a Safety and Efficacy Study of β-globin Restored Autologous Hematopoietic Stem Cells for β-thalassemia Major Patients With CVS-654 Mutation
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 发起方
- 入组人数
- 2
- 试验地点
- 1
- 主要终点
- Proportion of subjects with engraftment;
研究概览
简要总结
This is a single center, single arm, open-label study to determine the safety and efficacy of β-globin restored autologous hematopoietic stem cells in β- thalassemia major patients with CVS-654 mutation.
详细描述
β-globin restored autologous hematopoietic stem cells will be manufactured using CRISPR/Cas9 gene editing system. Subject participation for this study will be 1 year. Subjects who enroll in this study will be asked to participate in a subsequent long-term follow up study that will monitor the safety and efficacy of the treatment they receive for up to 15 years post-transplant.
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
- 5 Years 至 15 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •5-15 years old. Clinically diagnosed as β-thalassemia major with IVS-654 gene mutation phenotype;
- •Subjects or at least one legal guardian/agent understand and voluntarily sign informed consent.
- •Subjects with no affection with EBV, HIV, CMV, TP, HAV, HBV and HCV.
- •Subjects body condition eligible for autologous stem cell transplant.
排除标准
- •Subjects acceptable for allogeneic hematopoietic stem cell transplantation and have an available fully matched related donor.
- •Active bacterial, viral, or fungal infection. Treated with erythropoietin prior 3 months. Immediate family member with any known hematological tumor. Subjects with severe psychiatric disorders to be unable to cooperate. Recently diagnosed as malaria. History of complex autoimmune disease. Persistent aspartate transaminase (AST), alanine transaminase (ALT), or total bilirubin value >3 x the upper limit of normal (ULN).
- •Subjects with severe heart, lung and kidney diseases. With serious iron overload. Any other condition that would render the subject ineligible for HSCT, as determined by the attending transplant physician or Investigator.
- •Subjects who are receiving treatment from another clinical study, or have received another gene therapy.
- •Subjects or guardians had resisted the guidance of the attending doctor. Subjects whom the investigators do not consider appropriate for participating in this clinical study.
结局指标
主要结局
Proportion of subjects with engraftment;
时间窗: up to 42 days post transplant
Incidence and severity of adverse events as a measure of safety and tolerability. Adverse events assessed according to NCI-CTCAE v5.0 criteria
时间窗: up to 60 days post transplant
次要结局
- Proportion of subjects achieving transfusion independence;(up to 24 months post transplant)
- Proportion of subjects with a > = 50% reduced annualized volume of packed RBC transfusions.(up to 24 months post transplant)