A Single-Arm, Open-Label, Multicenter, Phase 1b/2 Study Evaluating the Safety and Efficacy of AUTO1 (Obecabtagene Autoleucel [Obe-cel]) in Pediatric Patients With CD19-positive Relapsed/Refractory (R/R) B Cell Acute Lymphoblastic Leukemia (B ALL) or R/R Aggressive Mature B Cell Non-Hodgkin Lymphoma (B NHL).
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 30
- 试验地点
- 9
- 主要终点
- Frequency and severity of adverse events (AEs) and serious adverse events (SAEs)
研究概览
简要总结
This is a Phase 1b/2 study to evaluate the safety and efficacy of autologous T cells engineered with a chimeric antigen receptor (CAR) targeting cluster of differentiation (CD)19 in pediatric patients with relapsed or refractory (r/r) B cell acute lymphoblastic leukemia (B ALL) and r/r B cell Non-Hodgkin lymphoma (B NHL).
详细描述
This is a single-arm, open-label, multi-center, Phase 1b/2 study to determine the safety and efficacy of obe-cel administered intravenously in pediatric patients < 18 years old with r/r B ALL and with r/r aggressive mature B NHL.
The safety and tolerability of obe cel in pediatric patients will be continually monitored by the Sponsor. Efficacy endpoints will be determined by an Independent Response Review Committee (IRRC).
The study will involve consented patients going through the following sequential study periods: screening, leukapheresis, bridging as necessary, lymphodepletion, treatment evaluation, and follow-up.
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
- 0 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •< 18 years old at screening
- •≥ 6 kg body weight at screening
- •Pediatric patients with r/r B ALL
- •r/r CD19-positive aggressive mature B including the B NHL subtypes: i) diffuse large B cell lymphoma, ii) Burkitt's lymphoma, iii) primary mediastinal large B cell lymphoma, iv) high-grade B cell lymphoma (not otherwise specified).
- •Karnofsky (age ≥ 10 years) or Lansky (age < 10 year) performance status score ≥ 50%.
- •In participants with B ALL, local documentation of CD19 expression on leukemic blasts in the BM, peripheral blood, or cerebrospinal fluid or biopsy done no more than 30 days prior to consent.
- •Adequate renal, hepatic, pulmonary, and cardiac function.
排除标准
- •Diagnosis of chronic myelogenous leukemia in lymphoid blast crisis.
- •History or presence of clinically relevant central nervous system (CNS) pathology unrelated to CNS leukemia.
- •Presence of active or uncontrolled fungal, bacterial, viral, or other infection requiring systemic antimicrobials for management.
- •Received prior (< 3 months before obe cel infusion) stem cell transplantation.
- •Prior CD19 targeted therapy other than blinatumomab.
- •Experienced Grade ≥ 3 neurotoxicity following blinatumomab.
研究组 & 干预措施
AUTO1
干预措施: AUTO1 (Biological)
结局指标
主要结局
Frequency and severity of adverse events (AEs) and serious adverse events (SAEs)
时间窗: Up to 24 months
Incidence and duration of severe hypogammaglobulinemia
时间窗: Up to 24 months
Proportion of pediatric participants with r/r B ALL at screening who achieve complete remission (CR) within 3 months of obe-cel infusion per Independent Response Review Committee (IRRC) assessment
时间窗: 3 months
次要结局
- Overall survival (OS) in B ALL(Up to 24 months)
- Incidence of CD19-negative relapse at any time in B NHL(Up to 24 months)
- Progression-free survival in B NHL(Up to 24 months)
- OS in B NHL(Up to 24 months)
- Duration of response in B NHL(Up to 24 months)
- CR per IRRC assessment at any time in B ALL(Up to 24 months)
- Overall remission rate (ORR) (CR + complete remission with incomplete recovery of counts [CRi]) per IRRC assessment at any time in B ALL(Up to 24 months)
- Minimal residual disease (MRD)-negative ORR per IRRC assessment at any time in B ALL(Up to 24 months)
- Event-free survival in B ALL(Up to 24 months)
- ORR (CR or partial response [PR]) per Investigator assessment occurring at any time in B NHL(Up to 24 months)