Skip to content
Pharma Observatory a Scientist.com platform

Condition (MeSH)

Waldenstrom Macroglobulinemia

ClinicalTrials.gov lists 520 trials for Waldenstrom Macroglobulinemia or a narrower condition beneath it. National Cancer Institute (NCI) has the most among industry and government lead sponsors, with 51.

MeSH D008258 Neoplasms Neoplasm C04 C14 C15 C20 Listed by Orphanet as a rare disease
Email me this page as a PDF

Search trials, drugs, companies and investigators, and follow each one to the others. Individual plan, $249/seat/mo. Card required.

Phase 1 Phase 1 153 Phase 1/2 Phase 1/2 62 Phase 2 Phase 2 188 Phase 2/3 Phase 2/3 2 Phase 3 Phase 3 29 Phase 4 Phase 4 8 Not applicable Not applicable 32 Observational Observational 42 Phase 1 Phase 1 153 Phase 1/2 Phase 1/2 62 Phase 2 Phase 2 188 Phase 2/3 Phase 2/3 2 Phase 3 Phase 3 29 Phase 4 Phase 4 8 Not applicable Not applicable 32 Observational Observational 42

Trials per phase (log scale)

2 more sections with rows

520 of the 520 trials are tagged with Waldenstrom Macroglobulinemia itself; the rest are tagged with a narrower condition beneath it.

Get the Waldenstrom Macroglobulinemia report as a PDF

The PDF carries up to 50 rows of each section, where the page shows the first few, with the sources listed. Enter a work email and confirm it from the message we send, and the report follows by email.

When did Waldenstrom Macroglobulinemia trials start? (37)

Shown in this order: most recent first, 1990 onwards. 2026 is partial: trials registered with a 2026 start date so far, not a full year. 2 trials are registered to start in 2027 or later, and not listed here.

Trials over time, by phase

  • Phase 1
  • Phase 1/2
  • Phase 2
  • Phase 2/3
  • Phase 3
  • Phase 4
  • Not applicable
  • Observational
  • Other (Expanded access, Unknown)

Top of scale: 27

1990: 1 trials (Phase 2 1) 1991: 1 trials (Phase 2 1) 1992: 4 trials (Phase 2 2, Phase 1/2 1, Phase 1 1) 1993: 1 trials (Observational 1) 1994: 1 trials (Phase 2 1) 1995: 2 trials (Phase 3 1, Phase 2 1) 1996: 3 trials (Phase 2 2, Phase 1 1) 1997: 11 trials (Not applicable 2, Phase 3 2, Phase 2 2, Phase 1/2 1, Phase 1 4) 1998: 22 trials (Phase 4 1, Phase 3 1, Phase 2/3 1, Phase 2 12, Phase 1/2 1, Phase 1 6) 1999: 24 trials (Not applicable 3, Phase 3 2, Phase 2 10, Phase 1/2 2, Phase 1 7) 2000: 12 trials (Not applicable 1, Phase 3 2, Phase 2 7, Phase 1/2 2) 2001: 14 trials (Not applicable 3, Phase 3 1, Phase 2 5, Phase 1/2 2, Phase 1 3) 2002: 16 trials (Observational 2, Not applicable 2, Phase 2 6, Phase 1/2 1, Phase 1 5) 2003: 20 trials (Observational 2, Not applicable 1, Phase 3 2, Phase 2 6, Phase 1/2 4, Phase 1 5) 2004: 23 trials (Observational 1, Not applicable 1, Phase 2 11, Phase 1/2 2, Phase 1 8) 2005: 27 trials (Observational 4, Phase 3 1, Phase 2 9, Phase 1/2 6, Phase 1 7) 2006: 19 trials (Observational 1, Not applicable 2, Phase 3 2, Phase 2 8, Phase 1 6) 2007: 27 trials (Other 1, Observational 2, Not applicable 2, Phase 3 3, Phase 2 7, Phase 1/2 3, Phase 1 9) 2008: 23 trials (Observational 4, Not applicable 1, Phase 3 2, Phase 2 6, Phase 1/2 2, Phase 1 8) 2009: 27 trials (Other 1, Observational 4, Not applicable 2, Phase 3 1, Phase 2 7, Phase 1/2 4, Phase 1 8) 2010: 20 trials (Observational 1, Phase 3 1, Phase 2 7, Phase 1/2 2, Phase 1 9) 2011: 17 trials (Not applicable 2, Phase 2 8, Phase 1/2 4, Phase 1 3) 2012: 14 trials (Phase 2 6, Phase 1/2 1, Phase 1 7) 2013: 18 trials (Phase 3 4, Phase 2 6, Phase 1/2 3, Phase 1 5) 2014: 18 trials (Observational 2, Phase 3 1, Phase 2 4, Phase 1/2 3, Phase 1 8) 2015: 8 trials (Not applicable 1, Phase 2 4, Phase 1/2 1, Phase 1 2) 2016: 14 trials (Not applicable 1, Phase 4 3, Phase 3 1, Phase 2 4, Phase 1/2 2, Phase 1 3) 2017: 20 trials (Phase 3 1, Phase 2 10, Phase 1/2 6, Phase 1 3) 2018: 3 trials (Observational 1, Phase 2 2) 2019: 12 trials (Phase 4 1, Phase 2 5, Phase 1/2 1, Phase 1 5) 2020: 13 trials (Phase 2/3 1, Phase 2 4, Phase 1/2 2, Phase 1 6) 2021: 19 trials (Observational 5, Not applicable 1, Phase 2 4, Phase 1/2 1, Phase 1 8) 2022: 9 trials (Observational 1, Not applicable 1, Phase 2 3, Phase 1/2 1, Phase 1 3) 2023: 12 trials (Observational 3, Not applicable 1, Phase 2 4, Phase 1 4) 2024: 9 trials (Observational 5, Phase 2 2, Phase 1 2) 2025: 14 trials (Observational 2, Not applicable 2, Phase 4 1, Phase 2 5, Phase 1 4)

Trials registered with a start date in each year, 1990 onward, stacked by phase (complete years only; the current partial year is in the table's notes). The table below lists the same counts, most recent years first.

Trials started per year
Year Trials started
2026 (partial year) 13
2025 14
2024 9
2023 12
2022 9
2021 19
2020 13
2019 12
2018 3
2017 20

Showing 10 of 37 earlier years.

Start your 14-day trial Email me all 37 earlier years as a PDF

Forecast of the full-year count, planned starts included, not rows of this table: about 15 trials starting in 2026 and about 14 in 2027, the average of the 24 months through September, counted as registered and carried forward. The rows of this table count trials registered so far with a start date in the year, planned starts included; the forecast looks ahead to the count for the whole year, so it is not the same measure. In backtests on conditions of this size, forecasts made part-way through the year were typically off by about 28 percent and ran about 20 percent low against the count later registered; forecasts made before the year began were off by about 39 percent and ran about 37 percent low against the count later registered. These figures rest on a few years of history (target years from 2018 to 2025, varying by figure), place each condition as it is mapped today rather than as it was then, and score against a count for the newest year that was still being revised. Only condition series were scored.

Prediction, not a registry value: median of 36 months from start to primary completion across 34 trials still running at the 2026-10-09 snapshot; registered median of 46 months across 390 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.

What phase are Waldenstrom Macroglobulinemia trials in? (10)

Shown in this order: phase order.

Show the trials by phase as a table
Trials by phase
Phase Trials
Phase 1 153
Phase 1/2 62
Phase 2 188
Phase 2/3 2
Phase 3 29
Phase 4 8
Not applicable 32
Observational 42
Expanded access 3
Unknown 1

How many Waldenstrom Macroglobulinemia trials are recruiting? (10)

Shown in this order: the order a trial moves through, from not yet recruiting to unknown.

Trials by recruitment status
Status Trials
Not yet recruiting 12
Recruiting 45
Enrolling by invitation 1
Active, not recruiting 30
Completed 307

Showing 5 of 10 statuses.

Start your 14-day trial Email me all 10 statuses as a PDF

Status as registered on ClinicalTrials.gov. Each trial is counted once.

Which lead sponsors run the most Waldenstrom Macroglobulinemia trials? (3)

Shown in this order: most trials first. Lead sponsors the registry classes as industry, NIH, US federal, other government or network. A hospital or university the registry files as government can appear here. Sponsors it classes as academic or other are shown on the full page, because that class cannot separate an institution from a named investigator.

Lead sponsors
Sponsor Trials
National Cancer Institute (NCI) 51
BeiGene 8
SWOG Cancer Research Network 8

Which drugs are being tested for Waldenstrom Macroglobulinemia? (5)

Shown in this order: most trials first. One row per drug, matched to a single molecule under all its names, ranked by number of trials.

Drugs tested
Drug Trials
RITUXIMAB 90
Cytoxan 60
IBRUTINIB 25
Busulfan 23
Melphalan 20

Interventions as named in the registry (10)

Shown in this order: most trials first.

Interventions as named in the registry
Drug Trials
SQ Fludarabine 60
Peripheral blood stem cell transplantation 52
Mofetil Mycophenolate 34
Total Body Irradiation 33
Allogeneic (Allo) hematopoietic stem cell transplantation 27

Showing 5 of 10 interventions.

Start your 14-day trial Email me all 10 interventions as a PDF

These are the names sponsors entered on ClinicalTrials.gov that we have not matched to a single molecule. Some are drugs under a brand or code name; others are procedures, doses or arm labels.

Which serious adverse events do Waldenstrom Macroglobulinemia trials report? (10)

Shown in this order: most subjects affected first. Subjects affected summed across trials, a cross-trial signal, not a rate.

Serious adverse events in this condition's trials
Adverse event Subjects affected Trials
Pneumonia 253 37
Febrile neutropenia 213 43
Pyrexia 152 17
Platelet count decreased 144 20
Sepsis 132 35

Showing 5 of 10 adverse events.

Start your 14-day trial Email me all 10 adverse events as a PDF

Which publications cover Waldenstrom Macroglobulinemia? (91)

Shown in this order: major-topic papers first, then newest, then most linked to trials.

Publications
Publication Journal Year Major topic
Ibrutinib plus rituximab vs ibrutinib monotherapy in patients with Waldenström macroglobulinemia: a pooled analysis. Blood advances 2025 Yes
Outcomes after transition from ibrutinib to zanubrutinib in patients with Waldenström macroglobulinemia from the ASPEN study. Blood advances 2025 Yes
Six-year follow-up of phase II study exploring chemo-free treatment association with idelalisib and obinutuzumab in symptomatic relapsed/ refractory patients with Waldenström's macroglobulinemia. Annals of hematology 2025 Yes
Peripheral neuropathy in the phase 3 ASPEN study of Bruton tyrosine kinase inhibitors for Waldenström macroglobulinemia. Blood advances 2025 Yes
Prospective study of immunogenicity to SARS-CoV-2 booster vaccines in multiple myeloma and Waldenström macroglobulinemia. Blood advances 2025 Yes

Showing 5 of 91 publications.

Start your 14-day trial Email me the first 50 publications as a PDF

Prevalence (21)

Shown in this order: jurisdiction, then disease name. Orphanet observation for the mapped ORPHA concept; the mapping relation column says whether that concept is exactly this condition or broader or narrower.

Prevalence
Orphanet disease Type Class Geography Jurisdiction Mapping relation
Waldenström macroglobulinemia Annual incidence 1-9 / 1 000 000 United Kingdom GB Exact
Waldenström macroglobulinemia Annual incidence 1-9 / 1 000 000 United Kingdom GB Unspecified
Waldenström macroglobulinemia Annual incidence 1-9 / 1 000 000 United Kingdom GB Exact
Waldenström macroglobulinemia Annual incidence 1-9 / 1 000 000 United States US Exact
Waldenström macroglobulinemia Annual incidence 1-9 / 1 000 000 United States US Unspecified

Showing 5 of 21 prevalence observations.

Start your 14-day trial Email me all 21 prevalence observations as a PDF

Rare-disease identity (2)

Shown in this order: exact, broader, narrower, other.

Rare-disease identity
Orphanet disease ORPHA code Relation Source
Waldenström macroglobulinemia 33226 Exact Orphanet Rare Disease Ontology (ORDO)
Waldenström macroglobulinemia 33226 Exact Orphadata Science knowledge files, Product 1

Not on this page

Not measured: delivery record.

Checked, with nothing recorded for this condition: sub-conditions in mesh.

Browse all condition evidence pages