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.

NCT ID: NCT06441214

Last Updated: 2026-01-05

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Total Enrollment

212 participants

Study Classification

OBSERVATIONAL

Study Start Date

2024-12-13

Study Completion Date

2026-12-31

Brief Summary

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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.

Detailed Description

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Conditions

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Waldenström's Macroglobulinemia (WM)

Study Design

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Observational Model Type

COHORT

Study Time Perspective

OTHER

Study Groups

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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.

No interventions assigned to this group

Prospective cohort

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

Zanubrutinib

Intervention Type DRUG

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

Interventions

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Zanubrutinib

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

Intervention Type DRUG

Eligibility Criteria

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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)
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Fondazione Italiana Linfomi - ETS

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Anna Maria Frustaci

Role: PRINCIPAL_INVESTIGATOR

ASST Grande Ospedale Metropolitano Niguarda, Milano, Italy

Locations

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

Alessandria, , Italy

Site Status

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

Ancona, , Italy

Site Status

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

Ascoli Piceno, , Italy

Site Status

AOU Policlinico Consorziale - U.O. Ematologia con Trapianto

Bari, , Italy

Site Status

Nuovo Ospedale degli Infermi - SSD Ematologia

Biella, , Italy

Site Status

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

Bologna, , Italy

Site Status

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

Bolzano, , Italy

Site Status

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

Catania, , Italy

Site Status

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

Ferrara, , Italy

Site Status

Azienda Ospedaliera Universitaria Careggi - Unità funzionale di Ematologia

Florence, , Italy

Site Status

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

Genova, , Italy

Site Status

ASST Grande Ospedale Metropolitano Niguarda - S.C. Ematologia

Milan, , Italy

Site Status

IEO Istituto Europeo di Oncologia - Divisione Ematoncologia

Milan, , Italy

Site Status

Ospedale Maggiore Policlinico - Fondazione IRCCS Ca Granda - Ematologia

Milan, , Italy

Site Status

AOU Maggiore della Carità di Novara - SCDU Ematologia

Novara, , Italy

Site Status

AOU di Padova - Ematologia

Padua, , Italy

Site Status

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

Palermo, , Italy

Site Status

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

Pavia, , Italy

Site Status

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

Pescara, , Italy

Site Status

AOU Pisana - U.O. Ematologia

Pisa, , Italy

Site Status

Ospedale delle Croci - Ematologia

Ravenna, , Italy

Site Status

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

Reggio Emilia, , Italy

Site Status

Università Cattolica Sacro Cuore - Ematologia

Roma, , Italy

Site Status

AOU Senese - U.O.C. Ematologia

Siena, , Italy

Site Status

Ospedale "G. Mazzini" - UOS Ematologia

Teramo, , Italy

Site Status

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

Torino, , Italy

Site Status

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

Torino, , Italy

Site Status

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

Udine, , Italy

Site Status

Countries

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Italy

Other Identifiers

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FIL_BRUCE-ITA

Identifier Type: -

Identifier Source: org_study_id

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