A Phase 1 Study Evaluating SC291, a Hypoimmune, Allogeneic CD19-directed CAR T Cell Therapy, in Subjects With Severe Relapsed or Refractory Autoimmune Diseases (GLEAM)
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 7
- 试验地点
- 5
- 主要终点
- Evaluate safety and tolerability of SC291
研究概览
简要总结
SC291-102 is a Phase 1 study to evaluate SC291 safety and tolerability, preliminary clinical response, cellular kinetics and exploratory assessments for subjects with severe autoimmune diseases.
详细描述
Systemic lupus erythematosus (SLE) is an autoimmune disease with multisystemic organ involvement that is often fatal. SLE is subcategorized as extrarenal lupus (ERL) or lupus nephritis (LN). B cell depletion therapies have played an important role in the treatment of multiple B cell-driven autoimmune diseases.
Subjects included in this trial will be subjects with diagnoses of systemic lupus erythematosus (SLE) including lupus nephritis (LN) and extrarenal systemic lupus erythematosus (ERL), or anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) (including Granulomatous Polyangitis and Microscopic Polyangiitis) who have refractory disease, have relapsed and have not shown appropriate clinical responses following prior systemic treatments.
This study is being conducted to evaluate the safety and efficacy of an investigational cell therapy, SC291, that can be given to patients with LN, ERL or AAV, in separate parallel cohorts, who have active disease.
A single dose of SC291 will be evaluated in patients who are pretreated with a standard regimen including cyclophosphamide (CY) and fludarabine (FLU).
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 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age ≥18 and ≤75
- •For LN cohort:
- •Diagnosis of SLE based on the 2019 European League Against Rheumatism (EULAR)/American College of Rheumatology (ACR)
- •Biopsy-proven LN class III or IV, according to 2018 Revised International Society of Nephrology/Renal Pathology Society (ISN/RPS) criteria
- •Refractory disease to ≥ 2 prior treatment regimens
- •For ERL cohort:
- •Diagnosis of SLE based on the 2019 European League Against Rheumatism (EULAR)/American College of Rheumatology (ACR) classification criteria for adult SLE
- •Severe or relapsing disease not responding to at least 2 prior recent disease-modifying therapies
- •For AAV Cohort, diagnosed with Granulomatous Polyangiitis (GPA) or Microscopic Polyangiitis (MPA) based on the 2022 ACR/EULAR classification criteria
排除标准
- •Prior CD19-directed cell therapy including CAR T treatment or other genetically modified cell therapy (e.g., Natural Killer (NK) cell)
- •For LN and ERL Cohorts, central nervous system (CNS) lupus manifestations or history or presence of CNS disorder
- •For LN and ERL Cohorts, diagnosis of anti-phospholipid antibody syndrome
- •For AAV Cohort only, Diagnosis of Eosinophilic Granulomatosis with Polyangiitis (EGPA) as defined by the 2022 ACR/EULAR classification criteria for EGPA -
研究组 & 干预措施
LN Cohort
SC291 with lymphodepleting therapy
干预措施: SC291 (Biological)
ERL Cohort
SC291 with lymphodepleting therapy
干预措施: SC291 (Biological)
AAV Cohort
SC291 with lymphodepleting therapy
干预措施: SC291 (Biological)
结局指标
主要结局
Evaluate safety and tolerability of SC291
时间窗: 24 months
Safety and Tolerability: Proportion of subjects experiencing adverse events and dose-limiting toxicities
次要结局
- Evaluate preliminary clinical response to SC291 (LN Cohort)(12 months)
- Evaluate preliminary clinical response to SC291 (LN and ERL Cohorts)(12 months)
- Evaluate preliminary clinical response to SC291 (ERL Cohort)(12 months)
- Evaluate cellular kinetics and persistence of SC291(24 months)
- Evaluate preliminary clinical response to SC291(12 months)
- Evaluate preliminary clinical response to SC291 (AAV Cohort)(12 months)