Non-invasive Diagnostics and Monitoring of MRD and Clonal Evolution in Waldenström's Macroglobulinemia and in IgM-MGUS

NCT ID: NCT03521596

Last Updated: 2025-11-18

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 300 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2018-08-28
• Study Completion Date: 2025-10-02

Brief Summary

Multicenter retrospective and prospective observational study including patients with WM or IgM-MGUS evaluated at the time of diagnosis and during the disease course using highly sensitive techniques.

Detailed Description

Multicenter retrospective and prospective observational study including patients with WM or IgM-MGUS evaluated at the time of diagnosis and during the disease course using highly sensitive techniques such as flow cytometry, real time quantitative PCR (RT-qPCR), digital droplet PCR (dd-PCR) and NGS, in order to: evaluate the mutational status on genomic DNA or cell-free DNA and compare the results to assess the most reliable source for mutation studies; perform and compare molecular and flow cytometry analyses on bone marrow, peripheral blood (both analyses), plasma and urine samples (only molecular analysis) to assess the best source for diagnosis and MRD monitoring.

Conditions

Waldenstrom Macroglobulinemia

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: OTHER

Study Groups

Patient enrolled

Part of the patients will be retrospectively enrolled (learning sample) and part prospectively (validation sample)

MRD and clonal evolution

Intervention Type: OTHER

Patient evaluation at the time of diagnosis and during the disease course using highly sensitive techniques

Interventions

MRD and clonal evolution

Patient evaluation at the time of diagnosis and during the disease course using highly sensitive techniques

Intervention Type: OTHER

Eligibility Criteria

Inclusion Criteria

• Diagnosis of IgM monoclonal gammopathy of undetermined significance (Ig-MGUS) or Waldenström's Macroglobulinemia (WM) according to criteria established at the second International Workshop on Waldenström's Macroglobulinemia [1]
• Age ≥ 18 years
• Previously untreated patients (only for the prospective cohort)
• Symptomatic or asymptomatic disease
• Subject understands and voluntarily signs an informed consent form approved by an Independent Ethics Committee (IEC)/Institutional Review Board (IRB)

Exclusion Criteria

• Active HBV, HCV or HIV infection (antiHBc+ patients with undetectable HBV-DNA are eligible to the study)

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Fondazione Italiana Linfomi - ETS

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Marzia Varettoni

Role: PRINCIPAL_INVESTIGATOR

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

Locations

Divisione di Ematologia Ospedale Niguarda

Milan, MI, Italy

SOS Ematologia Ospedale C. Massaia

Asti, , Italy

IRCCS Istituto Tumori Giovanni Paolo II - UOC Ematologia

Bari, , Italy

AOU Università degli Studi della Campania Luigi Vanvitelli

Napoli, , Italy

I.R.C.C.S. Istituto Oncologico Veneto - Oncologia 1

Padua, , Italy

A.O. Universitaria Di Parma

Parma, , Italy

Ematologia Policlinico San Matteo

Pavia, , Italy

AO Arcispedale Santa Maria Nuova - IRCCS

Reggio Emilia, , Italy

Ospedale degli Infermi di Rimini

Rimini, , Italy

Policlinico A. Gemelli Università Cattolica del Sacro Cuore

Roma, , Italy

UOC Ematologia, AOU Senese

Siena, , Italy

A.O. Città della Salute e della Scienza Ematologia Universitaria

Torino, , Italy

Azienda Sanitaria Universitaria Integrata di Udine (A.S.U.I. Udine) - PO S. Maria della Misericordia

Udine, , Italy

Ematologia - OSPEDALE DI CIRCOLO E FONDAZIONE MACCHI

Varese, , Italy

Countries

Italy

Other Identifiers

FIL_BIOWM

Identifier Type: -

Identifier Source: org_study_id