New hope for rare blood cancer: zanubrutinib trial targets untreated patients

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Clinical and definitive histologic diagnosis of WM. Participant must be treatment-naive. * Participant must meet at least 1 criterion for treatment according to consensus panel criteria from the Seventh International Workshop on Waldenström's macroglobulinemia (IWWM). * Participant must have measurable disease, as defined by serum immunoglobulin M (IgM) level \> 0.5 g/dL. * Participants must have Eastern Cooperative Oncology Group (ECOG) Performance Status of 0 to 2. * Participants must have adequate organ function as indicated by the following laboratory values ≤ 7 days before the first dose of study treatment: 1. Participants must not have required blood transfusion or growth factor support ≤ 7 days before sample collection at screening for the following: * Absolute neutrophil count (ANC) ≥ 0.75 x 10\^9/L. * Platelets ≥ 50 x 10\^9/L. 2. Creatinine clearance of ≥ 30 ml/min as estimated by the Cockcroft-Gault formula. 3. Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 3 x upper limit of normal (ULN). 4. Serum total bilirubin ≤ 2 x ULN (total bilirubin must be \< 3 x ULN for participants with Gilbert syndrome). * Female participants of childbearing potential must be willing to use a highly effective method of birth control and refrain from egg donation for the duration of the study and for at least 1 month after the last dose of zanubrutinib. They must also have a negative urine or serum pregnancy test result ≤ 7 days before the first dose of study treatment. Exclusion Criteria: * Evidence of disease transformation at the time of study entry. * Central nervous system (CNS) involvement by WM. Patients with a history of CNS involvement must undergo magnetic resonance imaging (MRI) and cerebrospinal fluid cytology studies to document no evidence of CNS disease prior to study entry. * Evidence of disease transformation at the time of study entry. * Participants with any of the following cardiovascular risk factors: 1. Active cardiac ischemia (eg, cardiac chest pain) ≤ 28 days before first dose of study drug. 2. Any history of acute myocardial infarction ≤ 6 months before the first dose of study drug. 3. Any history of heart failure meeting New York Heart Association (NYHA) Classification III or IV (Appendix 7)≤ 6 months before the first dose of study drug. 4. Any event of ventricular arrhythmia ≥ Grade 2 in severity ≤ 6 months before the first dose of study drug. 5. Active, clinically significant second-degree atrioventricular block Mobitz II, or third degree atrioventricular block. 6. Any history of cerebrovascular accident ≤ 6 months before the first dose of study drug. 7. Uncontrolled hypertension that cannot be managed by standard antihypertension medications ≤ 28 days before the first dose of study drug. 8. Any episode of syncope or seizure ≤ 28 days before first dose of study drug. * At the time of study entry, participants taking warfarin or other vitamin K antagonists. * Participants requiring ongoing therapy with strong or moderate cytochrome CYP3A inducers * Corticosteroids given with antineoplastic intent within 7 days, or chemotherapy, targeted therapy, or radiation therapy within 4 weeks, or antibody-based therapy within 4 weeks before the start of study drug. * Major surgical procedure within 4 weeks before the start of study treatment (bone marrow aspirate and biopsy procedures are not considered major surgical procedures). Note: Other protocol defined criteria may apply