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Blood & Immune

Waldenström Macroglobulinemia (WM) Clinical Trials

Also called WM, Lymphoplasmacytic Lymphoma, Waldenström's Macroglobulinemia

Waldenström Macroglobulinemia is a type of non-Hodgkin lymphoma arising from malignant B-lymphocytes (specifically, lymphoplasmacytic cells) that produce large quantities of monoclonal IgM, a high-molecular-weight immunoglobulin. The buildup of this protein in the blood causes hyperviscosity syndrome, which can lead to serious complications including vision loss, neurological symptoms, and bleeding.

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About Waldenström Macroglobulinemia

Waldenström Macroglobulinemia is a type of non-Hodgkin lymphoma arising from malignant B-lymphocytes (specifically, lymphoplasmacytic cells) that produce large quantities of monoclonal IgM, a high-molecular-weight immunoglobulin. The buildup of this protein in the blood causes hyperviscosity syndrome, which can lead to serious complications including vision loss, neurological symptoms, and bleeding.

The excess IgM can deposit in various tissues, causing cryoglobulinemia (cold-precipitating immune complexes) that damages blood vessels and tissues.

Patients often present with fatigue, anemia, and bleeding manifestations. Some individuals have been found to carry the MYD88 L265P mutation, which drives disease progression. Bone marrow infiltration by the malignant cells is common. The disease usually follows an indolent course with variable progression, though some patients may transform to more aggressive lymphomas. Symptoms related to hyperviscosity can include vision problems (from retinal bleeding), headaches, neurological symptoms, and heart problems.

Common Symptoms of Waldenström Macroglobulinemia

Recognizing the signs of Waldenström Macroglobulinemia early can lead to faster diagnosis and better outcomes. Symptoms may vary in severity from person to person. If you or a loved one are experiencing any of the following, consider speaking with a specialist.

  • Fatigue and weakness
  • Bleeding or bruising easily
  • Blurred or cloudy vision
  • Headaches and dizziness
  • Numbness, tingling, or neuropathy in hands and feet
  • Enlarged lymph nodes, spleen, or liver

Who Waldenström Macroglobulinemia Affects

Waldenström Macroglobulinemia typically affects older adults, with median age of diagnosis around 68 years, though cases can occur at younger ages. It affects males more frequently than females, with a male-to-female ratio of approximately 2:1.

The disease occurs across all racial and ethnic groups, though it is more common in Caucasians. About 20% of cases are familial, indicating a genetic predisposition.

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Help Paying for Waldenström Macroglobulinemia Treatment

Charity funds and drugmaker programs for Waldenström Macroglobulinemia, checked at the source. Pick your insurance to see what fits.

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Charity funds
  • From a charity · Blood Cancer United (formerly The Leukemia & Lymphoma Society)
    Clinical Trials Co-Pay Fund (all blood cancers) fund
    Open

    Pays for: Insurance premiums and treatment-related copays, deductibles and coinsurance, up to $3,500 per year. Requires health insurance (any kind).

  • From a charity · Blood Cancer United (formerly The Leukemia & Lymphoma Society)
    Patient Aid Program fund
    Open

    Pays for: One-time $100 stipend for non-medical expenses (transportation, food, housing, utilities); no income or insurance requirement, up to $100 per year.

    The foundation says: “CURRENT FUND STATUS: Open. Fund is currently Open.”
  • From a charity · HealthWell Foundation
    Waldenstrom Macroglobulinemia fund
    Open

    Pays for: Copays, premiums or other treatment costs.

  • From a charity · NORD RareCare
    IWMF Waldenstrom Macroglobulinemia Travel and Lodging Assistance fund
    Open

    Pays for: Travel and lodging for care.

    The foundation says: “Accepting Applications”
  • From a charity · TotalAssist (formerly PAN Foundation)
    Waldenstrom Macroglobulinemia fund
    Open

    Pays for: Out-of-pocket costs for approved medications, up to $9,500 per year. Requires health insurance (any kind).

Status as each foundation showed it on September 28, 2026.

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Side Effect Explorer

Real-world side effect reports from the FDA Adverse Event Reporting System (FAERS). Includes both FDA-approved drugs and investigational therapies from active clinical trials. Click any drug to see what patients reported.

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Genetic Testing

Genetic testing can confirm a diagnosis, guide treatment decisions, and identify family members who may be at risk.

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Trusted Waldenström Macroglobulinemia Resources

Reputable organizations and medical references for learning more about Waldenström Macroglobulinemia, including disease registries, foundation resources, and clinical guidelines.

Active Clinical Trials for Waldenström Macroglobulinemia

Use this Waldenström Macroglobulinemia clinical trial finder to see the 14 studies recruiting patients and 4 opening soon in the United States and worldwide, with eligibility criteria in plain English. These studies play a critical role in advancing care for blood & immune conditions and may offer access to treatments not yet widely available. Each trial below is sourced directly from ClinicalTrials.gov, with eligibility criteria translated into plain English to help patients and caregivers evaluate whether a study may be a fit.

TrialsSite mapPipeline timeline

Note: Trial recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator before making plans.

18 active trials worldwide
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RECRUITINGPHASE1, PHASE2Recently updatedNCT05006716

A Dose-Escalation and Expansion Study of Tacabrutideg (BGB-16673) in Participants With B-Cell Malignancies

Intervention: Tacabrutideg

Sponsor: BeOne Medicines

Study consists of two main parts to explore tacabrutideg recommended dosing, a Phase 1 monotherapy dose finding comprised of monotherapy dose escalation and monotherapy safety expansion of selected doses, and a Phase 2 (expansion cohorts)

Ages 18 Years+114 locations
Started Sep 2021Updated yesterdayEst. Nov 2026 (~2 months)
RECRUITINGPHASE2Recently updatedNCT06561347

Zanubrutinib, Bendamustine, Rituximab Prev. Untreated WM

Intervention: Zanubrutinib, Bendamustine, Rituximab

Sponsor: Massachusetts General Hospital · BeOne Medicines

The purpose of this study is to determine the very good partial response (VGPR) or better rate in participants with Waldenström macroglobulinemia (WM).

The names of the study drugs involved in this study are as follows: zanubrutinib, bendamustine, and rituximab.

Ages 18 Years+5 locations
Started Dec 2024Updated 4 weeks agoEst. Dec 2026 (~2 months)
RECRUITINGPHASE1, PHASE2Recently updatedNCT05294731

Treatment of Chinese Participants With B-Cell Malignancies With BGB-16673, a Bruton Tyrosine Kinase-Targeted Protein-Degrader

Intervention: BGB-16673

Sponsor: BeiGene

This study aims to explore the recommended phase 2 dose and evaluate the safety, tolerability and preliminary antitumor activity of BGB-16673 monotherapy at the recommended Phase 2 dose for the selected B-cell malignancy expansion cohorts

Ages 18 Years+29 locations
Started May 2022Updated 1 month agoEst. Jan 2029 (~2y 4m)
RECRUITINGRecently updatedNCT05640102

Observational Study Evaluating the Efficacy and Safety of Zanubrutinib in Participants With Waldenström Macroglobulinemia

Intervention: Zanubrutinib

Sponsor: BeiGene

This is a hybrid (retrospective and prospective) non-interventional registry study to further describe the clinical profile of zanubrutinib in Waldenström macroglobulinemia (WM) participants with and without specific mutations and from racial and ethnic minority groups. Data coll...

Ages 18 Years+8 locations
Started Mar 2023Updated 1 month agoEst. Dec 2026 (~3 months)
RECRUITINGPHASE1Recently updatedNCT06708897

A Study of Lonitoclax (ZE50-0134) in Relapsed or Refractory B-cell Malignancies

Intervention: Lonitoclax (ZE50-0134)

Sponsor: Lomond Therapeutics Holdings, Inc.

This Phase 1, open-label, multicenter study is evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary antitumor activity of lonitoclax (ZE50-0134) in adults with relapsed or refractory chronic lymphocytic leukemia (CLL), small lymphocytic lymphom...

Ages 18 Years+5 locations
Started Apr 2025Updated 2 months agoEst. Jan 2028 (~1y 4m)
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Active trial locations46 cities in the US
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Trial Pipeline

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Data from ClinicalTrials.gov, U.S. National Library of Medicine.
Always talk to your doctor before considering a clinical trial.

Patient Communities

Connect with other Waldenström Macroglobulinemia patients, caregivers, and advocacy groups across Facebook groups, Reddit communities, and YouTube channels. These patient communities offer peer support, shared experiences, caregiver resources, and real-time discussion about Waldenström Macroglobulinemia treatments, clinical trial participation, and day-to-day disease management.

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Related Blood & Immune Conditions

Other rare diseases in the blood & immune category. Patients with Waldenström Macroglobulinemia may find relevant research, shared treatment pathways, or overlapping clinical trials among these related conditions.

Companies Developing Waldenström Macroglobulinemia Treatments

12 pharmaceutical companies have Waldenström Macroglobulinemia in their rare disease portfolio

Frequently Asked Questions About Waldenström Macroglobulinemia