A Phase 4, Observational Study Evaluating the Efficacy and Safety of the Bruton Tyrosine Kinase (BTK) Inhibitor Zanubrutinib in Patients With Waldenström Macroglobulinemia
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 111
- 试验地点
- 15
- 主要终点
- Major Response Rate (MRR)
研究概览
简要总结
This is a hybrid (retrospective and prospective) non-interventional registry study to further describe the clinical profile of zanubrutinib in Waldenström macroglobulinemia (WM) participants with and without specific mutations and from racial and ethnic minority groups. Data collected from this registry study will be used to better understand the clinical benefit and safety of zanubrutinib for the treatment of participants in these populations.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Clinical and definitive histologic diagnosis of WM
- •Measurable disease, as defined by a serum immunoglobulin M (IgM) level > 0.5 g/dL at the time of zanubrutinib initiation
- •Started treatment with zanubrutinib, has been treated with zanubrutinib, or is planned to be prescribed zanubrutinib for the treatment of WM
- •Bone marrow specimens with central MYD88 test results of:
- •Cohort 1: MYD88 L265P mutation; enrollment of TN participants will be stopped in each racial and ethnic participant group when the required numbers of participants in the group are met
- •Cohort 2: non-L265P MYD88 mutation(s) and MYD88WT
排除标准
- •Evidence of disease transformation before the first dose of zanubrutinib
- •Evidence of other non-Hodgkin Lymphoma (NHL) subtypes
- •Prior or concurrent active malignancy ≤ 2 years before the first dose of zanubrutinib, except for malignancies that, in the investigator's opinion, will not obscure the interpretation of safety or efficacy results
- •Concurrent participation in another therapeutic clinical study while receiving zanubrutinib, although the participant may be eligible depending on the status of the interventional study after discussion with the Medical Monitor or designee on an individual basis
研究组 & 干预措施
Cohort 1: MYD88 L265P mutation
Arm A: Treatment-naïve (TN); Arm B: Relapsed/refractory (R/R)
干预措施: Zanubrutinib (Drug)
Cohort 2: Non-L265P MYD88 mutation(s) and MYD88 wildtype
Arm C: TN and R/R
干预措施: Zanubrutinib (Drug)
结局指标
主要结局
Major Response Rate (MRR)
时间窗: Up to approximately 4 years
MRR is defined as the proportion of participants achieving either complete response (CR), very good partial response (VGPR), or partial response (PR) as determined by the investigator using an adaptation of the response criteria updated at the Sixth International Workshop on WM (IWWM)
次要结局
- Number of Participants with Treatment-emergent Adverse Events(Up to approximately 5 years)
- VGPR+ Rate(Up to approximately 5 years)
- Overall Response Rate (ORR)(Up to approximately 5 years)
- Duration of Response (DOR)(Up to approximately 5 years)