Mode
Home
Research
Clinical Trials Drug Pipeline Weekly Digests
Reference
Conditions Medications
Community
Questions
Text Size
Log in / Sign up
Home › Conditions › Waldenstroms Macroglobulinemia

Waldenstroms Macroglobulinemia

Part of Plasma cell dyscrasias

1 published article · Updated continuously

Clinical Trial Landscape

Clinical Trials for Waldenstroms Macroglobulinemia

18 trials tracked for Waldenstroms Macroglobulinemia: 2 in phase 3 or 4 and 2 with published results. The most-cited published study has 351 citations.

18Trials tracked
2Phase 3 & 4
0Recruiting
2With published results
Phase distribution
Phase 3 2 Phase 2 14 Phase 1 2
  1. Phase 3 Ibrutinib With Rituximab in Adults With Waldenström's Macroglobulinemia Completed · 351 cited
  2. Phase 3 A Study Comparing BGB-3111 and Ibrutinib in Participants With Waldenström's Macroglobulinemia (WM) Completed · 38 cited
  3. Phase 2 LBH589 in Relapsed or Relapsed and Refractory Waldenstrom's Macroglobulinemia Completed
  4. Phase 2 Perifosine in Patients With Relapsed/Refractory Waldenstrom's Macroglobulinemia Completed
  5. Phase 2 A Study for Patients That Have Been Previously Been Treated in Waldenstrom's Macroglobulinemia or Multiple Myeloma Completed
  6. Phase 2 Ibrutinib (PCI-32765) in Waldenstrom's Macroglobulinemia Completed
Show 12 more trials
  1. Phase 2 Study of the Combination of Bortezomib, Dexamethasone, and Rituximab in Patients With Waldenstroms Macroglobulinemia Completed
  2. Phase 2 Carfilzomib, Rituximab and Dexamethasone in Waldenstrom's Macroglobulinemia Completed
  3. Phase 2 Study of Ibrutinib in Patients With Symptomatic, Previously Untreated Waldenstrom's Macroglobulinemia, and Impact on Tumor Genomic Evolution Using Whole Genome Sequencing Completed
  4. Phase 2 Everolimus (RAD001) in Primary Therapy of Waldenstrom's Macroglobulinemia Completed
  5. Phase 2 Extension Study of IMO-8400 in Patients With Waldenström's Macroglobulinemia Who Completed Study 8400-401 Completed
  6. Phase 2 Trial of Ixazomib, Dexamethasone and Rituximab in Patients With Untreated Waldenstrom's Macroglobulinemia Completed
  7. Phase 2 Thalidomide and Rituximab in Waldenstrom's Macroglobulinemia Completed
  8. Phase 2 Combination Bortezomib and Rituximab in Patients With Waldenstrom's Macroglobulinemia (WM) Completed
  9. Phase 2 Study of Pralatrexate & Gemcitabine With B12 & Folic Acid to Treat Relapsed/Refractory Lymphoproliferative Malignancies Completed
  10. Phase 2 Study of High-dose Influenza Vaccine Efficacy by Repeated Dosing IN Gammopathy Patients Completed
  11. Phase 1 Safety Study of the Proteasome Inhibitor PR-171 (Carfilzomib for Injection) in Patients With Hematological Malignancies Completed
  12. Phase 1 Study of MLN8237 in Participants With Advanced Hematological Malignancies Completed

Showing the 18 most-cited and recently-updated of 18 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

What the trials found For clinicians

Waldenstroms Macroglobulinemia: what the trials found

Ibrutinib has demonstrated significant clinical efficacy in patients with Waldenstrom's Macroglobulinemia. Specifically, it showed a statistically significant improvement in Time to Next Treatment (TnT) compared to the control group (87.4 vs 29.4; p<0.0001) 1. Additionally, there was a significantly higher percentage of participants achieving sustained hemoglobin (Hgb) improvement up to 3 years after the last participant was randomized in the Ibrutinib arm compared to the control group (77.3% vs 42.7%; p<0.0001) 1.

BGB-3111 was evaluated for its efficacy in Waldenstrom's Macroglobulinemia. While the Major Response Rate (MRR) as assessed by an Independent Review Committee (IRC) was comparable between groups (77.8% vs 77.5%) 2, the percentage of participants achieving a Complete Response (CR) or Very Good Partial Response (VGPR) as assessed by IRC showed no statistically significant difference between BGB-3111 and the comparator (19.2% vs 28.4%; p=0.0921) 2.

Bortezomib has been evaluated in multiple Phase 2 trials for the treatment of Waldenstrom's Macroglobulinemia [9, 12].

Recent results — preliminary, needs further review

  • LBH589: 24 participants experienced Grade 3 and 4 treatment-related thrombocytopenia 4.
  • Enzasturim: Reported response rates (CR, PR, MR, or MinR) were 7.1% vs 38.1% 5.
  • Perifosine: Reported a treatment-related Grade 3-4 adverse event rate of 35 16.
  • Alisertib: Pharmacokinetic data showed Accumulation Ratios (Rac) and Peak/Trough Ratios for various dosing schedules 17.

For the clinician treating this condition

  • Ibrutinib is associated with significantly longer Time to Next Treatment and higher rates of sustained hemoglobin improvement over a 3-year period compared to the control group.
  • BGB-3111 demonstrated comparable Major Response Rates (MRR) to its comparator, though results for CR/VGPR were not statistically significant.
  • Bortezomib is established in Phase 2 clinical evidence for this condition.

AI synthesis of 6 cited trials, updated Jun 27, 2026. Informational only — not medical advice; trial data sourced from ClinicalTrials.gov. How we use AI.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.
BR regimen shows survival outcomes in newly diagnosed Waldenstroms Macroglobulinemia patients
Hematology
BR regimen shows survival outcomes in newly diagnosed Waldenstroms Macroglobulinemia patients A Rare Blood Cancer With an 8-Year Survival: What Real-World Data Shows
This single-center retrospective analysis evaluated 89 newly diagnosed Waldenstroms Macroglobulinemia patients treated with the BR regimen.
New real-world data shows patients with the rare blood cancer Waldenstrom's macroglobulinemia live a median of over 8 years after diagnosis.
medRxiv Apr 16, 2026