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Essential ThrombocythemiaPhase 2NCT04644211

Ruxolitinib in Thrombocythemia and Polycythemia Vera

A Phase 2 study testing Ruxolitinib in people with Essential Thrombocythemia and Polycythemia Vera, run by Massachusetts General Hospital at 4 sites in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial summary

This research is being done to see if the drug ruxolitinib is effective in reducing the symptoms caused by low-risk essential thrombocythemia (ET) and polycythemia vera (PV). This research study involves the study drug Ruxolitinib. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Essential Thrombocythemia, Polycythemia Vera
What is being tested?
Ruxolitinib
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
60 participants
Where
4 sitesUnited States
Study dates
21 Mar 2022to Jul 2034
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Patients who have been diagnosed with essential thrombocythemia or polycythemia vera by World Health Organization 2016 diagnostic criteria.
  • Patients with essential thrombocythemia must be very low (no history of thrombosis, age <60, and no JAK2 mutation), low (no history of thrombosis, age <60, presence of JAK2 mutation), or intermediate risk (no history of thrombosis, age >60, no JAK2 mutation) by IPSET criteria. Patients with polycythemia vera must be low risk (no history of thrombosis and age <60) by NCCN guidelines.
  • Patients with an MPN-SAF TSS (MPN-10) score >10 AND at least one individual feature >5 documented on a separate visit within 3 months prior to study registration, as documented in the clinical record or obtained by clinician. If not previously documented in the electronic medical record, participants must be blinded to purpose of MPN SAF TSS scoring for eligibility determination. Average daily MPN-SAF TSS (MPN-10) score must remain >10 with any individual feature >5 for the week-long baseline assessment prior to ruxolitinib initiation.
  • Patients who have previously received or are receiving cytoreductive therapy (i.e. hydroxyurea, anagrelide, interferon) are eligible for the study if therapy was used for the indication of symptom control, or if therapy was used for pre-operative control of blood counts. If a subject is still receiving cytoreductive therapy at the time of screening and enrollment, there will be a wash-out period from prior cytoreductive therapy at least 7 days prior to ruxolitinib initiation.
  • Age ≥18 years.
  • ECOG performance status ≤2 (Karnofsky ≥60%)
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  • Participants must have adequate organ and marrow function as defined below:
  • leukocytes ≥3,000/mcL
  • absolute neutrophil count ≥1,500/mcL
  • platelets ≥100,000/mcL
  • total bilirubin ≤ institutional upper limit of normal (ULN)
  • AST(SGOT)/ALT(SGPT) ≤3 × institutional ULN
  • creatinine ≤ institutional ULN OR glomerular filtration rate (GFR) ≥60 mL/min/1.73 m2 unless data exists supporting safe use at lower kidney function values, no lower than 30 mL/min/1.73 m2
  • Participants with a prior or concurrent malignancy not receiving treatment for concurrent cancer diagnosis and/or prior concurrent malignancy within 5 years except for basal cell carcinoma or squamous cell carcinoma of the skin.
  • For participants with evidence of chronic hepatitis B virus (HBV) infection, the HBV viral load must be undetectable on suppressive therapy, if indicated.
  • For participants with evidence of chronic human immunodeficiency virus (HIV) infection, they must be negative for HBV DNA, HCV RNA, or hepatitis B surface antigen (BsAg) on suppressive therapy, if indicated.
  • Participants must be previously vaccinated with the Herpes Zoster (Shingles) vaccine or must be willing to start prophylactic Acyclovir 400 mg twice daily (BID) or suitable alternative for duration of treatment with ruxolitinib.
  • Participants with known history or current symptoms of cardiac disease, or history of treatment with cardiotoxic agents, should have a clinical risk assessment of cardiac function using the New York Heart Association Functional Classification. To be eligible for this trial, participants should be class 2B or better.
  • Ability to understand and the willingness to sign a written informed consent document.

Who cannot join

  • Essential thrombocythemia patients who are high risk by IPSET-R criteria (age > 60 with JAK2 V617F mutation and/or history of thrombosis).1 Polycythemia vera patients who are high risk by NCCN guidelines (age > 60 and/or history of thrombosis).
  • Patients with >5% blasts on baseline marrow exam or at any other time in peripheral blood
  • Participants who are receiving any other investigational agents.
  • Participants with a history of splenectomy. Participants may still be eligible after discussion with and approval by the Overall PI, however.
  • History of allergic reactions attributed to compounds of similar chemical or biologic composition to ruxolitinib or excipients of ruxolitinib.
  • Participants requiring any medications or substances that are strong inhibitors or 3A4 isozyme are ineligible. Because the lists of these agents are constantly changing, it is important to regularly consult a frequently-updated medical reference. As part of the enrollment/informed consent procedures, the participant will be counseled on the risk of interactions with other agents, and what to do if new medications need to be prescribed or if the participant is considering a new over-the-counter medicine or herbal product.
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  • Participants with uncontrolled intercurrent illness.
  • Participants with inadequate liver or renal function at screening as evidenced by lab values not meeting criteria
  • Participants with psychiatric illness/social situations that would limit compliance with study requirements.
  • Pregnant women are excluded from this study because ruxolitinib is a Class C agent with the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with ruxolitinib, breastfeeding should be discontinued if the mother is treated with ruxolitinib.
  • The effects of ruxolitinib on the developing human fetus are unknown. Pregnant women and subjects of childbearing potential who are unwilling to take appropriate precautions to avoid becoming pregnant or fathering a child are ineligible. Women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and 4 months after completion of ruxolitinib administration.

Trial locations and contacts

4 sites in 1 country. Contact the study team or a site near you to ask about taking part.

Gabriela Hobbs, MD contact

(617) 726-8748

ghobbs@partners.org

United States4 sites, 4 recruiting

  • Massachusetts General Hospital

    Boston, Massachusetts, 02115

    Gabriela Hobbs, MD, (617) 726-8748, ghobbs@partners.org

    Gabriela Hobbs, MD

    Recruiting

  • Beth-Israel Deaconess Medical Center

    Boston, Massachusetts, 02215

    Jeffrey Zwicker, MD, (617) 667-9920, jwicker@bidmc.harvard.edu

    Jeffrey Zwicker, MD

    Not yet recruiting

  • Dana Farber Cancer Institute

    Boston, Massachusetts, 02215

    Marlise Luskin, MD, (617) 632-1906, Marlise_Luskin@DFCI.HARVARD.EDU

    Marlise Luskin, MD

    Recruiting

  • Massachusetts General North Shore Cancer Center

    Danvers, Massachusetts, 01923

    Michelle H. Lee, MD, mlee37@mgb.org

    Recruiting

Other trials in United States

About this study

Official title: A Phase 2 Study Of Ruxolitinib In Low-Risk Essential Thrombocythemia And Polycythemia Vera With Significant Symptom Burden

This research is being done to see if the drug ruxolitinib is effective in reducing the symptoms caused by low-risk essential thrombocythemia (ET) and polycythemia vera (PV).

  • This research study involves the study drug Ruxolitinib.
Read the detailed description

This is a multi-center, non-randomized, two-stage phase II clinical trial evaluating ruxolitinib in low-risk but symptomatic essential thrombocythemia (ET) and polycythemia vera (PV) patients. This research is being done to see if Ruxolitinib is effective in reducing the symptoms people with essential thrombocythemia (ET) and polycythemia vera (PV) are experiencing. Ruxolitinib is a type of drug that blocks the specific proteins that may be causing the symptoms people with essential thrombocythemia (ET) and polycythemia vera (PV are experiencing.

The research study procedures include screening for eligibility and study treatment, including evaluations and follow up visits.

  • Participants will receive Ruxolitinib for approximately 6 months and if benefitting from it may continue to receive Ruxolitinib for as long as there is no unacceptable side effects or disease progression.

It is expected that about 60 people will take part in this research study.

The U.S. Food and Drug Administration (FDA) has approved Ruxolitinib for polycythemia vera (PV) but not for people with essential thrombocythemia (ET) and polycythemia vera (PV).

Incyte, a biopharmaceutical company, is supporting this research study by providing funding for the study, including the study drug.

Being tested
Ruxolitinib
Keywords
Essential Thrombocythemia, Polycythemia Vera, Sympton Burden
Medical subject headings
Thrombocythemia, Essential, Polycythemia Vera

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Study groups in NCT04644211
Study groupRoleWhat they receive
Ruxolitinib Stage 1ExperimentalIn stage 1, participants will be divided into two cohorts: * Very low, Low, and Intermediate-risk ETpatients with significant symptom burden and Low-risk PV patients with significant symptom burden * Study cycles are 28 days long, participants in both cohorts will receive: * Ruxolitinib 2x daily for 6 study cycles.
Receives: Drug: Ruxolitinib
Ruxolitinib Stage 2ExperimentalStage 2 will commence based on 3 or more participants in Stage 1 showing a predetermined positive response to Ruxolitinib. In stage 2, participants will be divided into two cohorts: * Very low, Low, and Intermediate-risk ET patients with significant symptom burden and Low-risk PV patients with significant symptom burden * Study cycles are 28 days long, participants in both cohorts will receive: * Ruxolitinib 2x daily for 6 study cycles.
Receives: Drug: Ruxolitinib

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Percentage of patients who achieve >50% reduction from baseline to Myeloproliferative Neoplasm Symptom Assessment Total Symptom Score

    Percentage of patients with >50% change in Myeloproliferative Neoplasm Symptom Assessment Total Symptom Score (MPN-SAF TSS), which has use in major clinical trials and as symptom assessment in all patients in clinical practice. Modeling has established baseline MPN-SAF TSS cut-off scores (total MPN-SAF TSS >20 or an individual component score >5) at which symptomatic treatment would be significantly beneficial

    Measured: baseline to 12 weeks

Secondary outcomes (6)

  1. Proportion of patients achieving complete hematologic rate at week 12

    Evaluate the efficacy of ruxolitinib in improving hematologic remission rates, as measured by International Working Group (IWG) working criteria ET: Platelet count ≤ 400 x 109/L, WBC count <10 x 109/L, and absence of leukoerythroblastosis PV: hematocrit <45% and WBC count <10 x 109/L

    Measured: Week 12

  2. Proportion of patients achieving complete hematologic rate at 24 Weeks

    Evaluate the efficacy of ruxolitinib in improving hematologic remission rates, as measured by International Working Group (IWG) working criteria ET: Platelet count ≤ 400 x 109/L, WBC count <10 x 109/L, and absence of leukoerythroblastosis PV: hematocrit <45% and WBC count <10 x 109/L

    Measured: Week 24

  3. Best MPN-SAF TSS score

    Myeloproliferative Neoplasm Symptom Assessment Total Symptom Score (MPN-SAF TSS) for use in major clinical trials and as symptom assessment in all patients in clinical practice. Modeling has established baseline MPN-SAF TSS cut-off scores (total MPN-SAF TSS >20 or an individual component score >5) at which symptomatic treatment would be significantly beneficial. Scale title includes absence of symptom (0) and worst imaginable symptoms (10) per question, with minimum score 0 and maximum score 100. Higher scores indicate worse outcomes.

    Measured: 12 Weeks

  4. Best MPN-SAF TSS score

    Myeloproliferative Neoplasm Symptom Assessment Total Symptom Score (MPN-SAF TSS) for use in major clinical trials and as symptom assessment in all patients in clinical practice. Modeling has established baseline MPN-SAF TSS cut-off scores (total MPN-SAF TSS >20 or an individual component score >5) at which symptomatic treatment would be significantly beneficial. . Scale title includes absence of symptom (0) and worst imaginable symptoms (10) per question, with minimum score 0 and maximum score 100. Higher scores indicate worse outcomes.

    Measured: 24 Weeks

  5. Percentage of change in Spleen Volume

    Spleen volume reduction will be measured as the change in percentage spleen volume from baseline at 12 weeks as measured by CT, or MRI in patients with medical contraindication to CT. Summary statistics will be used to describe changes in spleen volume. Volume will be calculated by a computer-assisted perimeter method based on Sorenson et al. Spleen and liver volume will be obtained by outlining the circumference of the organ and determining the volume using the validated technique of least squares

    Measured: baseline to 12 weeks

  6. Number of Participants with Treatment Related Adverse Events as Assessed by CTCAE v 5.0

    descriptions and grading scales found in the revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 5.0 will be utilized for AE reporting

    Measured: All patients who initiate treatment with study drug up to 60 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted25 Nov 2020
  2. Study start21 Mar 2022
  3. Primary completionJul 2027 (estimated)
  4. Study completionJul 2034 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Sequential
Masking
None (open label)
Enrolment
60 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04644211
Sponsor's protocol ID
20-364
Status last verified
Jan 2026
First submitted
10 Nov 2020
Last updated
8 Jan 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: The Dana-Farber / Harvard Cancer Center encourages and supports the responsible and ethical sharing of data from clinical trials. De-identified participant data from the final research dataset used in the published manuscript may only be shared under the terms of a Data Use Agreement. Requests may be directed to Sponsor Investigator or designee. The protocol and statistical analysis plan will be made available on Clinicaltrials.gov only as required by federal regulation or as a condition of awards and agreements supporting the research.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Ruxolitinib in people with Essential Thrombocythemia and Polycythemia Vera, run by Massachusetts General Hospital at 4 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 19 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 8 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Gabriela Hobbs, MD ((617) 726-8748, ghobbs@partners.org), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Percentage of patients who achieve >50% reduction from baseline to Myeloproliferative Neoplasm Symptom Assessment Total Symptom Score (time frame: baseline to 12 weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04644211.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Jan 2026.

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