Study to Evaluate the Safety and Efficacy of RM-004 Cells for the Treatment of Hemoglobin H-Constant Spring Disease
试验速览
- 阶段
- 早期 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 5
- 试验地点
- 1
- 主要终点
- Proportion of subjects who achieve transfusion independence
研究概览
简要总结
The purpose of this study is to evaluate the safety and efficacy of RM-004 for Hemoglobin H-Constant Spring disease.
详细描述
This is a non-randomized, one-arm, open label study to evaluate the safety and efficacy of RM-004 for autologous hematopoietic stem cell transplantation (HSCT) for the treatment of Hemoglobin H-Constant Spring disease. Five subjects aged from 12 to 35 years will be recruited in this study.
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
- 12 Years 至 35 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects voluntarily sign informed consent by themselves or their legal guardians and complete the study procedures, follow-up examination and treatment.
- •At the time of screening, subjects should be aged from 12 to 35 years old, regardless of gender.
- •History of at least 100 mL/kilograms (kg)/year of packed red blood cells (pRBC) transfusions in the prior 2 years before screening.
- •Subjects diagnosed with Hemoglobin H-Constant Spring disease (--/ααCS) with HBA2 c.427T>C mutation.
排除标准
- •Subject who has an available HLA-matched/well-matched HSCT donor for allogeneic hematopoietic stem cell transplantation (HSCT).
- •Prior HSCT or gene therapy.
- •History of severe hemorrhagic disease.
- •Clinically significant active bacterial, viral, fungal or parasitic infections per investigator's judgement at the time of screening.
研究组 & 干预措施
RM-004
RM-004 (ex vivo gene-edited CD34+ autologous hematopoietic stem cells) will be administered as a single intravenous infusion
干预措施: RM-004 (Genetic)
结局指标
主要结局
Proportion of subjects who achieve transfusion independence
时间窗: Up to 24 months after RM-004 infusion
Transfusion independence (TI) was defined as a weighted average hemoglobin (Hb) ≥ 9 g/dL without any red blood cells transfusions for a continuous period of ≥12 months at any time during the study after RM-004 infusion.
Proportion of subjects with engraftment
时间窗: Within 42 days after RM-004 infusion
Proportion of subjects with successful neutrophil engraftment (first day of 3 consecutive measurements of absolute neutrophil count \[ANC\] ≥0.5×10\^9/L on 3 different days) after RM-004 infusion.
Safety of RM-004 infusion
时间窗: From signing of informed consent up to 24 months after RM-004 infusion
Number of subjects with adverse events (AEs) and serious adverse events (SAEs)
次要结局
- Incidence of all-cause mortality(From signing of informed consent up to 24 months after RM-004 infusion)
- Proportion of subjects who stop receiveing transfusion ≥ 6 months(Up to 24 months after RM-004 infusion)
- Duration of transfusion independence(Up to 24 months after RM-004 infusion)