Allogeneic Stem Cell Transplantation of CordIn™, Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients With Hemoglobinopathies
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 入组人数
- 1
- 试验地点
- 5
- 主要终点
- The Percentage of Patients With Donor-derived Engraftment at 42 Days Following Transplantation
研究概览
简要总结
CordIn™ is a cryopreserved stem/progenitor cell-based product of purified CD133+ cells composed of ex vivo expanded allogeneic UCB cells. The overall study objectives are to evaluate the safety and efficacy of CordIn™.
详细描述
CordIn™ is a cryopreserved stem/progenitor cell-based product of purified CD133+ cells composed of ex vivo expanded allogeneic UCB cells.
The trial ends when the last patient completes their last visit. The overall study objectives are to evaluate the safety and efficacy of CordIn™: single ex-vivo expanded cord blood unit transplantation in patients with hemoglobinopathies (sickle cell disease or thalassemia major) following a preparative therapy.
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 25 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patient is a candidate for allogeneic SCT for treatment of SCD or thalassemia
- •Patients must have one partially HLA-matched CBUs
- •Adequate Karnofsky Performance score or Lansky Play-Performance scale
- •Sufficient physiological reserves
- •Signed written informed consent
排除标准
- •Prior allogeneic HSCT
- •Evidence of HIV infection or HIV positive serology
- •Evidence of active Hepatitis B, Hepatitis C or EBV as determined by serology or PCR
- •Active or uncontrolled infection
- •Pregnancy or lactation
结局指标
主要结局
The Percentage of Patients With Donor-derived Engraftment at 42 Days Following Transplantation
时间窗: 42 days
One hundred percent of patients engrafted by day forty-two post-transplantation with donor-derived cells.
Number of Participants With no Acute Toxicity Associated With the Infusion of CordIn, Within 24 Hours Post-infusion.
时间窗: 24 hours
The number of patients with grade 4 or 5 toxicity were estimated together with 95% confidence limits based on the binomial distribution. The proportion with toxicity grades 1, 2, and 3 was also estimated.
次要结局
- Percentage of Overall Survival at 365 Days After Transplantation(365 days)