi and Griscelli Syndromes
- •X-Linked Lymphoproliferative Diseases
- •IPEX Syndrome
- •NEMO Syndrome
- •other severe immunodeficiency diseases not stated above at the discretion of the Principal Investigator
- •Informed Consent
- •Adequate Renal Function
- •Adequate Liver Function
- •Adequate Cardiac Function
- •Adequate Pulmonary Function
- •Adequate Performance Statue
- •Adequate Venous Access
排除标准
- •Patient/Family has not signed informed consent
- •Patient does not have a clear diagnosis of a severe immunodeficiency disease
- •A suitable donor for the patient cannot be found
- •Patient is HIV positive
- •Patient has active Hepatitis B
- •Patient is pregnant
- •Patient is considered unsuitable for transplant at the discretion of the Principal Investigators or the medical director
结局指标
主要结局
To evaluate the efficacy of an optimized reduced intensity conditioning regimen using Fludarabine, Busulfan, and Anti-Thymocyte Globulin in preparing severe immunodeficiency patients for allogeneic hematopoietic progenitor cell transplantation
时间窗: 5 years
次要结局
- To prospectively follow the natural course of severe immunodeficiency diseases after transplantation(5 years)
- To measure the outcomes after stem cell transplantation using the reduced intensity transplant regimen.(5 years)
研究者
研究点 (2)
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