Clinical trial · Observational
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.
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
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Waldenström's Macroglobulinemia (WM) | Waldenstrom Macroglobulinemia | ALIAS | 0.85 |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Zanubrutinib | Drug | Zanubrutinib | ALIAS |
Design
Arms and outcomes
Arms (2)
- label
- Retrospective cohort
- description
- 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.
- label
- Prospective cohort
- description
- Patients with Waldenström's macroglobulinemia treated with Zanubrutinibin clinical practice enrolled from the study start up to 12 months.
- interventionNames
- Drug: Zanubrutinib
Primary outcomes (1)
- 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
- description
- Cumulative incidence of treatment discontinuation due to toxicity
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
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)
References
Publications (0)
Data not yet available