A Phase 2, Multicenter, Single-arm Study of Moxetumomab Pasudotox in Pediatric Subjects With Relapsed or Refractory Pediatric Acute Lymphoblastic Leukemia (pALL) or Lymphoblastic Lymphoma of B-cell Origin
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 37
- 试验地点
- 1
- 主要终点
- Percentage of Participants With Composite Complete Response (CRc)
研究概览
简要总结
The primary objective of this study is to evaluate the efficacy of moxetumomab pasudotox in pediatric participants with relapsed or refractory B-cell acute lymphoblastic leukemia (ALL) or B-cell lymphoblastic lymphoma.
详细描述
This is a global, multicenter, open-label, single-arm Phase 2 study to evaluate the efficacy and safety of moxetumomab pasudotox monotherapy in pediatric participants with relapsed or refractory B-cell ALL or B-cell lymphoblastic lymphoma. Participants will be enrolled at sites in North America, Europe, and Australia. This is an approximate 35 month study.
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
- 6 Months 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Moxetumomab Pasudotox 40 mcg/kg
Participants received 6 doses of moxetumomab pasudotox 40 microgram per kilogram (mcg/kg) intravenous infusion over 30 minutes every other day (Days 1, 3, 5, 7, 9, and 11) in 21-day treatment cycles until completion of a maximum of 6 cycles of therapy.
干预措施: Moxetumomab Pasudotox (Drug)
结局指标
主要结局
Percentage of Participants With Composite Complete Response (CRc)
时间窗: Prior to Cycle 1, and prior to every cycle, at the end of treatment, at post-treatment follow-up visits, and at the end of the study, up to 1 year
The CRc is defined as achieving complete response (CR), or CR with incomplete count recovery \[CRi\]) in participants with relapsed or refractory B-cell ALL or B-cell lymphoblastic lymphoma. Complete response (CR) as per International Working Group (IWG) is complete disappearance of all detectable clinical evidence of disease and disease-related symptoms if present before therapy. Morphologic CR with incomplete blood count recovery (CRi) is defined as the above CR criteria without specified blood counts. The efficacy assessments were evaluated as per investigator assessment.
次要结局
- Best Overall Response (BOR)(Prior to Cycle 1, and prior to every cycle, at the end of treatment, at post-treatment follow-up visits, and at the end of the study, up to 1 year)
- Bone Marrow Blast Percentage Change(Prior to Cycle 1, and prior to every cycle, at the end of treatment, at post-treatment follow-up visits, and at the end of the study, up to 1 year)
- Percentage of Participants With Minimal Residual Disease (MRD)-Negative CRc Rate(Prior to Cycle 1, and prior to every cycle, at the end of treatment, at post-treatment follow-up visits, and at the end of the study, up to 1 year)
- Overall Response Rate (ORR)(Prior to Cycle 1, and prior to every cycle, at the end of treatment, at post-treatment follow-up visits, and at the end of the study, up to 1 year)
- Time to Overall Response(Prior to Cycle 1, and prior to every cycle, at the end of treatment, at post-treatment follow-up visits, and at the end of the study, up to 1 year)
- Percentage of Participants Who Became Eligible to Receive an Stem Cell Transplant (SCT) After Treatment With Moxetumomab Pasudotox(Prior to Cycle 1, and prior to every cycle, at the end of treatment, at post-treatment follow-up visits, and at the end of the study, up to 1 year)
- Time to Transplant to Receive an Stem Cell Transplant (SCT) After Treatment With Moxetumomab Pasudotox(Prior to Cycle 1, and prior to every cycle, at the end of treatment, at post-treatment follow-up visits, and at the end of the study, up to 1 year)
- Percentage of Participants Who Were Neutropenic at Study Entry and Who Experienced Hematologic Activity (HA)(Prior to Cycle 1, and prior to every cycle, at the end of treatment, at post-treatment follow-up visits, and at the end of the study, up to 1 year)
- Duration of Complete Response (DOCR)(Prior to Cycle 1, and prior to every cycle, at the end of treatment, at post-treatment follow-up visits, and at the end of the study, up to 1 year)
- Duration of Overall Response (DOR)(Prior to Cycle 1, and prior to every cycle, at the end of treatment, at post-treatment follow-up visits, and at the end of the study, up to 1 year)
- Progression-Free Survival (PFS)(Prior to Cycle 1, and prior to every cycle, at the end of treatment, at post-treatment follow-up visits, and at the end of the study, up to 1 year)
- Overall Survival (OS)(Baseline to end of study or last contact date, up to 1 year)
- Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (TESAEs)(Baseline up to 30 days after the last dose of study drug, up to 1 year)
- Number of Participants With Abnormal Clinical Laboratory Parameters Reported as Treatment-Emergent Adverse Events (TEAE)(Baseline up to 30 days after the last dose of study drug, up to 1 year)
- Number of Participants With Clinically Significant Electrocardiogram (ECG) Abnormalities(Baseline up to 30 days after the last dose of study drug, up to 1 year)
- Number of Participants With Abnormal Vital Signs Reported as Treatment-Emergent Adverse Events (TEAEs)(Baseline up to 30 days after the last dose of study drug, up to 1 year)
- Number of Participants With Positive Anti-drug Antibody (ADA) and Neutralizing Antibodies (NAb)(Prior to the Start of Each Cycle for Cycles 1, 2, 3, and Subsequent Odd-Numbered Cycles, End of Treatment, and 30 Day Follow-up Visit, up to 1 year)
- Area Under the Plasma Concentration Time Curve From Time 0 to Infinity (AUC0-inf) After the First Dose of Cycle 1(Pre-infusion, end of infusion (EOI); 1, 3, and 6 hours post-infusion of Day 1 of Cycle 1)
- Area Under the Concentration Versus Time Curve From Time Zero to Last Quantifiable Concentration [AUC0-last] After the First Dose of Cycle 1(Pre-infusion, end of infusion (EOI); 1, 3, and 6 hours post-infusion at Day 1 of Cycle 1)
- Maximum Observed Drug Concentration in Plasma (Cmax) After the First Dose of Cycle 1(Pre-infusion, end of infusion (EOI); 1, 3, and 6 hours post-infusion at Day 1 of Cycle 1)
- Time to Reach Maximum Drug Concentration in Plasma (Tmax) After the First Dose of Cycle 1(Pre-infusion, end of infusion (EOI); 1, 3, and 6 hours post-infusion at Day 1 of Cycle 1)
- Terminal Phase Elimination Half Life (t1/2) After the First Dose of Cycle 1(Pre-infusion, end of infusion (EOI); 1, 3, and 6 hours post-infusion at Day 1 of Cycle 1)
- Systemic Clearance (CL) After the First Dose of Cycle 1(Pre-infusion, end of infusion (EOI); 1, 3, and 6 hours post-infusion at Day 1 of Cycle 1)