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Blood & Immune

Polycythemia Vera (PV) Clinical Trials and Treatments

Also called PV, Primary Polycythemia, Erythremia

Polycythemia Vera is one of the myeloproliferative neoplasms, a group of blood cancers in which bone marrow cells multiply uncontrollably. In PV, the overproduction of red blood cells increases blood volume and viscosity (thickness), which impairs blood flow and oxygen delivery despite elevated hemoglobin levels.

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About Polycythemia Vera

Polycythemia Vera is one of the myeloproliferative neoplasms, a group of blood cancers in which bone marrow cells multiply uncontrollably. In PV, the overproduction of red blood cells increases blood volume and viscosity (thickness), which impairs blood flow and oxygen delivery despite elevated hemoglobin levels. The high blood viscosity also increases risk of blood clots (thrombosis) and bleeding complications. About 95% of PV patients carry the JAK2 V617F mutation, with most remaining cases having mutations in CALR or MPL genes.

These mutations cause uncontrolled cell growth independent of normal growth signal regulation. Patients often present with constitutional symptoms including fatigue, headaches, and visual disturbances. The pathognomonic symptom of aquagenic pruritus (itching triggered by water contact) occurs in about half of patients. Splenomegaly (enlarged spleen) occurs in 75% of patients. Long-term complications include risk of thrombotic events (stroke, heart attack, deep vein thrombosis) and potential transformation to myelofibrosis or acute leukemia.

Common Symptoms of Polycythemia Vera

Recognizing the signs of Polycythemia Vera 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
  • Headaches and dizziness
  • Vision disturbances or blurred vision
  • Itching, especially after bathing
  • Burning sensations in the hands and feet
  • Easy bruising or bleeding

Who Polycythemia Vera Affects

Polycythemia Vera typically affects older adults, with median age of diagnosis around 65 years, though it can occur at younger ages. It affects males and females approximately equally. The disease occurs across all racial and ethnic groups. Approximately 95% of PV patients carry the JAK2 V617F mutation, which can be detected through blood testing.

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FDA-Approved Treatments for Polycythemia Vera

There is currently 1 FDA-approved medication for Polycythemia Vera. These therapies represent the current standard of care and may be used alongside or compared against investigational treatments in active clinical trials.

rusfertide
Takeda (discovered by Protagonist Therapeutics)
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Source: openFDA drug labeling data. This list may not include all treatments. Always consult your doctor.

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Help Paying for Polycythemia Vera Treatment

Charity funds and drugmaker programs for Polycythemia Vera, 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 · CancerCare Co-Payment Assistance Foundation
    Myeloproliferative Neoplasms fund
    Open

    Pays for: Copays, coinsurance and deductibles for treatment, up to $7,000 per year. Requires Medicare, Medicaid or TRICARE.

    The foundation says: “Status: Open. Grant Amount: $7,000 (Initial Grant Amount $7,000; Program CAP Amount $10,000)”
  • From a charity · TotalAssist (formerly PAN Foundation)
    Myeloproliferative Neoplasms fund
    Open

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

  • From a charity · The Assistance Fund
    Myeloproliferative Neoplasms (MPN) fund
    Waitlist

    Pays for: Copays, coinsurance, deductibles and other health-related expenses.

    The foundation says: “WAITLIST — Accepting Waitlist Patients. TAF is currently accepting requests to join the enrollment waitlist for this program. Waitlists a…”
Status as each foundation showed it on September 28, 2026.
Drugmaker programs
Open a medicine for who qualifies, by insurance type.

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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.

The JAK2 gene page lists every condition Orphanet links to the gene and the open trials that name it.

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Trusted Polycythemia Vera Resources

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

Active Clinical Trials for Polycythemia Vera

Use this Polycythemia Vera clinical trial finder to see the 18 studies recruiting patients and 2 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.

61 active trials worldwide
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RECRUITINGPHASE3Recently updatedNCT07357727

A Phase 3 Study of Pelabresib (DAK539) and Ruxolitinib in Myelofibrosis (MF)

Intervention: Pelabresib, Ruxolitinib, Placebo

Sponsor: Novartis Pharmaceuticals

The purpose of this trial is to evaluate whether treatment with pelabresib in combination with ruxolitinib leads to improved clinical outcomes compared to ruxolitinib alone in patients with primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (PPV-MF), or post-essent...

Ages 18 Years+103 locations
Started May 2026Updated todayEst. May 2028 (~1y 7m)
NOT YET RECRUITINGNARecently updatedNCT07349134

WELL-MPN: A Digital Lifestyle App for Patients With MPNs

Intervention: WELL-MPN Digital App

Sponsor: Brigham and Women's Hospital

The goal of this clinical trial is to learn whether a digital lifestyle app can help improve diet, physical activity, symptoms, emotional well-being, and quality of life in adults with myeloproliferative neoplasms (MPNs).

The main questions it aims to answer are:

Ages 18 Years+1 location
Started Jan 2027Updated todayEst. Jul 2029 (~2y 10m)
RECRUITINGPHASE2Recently updatedNCT06661915

A Randomized Study of ASTX727 With or Without Iadademstat in Advanced Myeloproliferative Neoplasms (MPNs)

Intervention: Biospecimen Collection, Bone Marrow Aspiration, Bone Marrow Biopsy, Decitabine and Cedazuridine, Iadademstat

Sponsor: National Cancer Institute (NCI)

This phase II trial compares the effect of ASTX727 in combination with iadademstat to ASTX727 alone in treating patients with accelerated or blast phase Philadelphia chromosome negative myeloproliferative neoplasms (MPNs). ASTX727 is a combination of two drugs, cedazuridine and d...

Ages 18 Years+31 locations
Started Aug 2025Updated todayEst. Dec 2027 (~1y 3m)
RECRUITINGPHASE1, PHASE2Recently updatedNCT05320198

Study of DISC-0974 (RALLY-MF) in Participants With Myelofibrosis or Myelodysplastic Syndrome and Anemia

Intervention: DISC-0974

Sponsor: Disc Medicine, Inc

This phase 1b/2a open-label study will evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and clinical activity of DISC-0974 as well as categorize the effects on hematologic response in participants with myelofibrosis or myelodysplastic syndrome and anemia.

Ages 18 Years+32 locations
Started Jun 2022Updated 6 days agoEst. May 2027 (~8 months)
RECRUITINGPHASE1Recently updatedNCT07469891

A Phase 1 Study of PRT12396 in Participants With Select Myeloproliferative Neoplasms

Intervention: PRT12396

Sponsor: Prelude Therapeutics Incorporated

This is a first-in-human, open-label, multi-center Phase 1 study designed to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of PRT12396 in participants with high-risk polycythemia vera (PV) and myelofibrosis (MF), and to determine the maximum tolera...

Ages 18 Years+9 locations
Started Apr 2026Updated 1 week agoEst. Apr 2028 (~1y 7m)
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Active trial locations91 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 Polycythemia Vera 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 Polycythemia Vera 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 Polycythemia Vera may find relevant research, shared treatment pathways, or overlapping clinical trials among these related conditions.

Companies Developing Polycythemia Vera Treatments

12 pharmaceutical companies have Polycythemia Vera in their rare disease portfolio

Frequently Asked Questions About Polycythemia Vera