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NCT Number: NCT06441214

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.

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.

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Waldenström's Macroglobulinemia (WM) Blood Protein Disorders Cardiovascular Diseases Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders Hemostatic Disorders Immune System Diseases Immunoproliferative Disorders Lymphatic Diseases Lymphoproliferative Disorders Neoplasms Neoplasms by Histologic Type Neoplasms, Plasma Cell Paraproteinemias Vascular Diseases Waldenstrom Macroglobulinemia

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

A.O. SS. Antonio e Biagio e Cesare Arrigo - S.C.D.U. Ematologia, Alessandria, Italy

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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)

Treatment and study plan

Zanubrutinib

Drug

Patients will be treated with Zanubrutinib as per routine clinical practice.

Primary outcomes

  1. 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

  1. Incidence of adverse events (AEs)

    Time frame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)

    Incidence of adverse events (AEs)

  2. Incidence of adverse events related to zanubrutinib.

    Time frame: 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.

  3. Incidence of relevant adverse events.

    Time frame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)

    Incidence of relevant adverse events.

  4. Incidence of serious adverse events (SAEs).

    Time frame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)

    Incidence of serious adverse events (SAEs).

  5. Incidence of adverse events leading to death.

    Time frame: 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.

  6. Incidence of adverse events leading to treatment discontinuation.

    Time frame: 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.

  7. Incidence of adverse events leading to dose reduction/interruption.

    Time frame: 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.

  8. Time to first onset of relevant adverse event.

    Time frame: 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.

  9. AEs ≥ G3 Hematological and non-hematological.

    Time frame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)

    AEs ≥ G3 Hematological and non-hematological.

  10. Impact of patients' characteristics and comorbidities on G3 or higher AEs and relevant adverse events development.

    Time frame: 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.

  11. Frequency of transformation of WM to an aggressive lymphoma and frequency of secondary malignancies.

    Time frame: 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.

  12. Major response rate (MRR)

    Time frame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)

    Major response rate (MRR) (≥PR) (best reported response).

  13. Overall response rate (ORR)

    Time frame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)

    Overall response rate (ORR, defined as MRR plus minor response rate)

  14. Best response

    Time frame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)

    Best response (best reported response).

  15. Progression-free survival (PFS)

    Time frame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)

    Progression-free survival (PFS) including 6, 12, and 24-month PFS rate

  16. Overall survival (OS)

    Time frame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)

    Overall survival (OS) including 6, 12, and 24-month OS rate.

  17. Time to treatment failure

    Time frame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)

    Time to treatment failure (any treatment definitive discontinuation, incl. patient or investigator decision, toxicity, progression or death).

  18. Duration of response (DOR)

    Time frame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)

    Duration of response (DOR)

  19. Change of IgM levels until end of zanubrutinib treatment.

    Time frame: 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.

  20. Impact of patients and disease characteristics, and biomarker test results (MYD88 and CXCR4, if available) on treatment response.

    Time frame: 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.

  21. Hospital stays

    Time frame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)

    Hospital stays comprises all planned and unplanned hospitalizations as well as emergency unit visits, regardless of whether there is an association with WM or not. All hospitalizations and emergency unit visits that started during therapy with zanubrutinib will be considered for hospital stays.

  22. EORTC-QLQ-C30

    Time frame: Prospective cohort: up to 24 months (duration of study)

    Quality of life (QoL) assessed with EORTC-QLQ-C30 questionnaire

  23. EQ-5D-5L

    Time frame: Prospective cohort: up to 24 months (duration of study)

    Quality of life (QoL) assessed with EQ-5D-5L questionnaire

Sponsors and collaborators

Lead sponsor

Fondazione Italiana Linfomi - ETS

Other

Registry information

Official study title

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

Important dates

Study start
2024
Primary completion
2026
Study completion
2026
First posted
Jun 4, 2024
Registry last updated
Jan 5, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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