Long-term Follow-up (LTFU) of Patients Treated With Genome-edited Autologous Hematopoietic Stem and Progenitor Cells (HSPC)
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 4
- 试验地点
- 6
- 主要终点
- Number of participants with delayed adverse events that are suspected to be related to previous OTQ923 therapy
研究概览
简要总结
CADPT03A12001 is a prospective, multi-center study that is designed to follow all enrolled patients who have received treatment with OTQ923 for long-term safety and efficacy.
详细描述
This study is monitoring patients treated with OTQ923, an investigational drug product of ex vivo genome-edited autologous hematopoietic stem and progenitor cells (HSPCs) that induces fetal hemoglobin (HbF) production, for a total of 15 years following infusion to monitor long-term safety and efficacy.
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
- 18 Years 至 100 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients must have received gene therapy treatment from the parent treatment protocol (CADPT03A12101).
- •Patients must provide informed consent prior to their entry into this study.
排除标准
- •1. Completion of less than 1 year of safety follow-up in the treatment protocol (CADPT03A12101).
研究组 & 干预措施
OTQ923
Patients were administered OTQ923 while enrolled on the treatment protocol (CADPT03A12101). Patients enrolled on this LTFU study will not be administered any study treatment.
干预措施: OTQ923 (Biological)
结局指标
主要结局
Number of participants with delayed adverse events that are suspected to be related to previous OTQ923 therapy
时间窗: Up to 15 years
Number of participants with delayed adverse events including new secondary malignancies, new incidence or exacerbation of a prior autoimmune disorder, new incidence or exacerbation of a prior rheumatologic disorder, new hematologic disorder, and other adverse events considered to be related to OTQ923 therapy.
次要结局
- Persistence of fetal hemoglobin expression(Up to 15 years)
- WBC chimerism in peripheral blood(5 years)