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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.

In plain words, Waldenström macroglobulinemia is a rare blood cancer where abnormal white blood cells make too much of an immune protein called IgM. This extra protein thickens the blood and can cause tiredness, easy bleeding, blurry vision and headaches.

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Waldenström Macroglobulinemia

Where treatments stand, from ClinicalTrials.gov and FDA labels

Also 5 studies with no phase, like observational studies.

FDA review0

No decision pending. FDA calendar

FDA-approved medicines2

Imbruvica · Brukinsa

Waldenström Macroglobulinemia has 2 FDA-approved medicines, 17 open or active clinical trials (14 recruiting), and no pending FDA decision.

Labels checked October 21, 2026. Corticosteroids and immune globulins count once each.Download the data

Latest for Waldenström Macroglobulinemia

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More about Waldenström Macroglobulinemia

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

Keep reading about Waldenström Macroglobulinemia

Patients often present with fatigue, anemia, and bleeding manifestations. About 9 in 10 people with WM carry the MYD88 L265P mutation in their tumor cells, which helps drive the disease (Treon et al., New England Journal of Medicine, 2012). 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.

What are the symptoms of Waldenström Macroglobulinemia?

These are common symptoms of Waldenström Macroglobulinemia, and how strong they are varies from person to person.

  • 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 gets Waldenström Macroglobulinemia?

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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Which medicines are FDA-approved for Waldenström Macroglobulinemia?

There are currently 2 FDA-approved medications for Waldenström Macroglobulinemia.

zanubrutinib
ibrutinib
Pharmacyclics LLC
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Source: openFDA drug labeling data. This list may not include all treatments. Always consult your doctor.

How to get help paying for Waldenström Macroglobulinemia treatment

Charity funds and drugmaker programs for Waldenström Macroglobulinemia, checked at the source.

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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 October 26, 2026.
Drugmaker programs
Open a medicine for who qualifies, by insurance type.

Every program and fund for Waldenström Macroglobulinemia, by insurance →

Side effects people have reported to the FDA

Side effects people have reported to the FDA

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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Diagnosis and genetic testing

Genetic testing for Waldenström Macroglobulinemia

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 3 not yet recruiting in the United States and worldwide, with eligibility criteria in plain English. Of these, 14 test a treatment and 3 are observational studies or registries; each card says which. Every trial comes from ClinicalTrials.gov.

Jump to the trial list, the site map or the pipeline 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.

17 active trials worldwide
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RecruitingTreatment trialPhase 2Recently 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+
Started Dec 2024Updated todayEst. Dec 2026 (17 days)
RecruitingTreatment trialPhase 1/2Recently updatedNCT07249905

Dose Escalation and Dose Expansion Study of MDX2003 in Patients With Different Types of Lymphoma

Intervention: MDX2003

Sponsor: ModeX Therapeutics, An OPKO Health Company

This study is designed to characterize the safety, tolerability, and anti-tumor activity of MDX2003 in patients with different types of lymphoma

Ages 18 Years+
Started Apr 2026Updated 1 week agoEst. Jan 2028 (~1y 2m)
RecruitingTreatment trialPhase 1/2Recently updatedNCT05294731

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

Intervention: Tacabrutideg

Sponsor: BeOne Medicines

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+
Started May 2022Updated 3 weeks agoEst. Jun 2027 (~7 months)
RecruitingTreatment trialPhase 1Recently updatedNCT07424833

A Study of APG-3288 in Relapsed/Refractory Blood Cancers

Intervention: APG-3288

Sponsor: Ascentage Pharma Group Inc.

This is a Phase I, multicenter, open-label, two-stage study of APG-3288 monotherapy, aiming to determine the Maximum Tolerated Dose (MTD) and Recommended Phase 2 Dose (RP2D) of APG-3288 administered orally once daily in patients with relapsed/refractory (R/R) hematologic malignancies.

Ages 18 Years+
Started Mar 2026Updated 4 weeks agoEst. Dec 2029 (~3y 1m)
RecruitingTreatment trialPhase 1/2Recently updatedNCT07420959

ABBV-383 for the Treatment of Relapsed Refractory Waldenström Macroglobulinemia

Intervention: Biospecimen Collection, Bone Marrow Aspiration, Bone Marrow Biopsy, Computed Tomography, Etentamig, Positron Emission Tomography, Questionnaire Administration

Sponsor: Mayo Clinic

This phase I/II trial studies the side effects and best dose of ABBV-383 and to see how well it works in treating patients with Waldenström macroglobulinemia that has come back after a period of improvement (relapsed) or that does not respond to treatment (refractory). ABBV-383 i...

Ages 18 Years+1 location
Expected to start Sep 2026Updated 1 month agoEst. Sep 2036 (~9y 10m)
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Active trial locations43 cities in the US
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Trial Pipeline

May 2021 to Nov 2031
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RecruitingNot yet recruitingDelayed startTodayHover a bar for trial details

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

Waldenström Macroglobulinemia patient communities

Facebook groups, Reddit communities and YouTube channels where Waldenström Macroglobulinemia patients, caregivers and advocacy groups talk.

Companies developing Waldenström Macroglobulinemia treatments

Drugmakers that list Waldenström Macroglobulinemia in their rare disease portfolio, 11 in all.

Questions people ask about Waldenström Macroglobulinemia