A Long-term Follow-up Study Evaluating the Safety and Efficacy of Subjects With β-thalassemia Treated With Base-edited Autologous Hematopoietic Stem Cell (CS-101) Transplantation
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 5
- 试验地点
- 2
- 主要终点
- Frequency and severity of SAEs and CS-101 related AES as assessed by CTCAE v5.0
研究概览
简要总结
This is a study to evaluate the long-term safety and efficacy of CS-101 in participants who received CS-101 in study CS -101-03 (NCT06065189)
详细描述
This is a study to evaluate the long-term safety and efficacy of CS-101 in participants who received CS-101 in study CS -101-03 (NCT06065189) .
Subjects in the CS-101-03 study will be entered into long-term follow-up of this study up to 2 years post-infusion at the completion of the last (6-month) follow-up visit after treatment with CS-101 Injection.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Other
- 盲法
- None
入排标准
- 年龄范围
- 3 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants (or his or her legally appointed and authorized representative or guardian) must sign and date informed consent form (ICF) and, where applicable, an assent form
- •Participants must have received CS-101 infusion in last IIT study
排除标准
- •There are no exclusion criteria
结局指标
主要结局
Frequency and severity of SAEs and CS-101 related AES as assessed by CTCAE v5.0
时间窗: Signing of informed consent up to 2 years post CS-101 infusion
CommonTerminology Criteria for Adverse Events（CTCAE）has 5 levels of AE determination, increasing in severity as the level increases
Occurrence of all-cause death
时间窗: Signing of informed consent up to 2 years post CS-101 infusion
Occurrence of achieving transfusion independence for at least 12 consecutive months
时间窗: From 3 months after last RBC transfusion up to 2 years post CS-101 infusion
New malignancies and hematologic disorders
时间窗: Signing of informed consent up to 2 years post CS-101 infusion
Based on ICD-11
次要结局
- Change in fetal hemoglobin(HbF) concentration over time(up to 2 years post-CS-101 infusion)
- Change in total hemoglobin(Hb) concentration over time(up to 2 years post-CS-101 infusion)
- Chimerism level in Peripheral blood and bone marrow Proportion of alleles with intended genetic modification in peripheral blood leukocytes and bone marrow over time(up to 2 years post-CS-101 infusion)