Clinical Phase II Trial to Evaluate Efficacy and Safety of CD34+ Cells Mobilization and Collection After Treatment With Plerixafor and Filgrastim in Patients With Fanconi Anemia for Subsequent Transduction With a Lentiviral Vector Carring FANCA Gene and Reinfusion in the Patient
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 13
- 试验地点
- 2
- 主要终点
- Toxicity of the mobilization procedure according to National Cancer Institute (CTC NCI, versión 3.0)
研究概览
简要总结
Fanconi anemia (FA) is a congenital disease characterized by bone marrow failure and increased incidence of malignant tumors. The Project pursue the optimization of the collection of hematopoietic progenitor cells for later use in another clinical trial entitled "Clinical Trial Phase I/II to evaluate the safety and efficacy of the infusion of autologous CD34+ cells mobilized with mozobil and filgrastim, and transduced with a lentiviral vector carrying the FANCA gene (Orphan Drug) for patients with Fanconi Anemia Subtype A ". The objectives of this study are, therefore, to assess the safety and efficacy of CD34+ cells mobilization with mozobil and filgrastim, which is postulated the most efficient for the collection of CD34+ cells from FA patients.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 64 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female > 1 year
- •diagnosed of Fanconi's anemia confirmed by instability chromosomal test with diepoxy-butane or mitomycin C
- •At least one of the following parameters must be higher than these values: Hemoglobin:8,0 g/dL; neutrophils: 750/mm3; platelets: 30.000/mm3
- •Lansky index> 60%.
- •Left ventricular ejection fraction >50%.
- •To grant informed consent in agreement with current law norms
- •Women in childbearing age must obtain a negative result in the pregnancy test in serum or urine in the visit of selection and accept the use of suitable contraceptive methods since at least 14 days prior to the first dose of mobilizing treatment until the 14 days following the last
排除标准
- •Evidence of myelodysplastic syndromes or leukemia, or cytogenetic abnormalities predicted of these syndromes in bone marrow aspiration. Cytogenetic analyses performed 2 months before starting study are accepted
- •Patients with active infection process or any other underlaying severe medical process
- •Severe Functional alteration of organs (hepatic, renal, respiratory)(?3), according to National Cancer Institute (NCI CTCAE v3) criteria
- •Haematopoietic transplant
- •Any disease or concomitant process that is not compatible with the study as per investigator opinion
- •Patients not elegible because of an psico-social evaluation
- •Patients that received transfusional support during the last 3 months.
- •Pregnant or breastfeeding women
研究组 & 干预措施
plerixafor and filgrastim treatment
to assess the safety and efficacy of CD34+ cells mobilization with plerixafor and filgrastim
干预措施: filgrastim (Drug)
plerixafor and filgrastim treatment
to assess the safety and efficacy of CD34+ cells mobilization with plerixafor and filgrastim
干预措施: plerixafor (Drug)
结局指标
主要结局
Toxicity of the mobilization procedure according to National Cancer Institute (CTC NCI, versión 3.0)
时间窗: after 12 months
At a year (± 30 days) after the last apheresis, a complete physical examination, blood cell count, basic biochemistry and bone marrow aspirate will be done to the patient in order to control their general health status.
次要结局
- Percentage of patients that reach >5 CD34+ cells/mcl after treatment with filgrastim and plerixafor(after 8 days)
- Percentage of patients that reach a total CD34+ yield >4x10E6/kg, using the estimated weight of the patient in 5 years(after 8 days)
- Percentage of samples in which the recovery of CD34+ cells after the immunomagnetic selection procedure is >50%(after 8 days)
- Percentage of patients in which the CD34+ cells after the immunomagnetic selection is ³ 4x10E6/kg, using the projected weight at 5 years(after 8 days)