A Single-arm, Multi-center, Open-label Proof of Concept Safety and Efficacy Study of FCR001 Cell-based Therapy in Adults With Rapidly Progressive Diffuse Cutaneous Systemic Sclerosis at Risk for Organ Failure
试验速览
- 阶段
- 1 期
- 状态
- 撤回
- 试验地点
- 2
- 主要终点
- Incidence of recipient adverse events (AEs)
研究概览
简要总结
This is a multicenter, open-label study to evaluate the safety and tolerability and explore the efficacy of FCR001 cell therapy in adults with rapidly progressive Diffuse Cutaneous Systemic Sclerosis (dcSSc) at risk for organ failure.
详细描述
The purpose of this multicenter, single-arm study is to evaluate the safety and tolerability and explore the efficacy of FCR001 cell therapy in adults with rapidly progressive dcSSc at risk for organ failure. It consists of 2 years of treatment and 3 years of follow-up, with the primary analysis performed at 24 months.
FCR001 is a cell therapy product that is administered by intravenous (IV) infusion, following nonmyeloablative (NMA) conditioning. It consists of mobilized peripheral blood cells, facilitating cells, and αβ T cells. This therapy is designed to induce donor-specific tolerance by establishing sustained chimerism and to protect against graft versus host disease (GvHD), the major impediment for advancing allogeneic hematopoietic stem cell therapy (HSCT) as a potential therapy in patients.
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 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
结局指标
主要结局
Incidence of recipient adverse events (AEs)
时间窗: From day before infusion to 60 months
Incidence of recipient serious adverse events (SAEs)
时间窗: From day before infusion to 60 months
Occurrence of Graft versus Host Disease (GvHD)
时间窗: From infusion to 60 months
Time to neutrophil recovery
时间窗: From infusion to 28 days
Time to platelet recovery
时间窗: From infusion to 28 days
次要结局
- Percent donor whole blood chimerism(From infusion to 60 months)
- Incidence of donor SAEs(From donation to 12 months)
- Incidence of donor AEs(From donation to 12 months)
- Percentage of donor T-cell chimerism(From infusion to 60 months)