Zanubrutinib in Patients With Waldenström’s Macroglobulinemia
Short title: ARIADNE
Title: Zanubrutinib in Patients With Waldenström’s Macroglobulinemia (ARIADNE)
Start: November 2022 (Austria)
Coordinating Investigator: Univ. Prof. Dr. Richard Greil (in Austria)
NIS Number: NA
Number of patients: 150 (planned)
Sponsor: iOMEDICO AG, AGMT Sponsor representative in Austria
ARIADNE will collect and analyze data from adult Waldenström’s macroglobulinemia patients in need of treatment. The study will explore the medical resource utilization during therapy with zanubrutinib (Brukinsa®).
Further aims are to assess effectiveness, safety and tolerability of the treatment as well as treatment satisfaction and biomarkers. These data will be supplemented by the assessment of patient-reported outcomes (PROs)/ health-related quality of life (QoL).
Since treatment options for Waldenström’s macroglobulinemia are limited and the most important factor is to keep or improve quality of life (QoL) of the patients, there is an urge for real-world clinical data of Waldenström’s macroglobulinemia patients treated with zanubrutinib, especially focusing on patients already treated upfront with a BTK inhibitor, older patients and patients with comorbidities.
- To assess medical resource utilization; Frequency of hospitalizations, i.e. number of hospital stays plus number of emergency unit visits (without hospitalization) per patient.
- Course of QoL during treatment and follow-up (collected via EORTC QLQ-C30 and EQ-5D-5L)
- Incidence of (S)AEs; (S)AEs will be summarized by the most recent Medical Dictionary for Regulatory Activities (MedDRA) system organ class and preferred term
- Incidence of (S)ADRs ((serious) adverse drug reactions) related to zanubrutinib
- Incidence of adverse events of special interest (AESIs)
- The time to first onset of AESIs
- The time to first onset of neutropenia grade ≥3 (MedDRA terms: neutropenia and neutrophil count decrease)
- Rate of patients with neutropenia grade ≥3 during zanubrutinib treatment. Neutropenia incorporates the MedDRA terms: neutropenia and neutrophil count decrease.
- The proportion of patients with a best overall response of CR or VGPR.
- Major response rate (MRR)
- Best response
- Progression-free Survival (PFS)
- Overall Survival (OS)
Physicians will select appropriate patients for enrollment. Appropriate patients are expected to:
- Signed and dated informed consent form
- Waldenström’s macroglobulinemia
- Treatment with zanubrutinib according to current SmPC
- Treatment decision before inclusion into this non-interventional study
- Age ≥18 years
- Contraindications according to SmPC for patients with Waldenström’s macroglobulinemia
- Participation in an interventional clinical trial during zanubrutinib treatment