A Phase 1/2, Open-label Study to Evaluate the Safety and Efficacy of Autologous CD19-specific Chimeric Antigen Receptor T Cells (CABA-201) in Subjects With Active Systemic Lupus Erythematosus
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 28
- 试验地点
- 44
- 主要终点
- To evaluate incidence of adverse events
研究概览
简要总结
RESET-SLE: A Phase 1/2 Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects With Active Systemic Lupus Erythematosus
详细描述
Systemic lupus erythematosus (SLE) is a chronic autoimmune disorder characterized by autoantibody production and abnormal B cell function. SLE presents with fluctuating severity and may cause tissue damage in a variety of organs over time. Lupus nephritis (LN) (renal involvement) is a common severe manifestation of SLE, which can lead to significant morbidity and mortality. This study is being conducted to evaluate the safety and efficacy of an investigational cell therapy, CABA-201, also called resecabtagene autoleucel, or "rese-cel". Rese-cel can be given to patients with either LN or SLE without renal involvement, in two separate parallel cohorts, who have active disease. Initially a single dose of CABA-201 in patients pretreated with a standard regimen including cyclophosphamide (CY) and fludarabine (FLU), will be evaluated. In addition, escalating doses of CABA-201 will be evaluated in patients without CY and FLU pretreatment.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age ≥18 and ≤65
- •A clinical diagnosis of SLE, based on the 2019 European League Against Rheumatism (EULAR)/American College of Rheumatology (ACR) classification criteria for adult SLE.
- •Positive antinuclear antibody (ANA) titer or anti-dsDNA antibody at screening.
- •For LN subjects only, active, biopsy-proven LN class III or IV, with or without the presence of class V, according to 2018 Revised International Society of Nephrology/Renal Pathology Society (ISN/RPS) criteria
- •For non-renal SLE subjects only: Active, moderate to severe SLE
排除标准
- •Contraindication to leukapheresis
- •History of anaphylactic or severe systemic reaction to fludarabine, cyclophosphamide or any of their metabolites
- •Active infection requiring medical intervention at screening
- •Current symptoms of severe, progressive, or uncontrolled renal, hepatic, hematological, gastrointestinal, pulmonary, psychiatric, cardiac, neurological, or cerebral disease, including severe and uncontrolled infections, such as sepsis and opportunistic infections.
- •Concomitant medical conditions that, in the opinion of the investigator, might place the subject at unacceptable risk for participation in this study, interfere with the assessment of the effects or safety of the investigational product or with the study procedures
- •For LN subjects only: The presence of kidney disease other than active lupus nephritis
- •Previous CAR T cell therapy
- •Prior solid organ (heart, liver, kidney, lung) transplant or hematopoietic cell transplant.
研究组 & 干预措施
CABA-201 with FLU/CY Preconditioning
LN Cohort: Infusion of CABA-201 with preconditioning in subjects with LN
Non-renal SLE Cohort: Infusion of CABA-201 with preconditioning in subjects with non-renal SLE who do not meet criteria for inclusion in the LN cohort
Expansion Cohort: Infusion of CABA-201 with preconditioning in subjects with LN and SLE
干预措施: CABA-201 (Biological)
CABA-201, No Preconditioning
Infusion of CABA-201 with no preconditioning in subjects with LN and non-renal SLE
干预措施: CABA-201 (Biological)
结局指标
主要结局
To evaluate incidence of adverse events
时间窗: Up to 28 days after CABA-201 infusion
次要结局
- To evaluate adverse events and laboratory abnormalities(Up to 156 weeks)
- To evaluate disease related biomarkers(Up to 156 Weeks)
- To characterize the pharmacodynamics (PD)(Up to 156 weeks)
- To characterize the pharmacokinetics (PK)(Up to 156 weeks)
- To evaluate efficacy(Up to 156 weeks)
- To evaluate disease related biomarkers(Up to 156 weeks)
- To evaluate efficacy(Up to 156 Weeks)