Autologous Hematopoietic Stem Cell Transplant for Children and Young Adults With Life Threatening Autoimmune Diseases
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Two-year progression free survival
研究概览
简要总结
A subset of autoimmune diseases (ADs) in children and young adults are life-threatening and unresponsive to conventional treatments. In these patients, the delivery of high dose immunosuppressive therapy followed by autologous stem cell transplant (ASCT) offers a treatment strategy capable of purging the pathogenic, autoreactive immune system and an opportunity for "immune reset." This strategy has been used in adults across a myriad of indications with evidence for efficacy. This study proposes a pilot study to evaluate this therapeutic strategy in children and young adults with systemic sclerosis (SSc) and systemic lupus erythematosis (SLE), two potentially life threatening autoimmune diseases that may response to this therapeutic approach.
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
- 8 Years 至 25 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age 8 ≤ 25 years at time of enrollment.
- •Severe systemic sclerosis or systemic lupus erythematosus based on specific criteria
- •Adequate organ function status
- •No active, untreated infections.
排除标准
- •Previous hematopoietic stem cell transplant (HSCT) or solid organ transplant
- •Ongoing participation in a clinical trial testing an investigational drug or ongoing receipt of disallowed disease modifying anti-rheumatic drugs (DMARD)
- •Severe comorbidity that jeopardizes the ability of the subject to tolerate therapy
研究组 & 干预措施
CD3/CD19 depleted ASCT
The test article is autologous stem cell transplant with a CD3/CD19-depleted stem cell product.
干预措施: Depletion of CD3/CD19 in an autologous stem cell transplant (Biological)
结局指标
主要结局
Two-year progression free survival
时间窗: 2 years
Survival without evidence of relapse or disease progression
次要结局
- Disease-specific response/progression endpoints: SSc cohort(24 months following transplant)
- Disease-specific response/progression endpoints: Systemic Lupus Erythematosus (SLE) cohort(24 months following transplant)
- Overall survival (OS)(2 and 5 years following transplant)
- Event free survival (EFS)(2 and 5 years following transplant)
- 100 day treatment-related mortality(100 days from stem cell infusion)
- Time to engraftment(3 days)
- Change in quality of life(prior to autologous stem cell transplant (ASCT) until 5 years post-transplant)
研究者
Stephan Grupp MD PhD
Director of Cancer Immunotherapy Program
Children's Hospital of Philadelphia