Waldenstrom's DiseaseSérieProWMNCT05911802
Prognostic Analyses on a Validation Series of Patients With Waldenström's Disease
An observational study of Waldenstrom's Disease and Prognostic Index, run by French Innovative Leukemia Organisation at 15 sites in France.
Trial summary
Waldenström's macroglobulinemia (WM) is defined by the association of bone marrow lymphoplasmocytic infiltration and monoclonal immunoglobulin M (IgM). A mutation in the MYD88 gene is found in up to 90% of patients, and a mutation in the CXCR4 gene in approximately one third of patients. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Waldenstrom's Disease, Prognostic Index
- How many people
- 500 participants
- Where
- 15 sitesFrance
- Study dates
- 11 Aug 2023to 15 Jun 2030
- How long will I take part?
- 5 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- Adult, Older adult
Study population: 250 symptomatic patients and 250 asymptomatic patients will be prospectively enrolled
Who may be able to join
- Patient with WM, fulfilling the diagnostic criteria defined at the 2nd Workshop on WM.
- Patient in whom follow-up is available until at least 01/01/2020. Each participating center should not enroll more 10% of patients lost to follow-up.
- Patient for whom a minimum annual follow-up is planned until 2024.
- Having given their consent for this study
Who cannot join
- Patient with other chronic lymphoid malignancy. Special attention will be paid to exclude other lymphoplasmacytic proliferations, especially marginal zone lymphoma.
- Patient with histological transformation in a diffuse large B-cell lymphoma or any other lymphoma at the time of the initiation of the 1st treatment.
- No consent for this study.
Trial locations and contacts
15 sites in 1 country. Contact the study team or a site near you to ask about taking part.
15 sites in 1 country
France15 sites, 15 recruiting
- Not yet recruiting
AMIENS - CH Amiens Picardie Site Sud
Amiens, 80054
Lydia MONTES, montes.lydia@chu-amiens.fr
- Not yet recruiting
Angers Chu
Angers, 49933
Christopher NUNES-GOMES, MD, +33 2 41 35 45 24, Christopher.nunesgomes@chu-angers.fr
- Not yet recruiting
Institut Bergonie
Bordeaux, 33076
Fontanet BIJOU, MD, +33556330448, f.bijou@bordeaux.unicancer.fr
- Not yet recruiting
Clermont-Ferrand - Chu Estaing
Clermont-Ferrand, 63000
Olivier TOURNILHAC, MD, otournilhac@chu-clermontferrand.fr
- Not yet recruiting
Le Mans CH
Le Mans
Kamel Laribi, Dr, klaribi@ch-lemans.fr
- Not yet recruiting
LENS - GHT Artois
Lens, 62300
Daniela ROBU, drobu@ch-lens.fr
- Not yet recruiting
LIBOURNE - Hôpital Robert Boulin
Libourne, 33505
Diane LARA, diane.lara@ch-libourne.fr
- Recruiting
LILLE GHICL - Hôpital Saint Vincent de Paul
Lille, 59000
Bénédicte HIVERT, Dr, 33 (0)3 20 87 45 32, hivert.benedicte@ghicl.net
- Not yet recruiting
Institut Paoli Calmette
Marseille, 130009
Thérèse AURRAN, MD, +33491223667, aurrant@ipc.unicancer.fr
- Not yet recruiting
APHP - Hôpital Pitié Salpêtrière - Hématologie
Paris, 75651
Damien ROOS-WEIL, Pr, 01 42 16 28 24, damien.roos-weil@psl.aphp.fr
- Not yet recruiting
POITIERS - Hématologie et Thérapie Cellulaire
Poitiers, 86021
Cécile TOMOWIAK, Dr, 05 49 44 43 07, cecile.tomowiak@chu-poitiers.fr
- Not yet recruiting
Reims Chu
Reims, 51092
Eric DUROT, Dr, edurot@chu-reims.fr
- Not yet recruiting
Strasbourg - Icans
Strasbourg, 67033
Elise TOUSSAINT, MD, e.toussaint@icans.eu
- Not yet recruiting
Toulouse - IUCT Oncopole - Service d'Hématologie
Toulouse, 31059
Loïc YSEBAERT, Pr, ysebaert.loic@iuct-oncopole.fr
- Not yet recruiting
VERSAILLES - Hôpital André Mignot
Versailles
Fatiha MERABET, Dr
About this study
Official title: Prognostic Analyses on a Validation Series of Patients With Waldenström's Disease: Validation of International Prognostic Indexes, Evaluation of Progression-free Survival as a Surrogate Endpoint for Overall Survival. A FILO Study.
Waldenström's macroglobulinemia (WM) is defined by the association of bone marrow lymphoplasmocytic infiltration and monoclonal immunoglobulin M (IgM). A mutation in the MYD88 gene is found in up to 90% of patients, and a mutation in the CXCR4 gene in approximately one third of patients. Treatment should be initiated in cases of cytopenia, bulky disease or when the physicochemical or immunological properties of IgM explain the occurrence of amyloidosis, cryoglobulin, neurological manifestations, or hyperviscosity syndrome (due to the presence of a large amount of IgM). However, approximately 30% of patients are diagnosed without any symptom and therefore they do not meet the criteria for initiating treatment.
At the time of initiation of the first treatment, the prognosis is usually estimated with the International Prognostic Index (IPSSWM) which is based on five variables: age, platelet count, haemoglobin concentrations, β2-microglobulin and monoclonal component concentration. Serum albumin and lactate dehydrogénase (LDH) levels also retain a prognostic role and these two characteristics have been incorporated in a proposal for a revision of this index.
Read more
Improving prognostic assessment at the time of the first treatment initiation and taking into account the prognostic impact of events occurring in the course of evolution, should improve the strength of treatment decision at the time of initial treatment and during the follow-up. It should also help to design clinical trial for fast and effective evaluation of new treatments. Our work should also help to adjust clinical monitoring of asymptomatic patients.
Prospective and retrospective multicenter prognostic study with a descriptive objective, associated with a biological collection appropriately annotated and stored. A retrospective series including 470 patients with symptomatic WM is already available. The follow-up of these patients will be updated and an additional series of 250 symptomatic patients will be prospectively enrolled. 250 asymptomatic patients will be also enrolled.
Read the detailed description
Waldenström's macroglobulinemia (WM) is defined by the association of bone marrow lymphoplasmocytic infiltration and monoclonal immunoglobulin M (IgM). A mutation in the MYD88 gene is found in up to 90% of patients, and a mutation in the CXCR4 gene in approximately one third of patients. Treatment should be initiated in cases of cytopenia, bulky disease or when the physicochemical or immunological properties of IgM explain the occurrence of amyloidosis, cryoglobulin, neurological manifestations, or hyperviscosity syndrome (due to the presence of a large amount of IgM). However, approximately 30% of patients are diagnosed without any symptom and therefore they do not meet the criteria for initiating treatment.
The prognosis of asymptomatic patients can be estimated with a prognostic index based on serum albumin, β2-microglobulin, the monoclonal component concentration and the bone marrow infiltration. Prognostic assessment of these patients could be improved by taking into account prior the free light chain concentrations and the molecular characteristics of the disease.
At the time of initiation of the first treatment, the prognosis is usually estimated with the International Prognostic Index (IPSSWM) which is based on five variables: age, platelet count, haemoglobin concentrations, β2-microglobulin and monoclonal component concentration. Serum albumin and LDH levels also retain a prognostic role and these two characteristics have been incorporated in a proposal for a revision of this index.
Thus improving prognostic assessment in patients with WM may rest on the following strategies:
- Modifying the variables to be considered before treatment initiation, particularly by considering albumin and lactate dehydrogenase concentrations or molecular characteristics of the disease in symptomatic patients, free-light chain concentration in asymptomatic patients and molecular abnormalities in both categories of patients.
- Evaluating the prognostic impact of events occurring during the course of treatment, such as response or progression in symptomatic patients.
Improving prognostic assessment at the time of the first treatment initiation and taking into account the prognostic impact of events occurring in the course of evolution, should improve the strength of treatment decision at the time of initial treatment and during the follow-up. It should also help to design clinical trial for fast and effective evaluation of new treatments. Our work should also help to adjust clinical monitoring of asymptomatic patients.
Two large subgroups of patients properly included with validated information and updated follow-up will be considered, namely: symptomatic and asymptomatic patients. This project is based on the assumption that it should be possible for each of these two cohorts to:
- validate a new prognostic system and compare its performance with previous systems
- to participate in a large international study of the validity of a surrogate endpoint of survival after initiation of the 1st treatment
- Conditions
- Waldenstrom's Disease, Prognostic Index
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| symptomatic WM | WM patients with symptom(s) : cytopenia, bulky disease or when the physicochemical or immunological properties of IgM explain the occurrence of amyloidosis, cryoglobulin, neurological manifestations, or hyperviscosity syndrome (due to the presence of a large amount of IgM) | |
| asymptomatic WM | WM patients without any symptom |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall survival
Percentage of living patients
Measured: 5 years after WM diagnosis
Secondary outcomes (2)
Progression free survival
percentage of living patients without disease progression
Measured: 1 year after WM diagnosis
Tolerance to treatment
percentage of patients discontinuing WM treatment due to toxicity
Measured: 1 year after initiating WM treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jun 2023
- Study start11 Aug 2023
- Primary completion15 Jun 2030 (estimated)
- Study completion15 Jun 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Waldenstrom's DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Other
- Enrolment
- 500 participants (estimated)
- Target duration
- 5 Years
Sponsor and investigators
- Lead sponsor
- French Innovative Leukemia Organisation
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05911802
- Sponsor's protocol ID
- FILObs_SérieProWM
- Acronym
- SérieProWM
- Status last verified
- Nov 2025
- First submitted
- 12 Jun 2023
- Last updated
- 26 Nov 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided: Specific or all individual participant data sets would be probably shared with the European ECWM Group. For example, data on use of BTKi ( Bruton tyrosine kinases inhibitors) in this population
Publications
Royston P, Altman DG. External validation of a Cox prognostic model: principles and methods. BMC Med Res Methodol. 2013 Mar 6;13:33. doi: 10.1186/1471-2288-13-33.
PubMed 23496923
Study at a glance
From the registry record, last updated by the sponsor on 26 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prognostic Analyses on a Validation Series of Patients With Waldenström's Disease: Validation of International Prognostic Indexes, Evaluation of Progression-free Survival as a Surrogate Endpoint for Overall Survival. A FILO Study.
- ClinicalTrials.gov ID
- NCT05911802
- Acronym / sponsor ID
- SérieProWM / FILObs_SérieProWM
- Conditions
- Waldenstrom's Disease, Prognostic Index
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 500 participants (estimated)
- Lead sponsor
- French Innovative Leukemia OrganisationUniversity, hospital or other
- Study start
- 11 Aug 2023 (actual)
- Primary completion
- 15 Jun 2030 (estimated)
- Study completion
- 15 Jun 2030 (estimated)
- First posted
- 22 Jun 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Waldenström's macroglobulinemia (WM) is defined by the association of bone marrow lymphoplasmocytic infiltration and monoclonal immunoglobulin M (IgM). A mutation in the MYD88 gene is found in up to 90% of patients, and a mutation in the CXCR4 gene in approximately one third of patients. Treatment should be initiated in cases of cytopenia, bulky disease or when the physicochemical or immunological properties of IgM explain the occurrence of amyloidosis, cryoglobulin, neurological manifestations, or hyperviscosity syndrome (due to the presence of a large amount of IgM). However, approximately 30% of patients are diagnosed without any symptom and therefore they do not meet the criteria for initiating treatment.
At the time of initiation of the first treatment, the prognosis is usually estimated with the International Prognostic Index (IPSSWM) which is based on five variables: age, platelet count, haemoglobin concentrations, β2-microglobulin and monoclonal component concentration. Serum albumin and lactate dehydrogénase (LDH) levels also retain a prognostic role and these two characteristics have been incorporated in a proposal for a revision of this index.
Improving prognostic assessment at the time of the first treatment initiation and taking into account the prognostic impact of events occurring in the course of evolution, should improve the strength of treatment decision at the time of initial treatment and during the follow-up. It should also help to design clinical trial for fast and effective evaluation of new treatments. Our work should also help to adjust clinical monitoring of asymptomatic patients.
Prospective and retrospective multicenter prognostic study with a descriptive objective, associated with a biological collection appropriately annotated and stored. A retrospective series including 470 patients with symptomatic WM is already available. The follow-up of these patients will be updated and an additional series of 250 symptomatic patients will be prospectively enrolled. 250 asymptomatic patients will be also enrolled.
Detailed description
Waldenström's macroglobulinemia (WM) is defined by the association of bone marrow lymphoplasmocytic infiltration and monoclonal immunoglobulin M (IgM). A mutation in the MYD88 gene is found in up to 90% of patients, and a mutation in the CXCR4 gene in approximately one third of patients. Treatment should be initiated in cases of cytopenia, bulky disease or when the physicochemical or immunological properties of IgM explain the occurrence of amyloidosis, cryoglobulin, neurological manifestations, or hyperviscosity syndrome (due to the presence of a large amount of IgM). However, approximately 30% of patients are diagnosed without any symptom and therefore they do not meet the criteria for initiating treatment.
The prognosis of asymptomatic patients can be estimated with a prognostic index based on serum albumin, β2-microglobulin, the monoclonal component concentration and the bone marrow infiltration. Prognostic assessment of these patients could be improved by taking into account prior the free light chain concentrations and the molecular characteristics of the disease.
At the time of initiation of the first treatment, the prognosis is usually estimated with the International Prognostic Index (IPSSWM) which is based on five variables: age, platelet count, haemoglobin concentrations, β2-microglobulin and monoclonal component concentration. Serum albumin and LDH levels also retain a prognostic role and these two characteristics have been incorporated in a proposal for a revision of this index.
Thus improving prognostic assessment in patients with WM may rest on the following strategies:
- Modifying the variables to be considered before treatment initiation, particularly by considering albumin and lactate dehydrogenase concentrations or molecular characteristics of the disease in symptomatic patients, free-light chain concentration in asymptomatic patients and molecular abnormalities in both categories of patients.
- Evaluating the prognostic impact of events occurring during the course of treatment, such as response or progression in symptomatic patients.
Improving prognostic assessment at the time of the first treatment initiation and taking into account the prognostic impact of events occurring in the course of evolution, should improve the strength of treatment decision at the time of initial treatment and during the follow-up. It should also help to design clinical trial for fast and effective evaluation of new treatments. Our work should also help to adjust clinical monitoring of asymptomatic patients.
Two large subgroups of patients properly included with validated information and updated follow-up will be considered, namely: symptomatic and asymptomatic patients. This project is based on the assumption that it should be possible for each of these two cohorts to:
- validate a new prognostic system and compare its performance with previous systems
- to participate in a large international study of the validity of a surrogate endpoint of survival after initiation of the 1st treatment
Study design
- Observational model
- Cohort
- Time perspective
- Other
- Target follow-up
- 5 Years
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| symptomatic WM | – | –WM patients with symptom(s) : cytopenia, bulky disease or when the physicochemical or immunological properties of IgM explain the occurrence of amyloidosis, cryoglobulin, neurological manifestations, or hyperviscosity syndrome (due to the presence of a large amount of IgM) |
| asymptomatic WM | – | –WM patients without any symptom |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Age groups
- Adult, Older adult
- Study population
- 250 symptomatic patients and 250 asymptomatic patients will be prospectively enrolled
Key inclusion criteria (4 listed)
- Patient with WM, fulfilling the diagnostic criteria defined at the 2nd Workshop on WM.
- Patient in whom follow-up is available until at least 01/01/2020. Each participating center should not enroll more 10% of patients lost to follow-up.
- Patient for whom a minimum annual follow-up is planned until 2024.
- Having given their consent for this study
Key exclusion criteria (3 listed)
- Patient with other chronic lymphoid malignancy. Special attention will be paid to exclude other lymphoplasmacytic proliferations, especially marginal zone lymphoma.
- Patient with histological transformation in a diffuse large B-cell lymphoma or any other lymphoma at the time of the initiation of the 1st treatment.
- No consent for this study.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Overall survival
Time frame: 5 years after WM diagnosis
Percentage of living patients
2 secondary outcomes
Progression free survival
Time frame: 1 year after WM diagnosis
Tolerance to treatment
Time frame: 1 year after initiating WM treatment
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Nov 2025.
- Background: PubMed 23496923Royston P, Altman DG. External validation of a Cox prognostic model: principles and methods. BMC Med Res Methodol. 2013 Mar 6;13:33. doi: 10.1186/1471-2288-13-33.
- Individual participant dataSharing planned: Undecided. Specific or all individual participant data sets would be probably shared with the European ECWM Group. For example, data on use of BTKi ( Bruton tyrosine kinases inhibitors) in this population
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
15 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Waldenstrom's Disease and Prognostic Index, run by French Innovative Leukemia Organisation at 15 sites in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 26 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall survival (time frame: 5 years after WM diagnosis). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05911802, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Nov 2025.