Clinical trial NCT05640102: Observational Study Evaluating the Efficacy and Safety of Zanubrutinib in Participants With Waldenström Macroglobulinemia
Official title: A Phase 4, Observational Study Evaluating the Efficacy and Safety of the Bruton Tyrosine Kinase (BTK) Inhibitor Zanubrutinib in Patients With Waldenström Macroglobulinemia
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Waldenstrom Macroglobulinemia
Lead sponsor: BeiGene
Sponsor class: INDUSTRY
First posted: 2022-12-07
Enrollment: 111
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Adult participants with WM who are either already receiving or are planned to start treatment with zanubrutinib

Brief summary: 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.


Eligibility criteria: Inclusion Criteria:

* 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:

  1. 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
  2. Cohort 2: non-L265P MYD88 mutation(s) and MYD88WT

Exclusion Criteria:

* 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

Primary outcomes:
measure: Major Response Rate (MRR)
timeFrame: Up to approximately 4 years
Secondary outcomes:
measure: VGPR+ Rate
timeFrame: Up to approximately 5 years
measure: Overall Response Rate (ORR)
timeFrame: Up to approximately 5 years
measure: Duration of Response (DOR)
timeFrame: Up to approximately 5 years
measure: Number of Participants with Treatment-emergent Adverse Events
timeFrame: Up to approximately 5 years
Other outcomes:
Locations:
city: Huntsville
country: United States
facility: Clearview Cancer Institute
state: Alabama
city: Mobile
country: United States
facility: Mitchell Cancer Institute
state: Alabama
city: Duarte
country: United States
facility: City of Hope National Medical Center
state: California
city: Glendale
country: United States
facility: Los Angeles Cancer Network (Lacn)
state: California
city: Los Angeles
country: United States
facility: Valkyrie Clinical Trials
state: California
city: Rancho Mirage
country: United States
facility: Eisenhower Medical Center, Lucy Curci Cancer Center
state: California
city: San Luis Obispo
country: United States
facility: Pacific Central Coast Health Centers, Slo Oncology and Hematology Health Center
state: California
city: Santa Maria
country: United States
facility: Pacific Central Coast Health Centers, Mission Hope Medical Oncology
state: California
city: Plantation
country: United States
facility: Brcr Medical Center, Inc
state: Florida
city: Hattiesburg
country: United States
facility: Hattiesburg Hematology and Oncology Clinic
state: Mississippi
city: Las Vegas
country: United States
facility: Comprehensive Cancer Centers of Nevada
state: Nevada
city: Florham Park
country: United States
facility: Summit Medical Group
state: New Jersey
city: Morristown
country: United States
facility: Morristown Medical Center
state: New Jersey
city: Westbury
country: United States
facility: Clinical Research Alliance, Inc
state: New York
city: Rio Piedras
country: Puerto Rico
facility: Pan American Oncology Trials, Llc
city: San Juan
country: Puerto Rico
facility: Auxilio Mutuo Cancer Center
Study officials:
affiliation: BeiGene
name: Study Director
role: STUDY_DIRECTOR
References: