Retrospective-prospective Study to Evaluate Treatment Management and Outcomes of Patients With Waldenström's Macroglobulinemia (WM) Treated in Italy According to the Zanubrutinib (Brukinsa®) Compassionate Use Program (CUP) and in Common Practice Following Commercial Approval

Fondazione Italiana Linfomi - ETS28 sites in 1 country212 target enrollmentDecember 13, 2024

ConditionsWaldenström's Macroglobulinemia (WM)

InterventionsZanubrutinib

DrugsZanubrutinib

Overview

Phase: Not Applicable
Intervention: Zanubrutinib
Conditions: Waldenström's Macroglobulinemia (WM)
Sponsor: Fondazione Italiana Linfomi - ETS
Enrollment: 212
Locations: 28
Primary Endpoint: Cumulative incidence of treatment discontinuation due to toxicity
Status: Active, not recruiting
Last Updated: 3 months ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

This is a non-interventional, observational, retrospective and prospective multicenter Italian study, to describe treatment management and outcomes of Waldenström's Macroglobulinemia (WM) patients treated according to the Italian Compassionate Use Program (CUP) and receiving zanubrutinib following its commercial approval.

Registry

clinicaltrials.gov

Start Date

December 13, 2024

End Date

December 1, 2026

Last Updated

3 months ago

Study Type

Observational

Sex

All

Investigators

Sponsor

Fondazione Italiana Linfomi - ETS

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Signed and dated informed consent form
•Waldenström's macroglobulinemia diagnosis in need of treatment according to ESMO (European Society for Medical Oncology) guideline 2018
•Patients who received Zanubrutinib according to the Italian CUP or in common practice following Zanubrutinib commercial approval
•Treatment with zanubrutinib according to current SmPC (Summary of Product Characteristics)
•Treatment decision before inclusion into this non-interventional study
•Age ≥18 years

Exclusion Criteria

•Contraindications according to SmPC for patients with WM
•Participation in an interventional clinical trial during zanubrutinib treatment
•Patients with disease progression during a BTKi treatment (if pre-treated with BTK, only those intolerants are considered eligible)

Arms & Interventions

Prospective cohort

Patients with Waldenström's macroglobulinemia treated with Zanubrutinibin clinical practice enrolled from the study start up to 12 months.

Intervention: Zanubrutinib

Outcomes

Primary Outcomes

Cumulative incidence of treatment discontinuation due to toxicity

Time Frame: From the date of first zanubrutinib administration to the date of definitive treatment discontinuation or dose reduction for any cause or death from any cause, from october 2020 up to 24 months since study start

Cumulative incidence of treatment discontinuation due to toxicity

Secondary Outcomes

Overall survival (OS)(Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study))
Incidence of serious adverse events (SAEs).(Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study))
Incidence of adverse events leading to death.(Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study))
Overall response rate (ORR)(Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study))
Best response(Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study))
Time to treatment failure(Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study))
EORTC-QLQ-C30(Prospective cohort: up to 24 months (duration of study))
Incidence of adverse events (AEs)(Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study))
Incidence of adverse events related to zanubrutinib.(Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study))
Incidence of relevant adverse events.(Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study))
Incidence of adverse events leading to treatment discontinuation.(Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study))
Incidence of adverse events leading to dose reduction/interruption.(Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study))
AEs ≥ G3 Hematological and non-hematological.(Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study))
Impact of patients' characteristics and comorbidities on G3 or higher AEs and relevant adverse events development.(Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study))
Major response rate (MRR)(Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study))
Change of IgM levels until end of zanubrutinib treatment.(Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study))
Time to first onset of relevant adverse event.(Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study))
Frequency of transformation of WM to an aggressive lymphoma and frequency of secondary malignancies.(Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study))
Progression-free survival (PFS)(Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study))
Duration of response (DOR)(Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study))
Impact of patients and disease characteristics, and biomarker test results (MYD88 and CXCR4, if available) on treatment response.(Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study))
Hospital stays(Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study))
EQ-5D-5L(Prospective cohort: up to 24 months (duration of study))