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Disease

Waldenstrom macroglobulinemia

Late-stage therapeutic development
Also known as Waldenstrom's macroglobulinemia, Waldenström Macroglobulinemia, Waldenström macroglobulinemia, lymphoplasmacytic lymphoma with IgM gammopathy+4 more

Waldenstrom's macroglobulinemia, Waldenström Macroglobulinemia, Waldenström macroglobulinemia, lymphoplasmacytic lymphoma with IgM gammopathy, Waldenstroem's macroglobulinemia, Waldenstrom's macroglobulinaemia, Waldenstrom's syndrome, macroglobulinemia of Waldenstrom.

21
Clinical trials
3
Associated genes
1
Related proteins
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this disease.

PCI-32765CAN3001: A Phase 3b, Multicenter, Open-label, PCI-32765 (Ibrutinib) Long-term Extension Study

Clinical trial2026-10-01Status changed to Active, not recruiting · ClinicalTrials.gov

A Phase 4, Single-Arm, Open-Label, Multicenter Study to Evaluate the Efficacy and Safety of Zanubrutinib in Chinese Patients With Treatment-Naive Waldenström Macroglobulinemia

Clinical trial2026-08-10Status changed to Active, not recruiting · ClinicalTrials.gov

A Phase 2 Study of Venetoclax Monotherapy in Japanese Subjects With Relapsed or Refractory Waldenström Macroglobulinemia/Lymphoplasmacytic Lymphoma

Clinical trial2026-07-31Status changed to Active, not recruiting · ClinicalTrials.gov

A Phase 1 Study of Oral LOXO-338, a Selective BCL-2 Inhibitor, in Patients With Advanced Hematologic Malignancies

Clinical trial2026-07-17Status changed to Completed · ClinicalTrials.gov

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

Executive briefingUpdating summary…Momentum: Low
Key developments
  • 3 clinical trials expected to report results, the earliest in Q2 2027.
Upcoming Milestones
Major developments
Upcoming trial readoutImportant
A Phase II Study Evaluating Venetoclax and Pirtobrutinib in Previously Treated Waldenström Macroglobulinemia
Results expected Q1 20292026-05-28
Upcoming trial readoutImportant
Efficacy of Venetoclax in Combination With Rituximab in Waldenström's Macroglobulinemia
Results expected Q1 20282026-05-05
Clinical Milestones6View
Results expected 3
Recently completed 2
Other updates 1
Activity timeline6

Clinical trials

16 sponsors · 0 new · 0 completed in the last 12 months (net -1)

The current development programme across all trial phases.

Clinical programme
21
All trials
6
Active
2
Late-stage
6
Completed
Recently completed

A Single Arm, Multicenter, Phase 4 Study of the Bruton's Tyrosine Kinase (BTK) Inhibitor, Ibrutinib (PCI-32765) in Chinese Subjects With Relapse or Refractory Waldenström's Macroglobulinemia

Phase 4 · Completed · Janssen Research & Development, LLC

A Phase II Trial to Investigate the Safety and Efficacy of Rituximab and Pembrolizumab in Relapsed/Refractory Waldenström's Macroglobulinaemia

Phase 2 · Completed · University College, London

Phase 2 Study of Bruton's Tyrosine Kinase (BTK) Inhibitor, Ibrutinib (PCI-32765) in Combination With Rituximab, in Japanese Patients With Waldenstrom's Macroglobulinemia (WM)

Phase 2 · Completed · Janssen Pharmaceutical K.K.

An Open Label Non-randomized Phase II Study Exploring "Chemo-free " Treatment Association With Idelalisib + Obinutuzumab in Patient With Relapsed/Refractory Waldenstrom's Macroglobulinemia

Phase 2 · Completed · French Innovative Leukemia Organisation

A Randomized Phase I/II Study of Bortezomib, Rituximab, Dexamethasone and Temsirolimus in Patients With Relapsed Waldenstrom Macroglobulinemia and Relapsed/Refractory Mantle Cell, Follicular, Marginal Zone or Small Lymphocytic Lymphomas (Phase I), and Untreated/Relapsed Waldenstrom Macroglobulinemia (Phase II)

Phase 1/2 · Completed · National Cancer Institute (NCI)

A Phase I, Open Label, Dose Escalation Study of Oral Administration of Single Agent MLN0128 in Subjects With Relapsed or Refractory Multiple Myeloma or Waldenstrom Macroglobulinemia

Phase 1 · Completed · Millennium Pharmaceuticals, Inc.

Associated genes

3 matches

Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.

BTK0.56MYD880.47GLRX50.08

Disease biology

1 match

Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.

Tyrosine-protein kinase BTKvia BTK0.56

Reference

Authoritative identity, definition & identifiers.

Defined in MeSH

A lymphoproliferative disorder characterized by pleomorphic B-LYMPHOCYTES including PLASMA CELLS, with increased levels of monoclonal serum IMMUNOGLOBULIN M. There is lymphoplasmacytic cells infiltration into bone marrow and often other tissues, also known as lymphoplasmacytic lymphoma. Clinical features include ANEMIA; HEMORRHAGES; and hyperviscosity.

Synonyms

Waldenstrom's macroglobulinemia, Waldenström Macroglobulinemia, Waldenström macroglobulinemia, lymphoplasmacytic lymphoma with IgM gammopathy, Waldenstroem's macroglobulinemia, Waldenstrom's macroglobulinaemia, Waldenstrom's syndrome, macroglobulinemia of Waldenstrom

References & data sources
  • Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
  • Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
  • Related entities are derived from literature co-mention (studied together) — associative, not causal.