REACT-01: Reversing Autoimmunity Through Cell Therapy
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 12
- 试验地点
- 1
- 主要终点
- Rate of SCRI-CAR19v3 Manufacturing Success
研究概览
简要总结
This is a phase 1, open-label, non-randomized study enrolling pediatric and young adult research participants with treatment-refractory Systemic Lupus Erythematosus (SLE), to examine the safety, feasibility, and efficacy of administering T cell products derived from peripheral blood mononuclear cells (PBMC) that have been genetically modified to express CD19 specific chimeric antigen receptor (CAR)
A child or young adult meeting all eligibility criteria and meeting none of the exclusion criteria will have their T cells collected. The T cells will then be bioengineered into a CAR T cell that targets circulating and tissue residing B cells.
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
- 2 Years 至 30 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and female subjects aged between 2-30 years old. The first 3 subjects will be aged ≥
- •The FDA will review safety data to determine if the age can be lowered first to ≥ 12 then, following the treatment of 3 further subjects aged 12-17, to ≥ 2
- •Serologically active Systemic Lupus Erythematosus that is refractory to treatment
- •Able to tolerate apheresis or already has an apheresis product available for use in manufacturing.
- •≥ 24 weeks post last Rituximab or related B cell depleting therapy
- •≥ 12 weeks post last Belimumab / Anifrolumab therapy
- •≥ 4 weeks post last calcineurin inhibitor treatment
- •For subjects receiving non-calcineurin immunosuppressive therapy, on a stable dose for ≥ 8 weeks before enrollment
- •For subjects receiving corticosteroid therapy, on a stable dose for ≥ 2 weeks before enrollment
- •Adequate organ function
- •Adequate laboratory values
- •Subjects of childbearing or child-fathering potential must agree to use highly effective contraception from consent through 12 months following infusion of investigational product on trial
- •Subjects must be willing to remain within 1 hour's drive of Seattle Children's Hospital for 4 weeks following CAR T cell infusion.
- •Subject and/or legally authorized representative has signed the informed consent form for this study
排除标准
- •History or presence of active CNS lupus or other CNS disease
- •Kidney dysfunction requiring renal replacement therapy
- •Pregnant or breastfeeding
- •Insufficient pulmonary reserve including history of COPD, >10 pack year smoking history or SLE lung disease with hypoxia at rest with oxygen saturation ≤92% on room air
- •Unable to tolerate repletion with any formulation of IgG.
- •Active or prior malignancy, unless the malignancy was treated and there is no evidence of recurrent disease <5 years from enrollment.
- •Prior solid organ transplantation.
- •Presence of an active severe infection
- •Presence of any condition that, in the opinion of the investigator, would prohibit the subject from undergoing treatment under this protocol
研究组 & 干预措施
SCRI-CAR19v3
Single infusion of SCRI-CAR19v3
干预措施: SCRI-CAR19v3 (Biological)
结局指标
主要结局
Rate of SCRI-CAR19v3 Manufacturing Success
时间窗: 28 days
We will measure the number of successfully manufactured SCRI-CAR19v3 products.
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
时间窗: 28 days post-infusion
The investigators will assess and described the type, frequency, severity, and duration of adverse events associated with the CAR T cell product.
次要结局
未报告次要终点
研究者
Colleen Annesley
Medical Director & Co-Chief Medical Officer
Seattle Children's Hospital