A Long-term Follow-up Study Evaluating the Safety and Efficacy of Subjects With β-thalassemia Treated With CS-101 Transplantation
试验速览
- 阶段
- 不适用
- 状态
- Enrolling By Invitation
- 发起方
- 入组人数
- 8
- 试验地点
- 1
- 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 -101-01 (NCT06291961),No investigational drug product will be administered in the study.
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 -101-01 (NCT06291961). Subjects in the CS-101-01 study will be entered into long-term follow-up of this study up to 15 years post-infusion at the completion of the last (12-month) follow up visit after treatment with CS-101 Injection.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Prospective
入排标准
- 年龄范围
- 12 Years 至 35 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants must have received CS-101 infusion in CS-101-01 study
- •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
排除标准
- •There are no exclusion criteria
研究组 & 干预措施
Safety and efficacy assessments
Safety evaluations,disease-specific assessments,and assessments to monitor for long-term complications of autologous transplant
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: Through 15 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: Through 15 years post CS-101 infusion
Occurrence of achieving transfusion independence for at least 12 consecutive months
时间窗: Through 15 years post CS-101 infusion
次要结局
- Change in fetal hemoglobin(HbF) concentration over time(Through 15 years post CS-101 infusion)
- Change in total hemoglobin(Hb) concentration over time(Through 15 years post CS-101 infusion)
- Chimerism level in Peripheral blood Proportion of alleles with intended genetic modification in(Through 15 years post CS-101 infusion)
- Chimerism level in bone marrow Proportion of alleles with intended genetic modification in(Through 15 years post CS-101 infusion)
- Change in F-Cell concentration over time(Through 15 years post CS-101 infusion)