Clinical trial 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.
Official 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
Study type: OBSERVATIONAL
Overall status: NOT_YET_RECRUITING
Conditions: Waldenström's Macroglobulinemia (WM)
Lead sponsor: Fondazione Italiana Linfomi - ETS
Sponsor class: OTHER
First posted: 2024-06-04
Enrollment: 125
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: 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.

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.


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)

Primary outcomes:
measure: Cumulative incidence of treatment discontinuation due to toxicity
timeFrame: 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:
measure: Incidence of adverse events (AEs)
timeFrame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)
measure: Incidence of adverse events related to zanubrutinib.
timeFrame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)
measure: Incidence of relevant adverse events.
timeFrame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)
measure: Incidence of serious adverse events (SAEs).
timeFrame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)
measure: Incidence of adverse events leading to death.
timeFrame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)
measure: Incidence of adverse events leading to treatment discontinuation.
timeFrame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)
measure: Incidence of adverse events leading to dose reduction/interruption.
timeFrame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)
measure: Time to first onset of relevant adverse event.
timeFrame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)
measure: AEs ≥ G3 Hematological and non-hematological.
timeFrame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)
measure: Impact of patients' characteristics and comorbidities on G3 or higher AEs and relevant adverse events development.
timeFrame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)
measure: Frequency of transformation of WM to an aggressive lymphoma and frequency of secondary malignancies.
timeFrame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)
measure: Major response rate (MRR)
timeFrame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)
measure: Overall response rate (ORR)
timeFrame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)
measure: Best response
timeFrame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)
measure: Progression-free survival (PFS)
timeFrame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)
measure: Overall survival (OS)
timeFrame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)
measure: Time to treatment failure
timeFrame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)
measure: Duration of response (DOR)
timeFrame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)
measure: Change of IgM levels until end of zanubrutinib treatment.
timeFrame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)
measure: Impact of patients and disease characteristics, and biomarker test results (MYD88 and CXCR4, if available) on treatment response.
timeFrame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)
measure: Hospital stays
timeFrame: Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study)
measure: EORTC-QLQ-C30
timeFrame: Prospective cohort: up to 24 months (duration of study)
measure: EQ-5D-5L
timeFrame: Prospective cohort: up to 24 months (duration of study)
Other outcomes:
Locations:
city: Alessandria
country: Italy
facility: A.O. SS. Antonio e Biagio e Cesare Arrigo - S.C.D.U. Ematologia
city: Ancona
country: Italy
facility: A.O.U. Ospedali Riuniti - Clinica di Ematologia
city: Ascoli Piceno
country: Italy
facility: Ospedale C. e G. Mazzoni - U.O.C. di Ematologia
city: Bari
country: Italy
facility: AOU Policlinico Consorziale - U.O. Ematologia con Trapianto
city: Biella
country: Italy
facility: Nuovo Ospedale degli Infermi - SSD Ematologia
city: Bologna
country: Italy
facility: Policlinico S. Orsola-Malpighi - Istituto di Ematologia "Seragnoli"
city: Bolzano
country: Italy
facility: Ospedale Centrale di Bolzano - Divisione di Ematologia e T.M.O.
city: Catania
country: Italy
facility: Azienda Ospedaliera Universitaria Policlinico - S. Marco - UOC di Ematologia
city: Ferrara
country: Italy
facility: Azienda Ospedaliero-Universitaria di Ferrara - Arcispedale Sant'Anna - Ematologia e fisiopatologia della coagulazione
city: Firenze
country: Italy
facility: Azienda Ospedaliera Universitaria Careggi - Unità funzionale di Ematologia
city: Genova
country: Italy
facility: Ospedale Policlinico San Martino S.S.R.L. - IRCCS per l'Oncologia - Ematologia e terapie cellulari
city: Milano
country: Italy
facility: ASST Grande Ospedale Metropolitano Niguarda - S.C. Ematologia
city: Milano
country: Italy
facility: IEO Istituto Europeo di Oncologia - Divisione Ematoncologia
city: Milano
country: Italy
facility: Ospedale Maggiore Policlinico - Fondazione IRCCS Ca Granda - Ematologia
city: Novara
country: Italy
facility: AOU Maggiore della Carità di Novara - SCDU Ematologia
city: Padova
country: Italy
facility: AOU di Padova - Ematologia
city: Palermo
country: Italy
facility: A.O. Ospedali Riuniti Villa Sofia-Cervello - Divisione di Ematologia
city: Pavia
country: Italy
facility: RCCS Policlinico S. Matteo di Pavia - Div. di Ematologia
city: Pescara
country: Italy
facility: P.O. Spirito Santo di Pescara - UOC Ematologia Dipartimento Oncologico Ematologico - ASL Pescara
city: Pisa
country: Italy
facility: AOU Pisana - U.O. Ematologia
city: Ravenna
country: Italy
facility: Ospedale delle Croci - Ematologia
city: Reggio Emilia
country: Italy
facility: Azienda Unità Sanitaria Locale-IRCCS - Arcispedale Santa Maria Nuova - Ematologia
city: Roma
country: Italy
facility: Università Cattolica Sacro Cuore - Ematologia
city: Siena
country: Italy
facility: AOU Senese - U.O.C. Ematologia
city: Teramo
country: Italy
facility: Ospedale "G. Mazzini" - UOS Ematologia
city: Torino
country: Italy
facility: A.O.U. Città della Salute e della Scienza di Torino - Ematologia Universitaria
city: Torino
country: Italy
facility: A.O.U. Città della Salute e della Scienza di Torino - S.C. Ematologia
city: Udine
country: Italy
facility: Azienda Sanitaria Universitaria Friuli Centrale (ASU FC) - SOC Clinica Ematologica
Study officials:
affiliation: ASST Grande Ospedale Metropolitano Niguarda, Milano, Italy
name: Anna Maria Frustaci
role: PRINCIPAL_INVESTIGATOR
References: