DrugEvidence

Diseases / Waldenstrom Macroglobulinemia

Waldenstrom Macroglobulinemia

Disease

Waldenstrom macroglobulinemia is a rare indolent B-cell lymphoplasmacytic lymphoma with monoclonal IgM overproduction and marrow involvement. It can lead to hyperviscosity, neuropathy, cytopenias, and organ-related complications.

Trials
520
Recruiting
43
Completed
307
With results
156

Last revised

Pipeline

Phase by status

PhaseRecruitingNot yetBy invitationActiveCompletedTerminatedWithdrawnUnknownApproved for marketingNo longer availableTotal
Early Phase 1 4 4
Phase 1 1416942554 149
Phase 1/2 414351224 62
Phase 2 751411528109 188
Phase 2/3 11 2
Phase 3 112124 29
Phase 4 1115 8
Not applicable 421664 32
Unknown 112151633212 46

Trials started per year

2021
4
2022
9
2023
12
2024
9
2025
14
2026
13
2027
2

Conditions studied, by phase

ConditionEarly Phase 1Phase 1Phase 1/2Phase 2Phase 2/3Phase 3Phase 4Not applicableUnknownTotal
Waldenstrom Macroglobulinemia 41496218822983246 520
Leukemia, Lymphocytic, Chronic, B-Cell 4117429912322630 344
Lymphoma, B-Cell, Marginal Zone 31254210112012426 343
Lymphoma, Follicular 3120449612112422 332
Lymphoma, Mantle-Cell 3121417812112324 313
Lymphoma, Non-Hodgkin 396328011812321 275
Lymphoma, Large B-Cell, Diffuse 396347112012120 267
Burkitt Lymphoma 284255911512118 226
Precursor Cell Lymphoblastic Leukemia-Lymphoma 277206111612220 220
Lymphoma, Large-Cell, Immunoblastic 268185611712017 200

Most active sponsors

#SponsorTrialsPhase 3
1National Cancer Institute (NCI)510
2Fred Hutchinson Cancer Center362
3Dana-Farber Cancer Institute260
4Mayo Clinic181
5Memorial Sloan Kettering Cancer Center133
6Roswell Park Cancer Institute111
7City of Hope Medical Center100
8Case Comprehensive Cancer Center91
9Jonsson Comprehensive Cancer Center91
10M.D. Anderson Cancer Center90

Most studied interventions

#InterventionTrials
1Rituximab101
2Cyclophosphamide63
3fludarabine phosphate55
4Peripheral Blood Stem Cell Transplantation52
5Mycophenolic Acid34
6Bortezomib33
7Whole-Body Irradiation33
8ibrutinib29
9Dexamethasone28
10Tacrolimus26

Evidence

Results reported
50.8%156 of 307 completed
Published
20.2%62 of 307 completed
Primary endpoint met
31.2%5 of 16 analyses
Median months to report
24

Documents disclosed

Protocol available
63
Statistical analysis plan
62
PubMed citations
95

Trial profile

Median enrollment
39
Median duration
62.2months
Countries
44
Study sites
3051

Who is enrolled

Adults
518
Children
98
Older adults
503
Healthy volunteers
8

Median enrollment by phase

Early Phase 1
32
Phase 1
30
Phase 1/2
45
Phase 2
37
Phase 2/3
255
Phase 3
219
Phase 4
45
Not applicable
39
Unknown
141

Where trials run

United States
418
France
40
Italy
37
Germany
31
United Kingdom
31
Canada
28
Spain
23
China
21
Australia
20
Greece
15

Busiest cities

#CityCountryTrials
1BostonUnited States80
2New YorkUnited States78
3SeattleUnited States76
4RochesterUnited States54
5HoustonUnited States50
6ColumbusUnited States42
7DenverUnited States37
8ChicagoUnited States36
9DuarteUnited States36
10Los AngelesUnited States36

Terminations

Terminated
76
Withdrawn
24
Stopped for recruitment
7.5%39 of 520 trials

Stated reasons

Other / unclear
40
Recruitment
39
Lack of efficacy
6
Business decision
4
Funding
4
Safety
4
COVID-19
2
Regulatory
1

Reasons are grouped from the sponsor's own wording with a rule-based classifier; the original text is shown below.

Recently stopped

TrialStatusStated reasonStopped
NCT04947618 Terminated Terminated The doctor retired and there's no one to take over the project 2026-06-29
NCT03506373 Terminated Terminated Discontinued after interim analysis for futility; no treatment successes observed in the first 12 patients 2026-02-03
NCT02298816 Withdrawn Withdrawn No patients enrolled 2025-12-31
NCT03246906 Terminated Terminated Study was terminated prior to enrolling protocol target accrual goal due to declining accrual and Data Safety Monitoring Board recommendations. 2025-05-03
NCT01198067 Terminated Terminated \<75% participant accrual 2025-05-02
NCT05537766 Terminated Terminated Sponsor decision to terminate study. 2025-01-27
NCT04635683 Withdrawn Withdrawn Investigational Drug removed from the market 2024-12-31
NCT03333486 Terminated Terminated Futility analysis determined closure 2023-08-28
NCT04804254 Terminated Terminated Strategic considerations 2023-01-11
NCT03225716 Terminated Terminated Sponsor decision to end follow-up early 2022-12-31

Trial records