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.

NCT06441214 | Active Not Recruiting

• 1 other identifier: FIL_BRUCE-ITA
• Study Type: observational
• Target: 212
• Locations: 1 country
• Sites: 28

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.

Conditions

Waldenström's Macroglobulinemia (WM)

Outcome Measures

Primary Outcomes (1)

• Cumulative incidence of treatment discontinuation due to toxicity

  Cumulative incidence of treatment discontinuation due to toxicity

  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

Secondary Outcomes (23)

• 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 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)

Study Arms (2)

Retrospective cohort

All patients with Waldenström's macroglobulinemia enrolled in Pre-Reimbursement Access Program (PRAP) of Zanubrutinib, as per Compassionate Use Program (CUP) of Zanubrutinib, and patients treated with commercial drug (Brukinsa®) from PRAP closure to the study start.

Prospective cohort

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

Drug: Zanubrutinib

Interventions

Zanubrutinib DRUG

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

Prospective cohort

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

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.

You may qualify if:

• 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)
• Age ≥18 years

You may not qualify if:

• 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)

Sponsors & Collaborators

• Fondazione Italiana Linfomi - ETS: lead

Study Sites (28)

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

Alessandria, Italy

Location

A.O.U. Ospedali Riuniti - Clinica di Ematologia

Ancona, Italy

Location

Ospedale C. e G. Mazzoni - U.O.C. di Ematologia

Ascoli Piceno, Italy

Location

AOU Policlinico Consorziale - U.O. Ematologia con Trapianto

Bari, Italy

Location

Nuovo Ospedale degli Infermi - SSD Ematologia

Biella, Italy

Location

Policlinico S. Orsola-Malpighi - Istituto di Ematologia "Seragnoli"

Bologna, Italy

Location

Ospedale Centrale di Bolzano - Divisione di Ematologia e T.M.O.

Bolzano, Italy

Location

Azienda Ospedaliera Universitaria Policlinico - S. Marco - UOC di Ematologia

Catania, Italy

Location

Azienda Ospedaliero-Universitaria di Ferrara - Arcispedale Sant'Anna - Ematologia e fisiopatologia della coagulazione

Ferrara, Italy

Location

Azienda Ospedaliera Universitaria Careggi - Unità funzionale di Ematologia

Florence, Italy

Location

Ospedale Policlinico San Martino S.S.R.L. - IRCCS per l'Oncologia - Ematologia e terapie cellulari

Genova, Italy

Location

ASST Grande Ospedale Metropolitano Niguarda - S.C. Ematologia

Milan, Italy

Location

IEO Istituto Europeo di Oncologia - Divisione Ematoncologia

Milan, Italy

Location

Ospedale Maggiore Policlinico - Fondazione IRCCS Ca Granda - Ematologia

Milan, Italy

Location

AOU Maggiore della Carità di Novara - SCDU Ematologia

Novara, Italy

Location

AOU di Padova - Ematologia

Padua, Italy

Location

A.O. Ospedali Riuniti Villa Sofia-Cervello - Divisione di Ematologia

Palermo, Italy

Location

RCCS Policlinico S. Matteo di Pavia - Div. di Ematologia

Pavia, Italy

Location

P.O. Spirito Santo di Pescara - UOC Ematologia Dipartimento Oncologico Ematologico - ASL Pescara

Pescara, Italy

Location

AOU Pisana - U.O. Ematologia

Pisa, Italy

Location

Ospedale delle Croci - Ematologia

Ravenna, Italy

Location

Azienda Unità Sanitaria Locale-IRCCS - Arcispedale Santa Maria Nuova - Ematologia

Reggio Emilia, Italy

Location

Università Cattolica Sacro Cuore - Ematologia

Roma, Italy

Location

AOU Senese - U.O.C. Ematologia

Siena, Italy

Location

Ospedale "G. Mazzini" - UOS Ematologia

Teramo, Italy

Location

A.O.U. Città della Salute e della Scienza di Torino - Ematologia Universitaria

Torino, Italy

Location

A.O.U. Città della Salute e della Scienza di Torino - S.C. Ematologia

Torino, Italy

Location

Azienda Sanitaria Universitaria Friuli Centrale (ASU FC) - SOC Clinica Ematologica

Udine, Italy

Location

MeSH Terms

Conditions

Waldenstrom Macroglobulinemia

Interventions

zanubrutinib

Condition Hierarchy (Ancestors)

Neoplasms, Plasma Cell; Neoplasms by Histologic Type; Neoplasms; Hemostatic Disorders; Vascular Diseases; Cardiovascular Diseases; Paraproteinemias; Blood Protein Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemorrhagic Disorders; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases

Study Officials

• Anna Maria Frustaci

  ASST Grande Ospedale Metropolitano Niguarda, Milano, Italy

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: OTHER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 15, 2024
• First Posted: June 4, 2024
• Study Start: December 13, 2024
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): December 1, 2026
• Last Updated: January 5, 2026

Record last verified: 2025-12

Locations

IT (28)