A Phase IIIb Study of the Safety and Efficacy of Tisagenlecleucel Out of Specification for Commercial Release in Patients Who Are Consistent With the Label Indication
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 200
- 试验地点
- 70
- 主要终点
- Percentage of participants with Adverse Events (AEs)
研究概览
简要总结
This study will evaluate the safety of tisagenlecleucel that is out of specification( OOS) for release as commercial product. Specifically, this study will evaluate the safety of CTL019 in the patients treated within the approved label by Japan Health Authority in Part 2. Only for Part 1, in addition to safety, key efficacy of CTL019 will also be evaluated.
详细描述
This is a single-arm, open-label, multicenter, interventional Phase IIIb study in pediatric/young adult patients with relapsed/refractory (r/r) B-cell acute lymphoblastic leukemia (pALL) and adult patients with r/r large B-cell lymphoma (LBCL) including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, high-grade B cell lymphoma, and DLBCL arising from follicular lymphoma for Part 1 and and r/r ALL and r/r non-Hodgkin's lymphomas (NHL) for Part 2
Patients whose final manufactured tisagenlecleucel patient-specific batch does not meet the approved local commercial release specifications are eligible for inclusion. Each case will be individually assessed and approved by the Novartis manufacturing facility and the Novartis global medical team (including Patient Safety). Following a single infusion of CTL019, the patient will be followed for 3 months for Part 1, and 1 day for Part 2.
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 100 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Signed informed consent/assent must be obtained for this study prior to participation in the study.
- •Patients for whom the final manufactured tisagenlecleucel product does not meet the commercial release specifications.
- •Not excluded from commercial manufacturing under the Health Authority-approved tisagenlecleucel prescribing information for their respective country/region.
- •OOS material has not been deemed to pose an undue safety risk to the patient.
- •Patient is suffering from a serious or life-threatening disease or condition.
- •Repeat leukapheresis is not clinically appropriate per the investigator assessment.
排除标准
- •For part 1, patients meeting any of the following criteria are not eligible for inclusion in this study:
- •Human immunodeficience virus (HIV) positive patients.
- •Patients with active replication of Hepatitis B virus (HBV) or Hepatitis C virus (HCV).
- •Patients with primary central nervous system (CNS) lymphoma.
- •History of hypersensitivity to any drugs or metabolites of similar chemical classes as tisagenlecleucel.
- •Uncontrolled active infection or inflammation.
- •Any medical condition identified by the investigator that may impact the assessment of the safety or efficacy outcomes in relation to study treatment.
- •Pregnant or nursing (lactating) women. For part 2, exclusion criteria are not set; however, administration should be performed in accordance with the latest versions of the package insert of CTL019.
研究组 & 干预措施
Group C: r/r NHL
Adult patients with r/r NHL in consistent with the Health Authority approved indication in the package insert for CTL019 in Japan whose final manufactured product is OOS for commercial release, but it is considered that the benefit-risk profile may remain favorable and the usual expected benefits of infusing such a product outweigh the potential risks for the patient.
干预措施: CTL019 (Biological)
Group A: pALL
Pediatric/young adult patients with r/r pALL who meet the indication in the Health Authority-approved CTL019 package insert in the respective country/region whose final manufactured product is OOS for commercial release, but it is considered that the benefit-risk profile may remain favorable and the usual expected benefits of infusing such a product outweigh the potential risks for the patient.
干预措施: CTL019 (Biological)
Group B: r/r LBCL
Adult patients with r/r LBCL including DLBCL not otherwise specified, high grade B-cell lymphoma, and DLBCL arising from follicular lymphoma, that is consistent with the Health Authority-approved indication in the package insert for CTL019 in the respective country/region whose final manufactured product is OOS for commercial release, but it is considered that the benefit-risk profile may remain favorable and the usual expected benefits of infusing such a product outweigh the potential risks for the patient.
干预措施: CTL019 (Biological)
结局指标
主要结局
Percentage of participants with Adverse Events (AEs)
时间窗: From Screening up to 3 months for Part 1 and 1 day for Part 2
Percentage of participants with Serious AEs (SAEs) and non-SAEs
次要结局
- Part 1: Overall Remission Rate in Group A (pALL)(Up to 3 months)
- Part 1: Overall Response Rate in Group B (LBCL)(Up to 3 months)