Could a JAK inhibitor improve stem cell transplant success in myelofibrosis?

NCT ID NCT03427866

What the study statuses mean

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Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jul 09, 2026 · Last updated Jul 10, 2026 · Updated 1 time

Summary

This phase 2 trial tests whether giving ruxolitinib (Jakafi) before, during, and after a stem cell transplant can improve outcomes for people with myelofibrosis. The study includes patients with intermediate-2 or high-risk disease, or intermediate-1 with additional risk factors. The main goal is to see if this approach increases the number of patients who survive one year without graft-versus-host disease or relapse.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Ruxolitinib (Jakafi)
What this could lead to
If successful, this approach could reduce the risk of graft-versus-host disease and relapse after stem cell transplant for myelofibrosis.
What could go wrong
This is a phase 2 trial with a small number of participants, so results may not apply broadly. Ruxolitinib can cause side effects like low blood counts and infections.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

44 people

The number who actually took part.

Started

Aug 2018

Finished

May 2025

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 75 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Participants must have pathologically confirmed primary myelofibrosis according to WHO criteria or secondary myelofibrosis as defined by the IWG-MRT criteria. * Intermediate-2/ high-risk disease as per Dynamic IPSS (DIPSS) criteria (Appendix G) OR * Intermediate-1 risk disease with one of the following additional unfavorable features known to impact the survival adversely * Red cell transfusion dependency * Unfavorable Karyotype * Platelet count ≤100 x 10\^9/L * Presence of a high risk molecular marker associated with worsened overall survival (ASXL1, EZH2, IDH1/2, SRSF2, U2AF1, p53) * Age 18-75 * Participants must be designated to undergo reduced intensity allogeneic peripheral blood (PB) or bone marrow (BM) hematopoietic stem cell transplantation. Consent will be obtained prior to admission for HCT. * Participants who will undergo HCT from the following donor types are eligible: * 6/6 (HLA-A, B, DR) fully matched related donor * 8/8 (HLA-A, B, DR, C) fully matched unrelated donor. Matching in the unrelated setting must be at the allele level * ECOG performance status ≤2 (Karnofsky ≥60%, see Appendix A) * Life expectancy of greater than 3 months * Able to give informed consent * Off all MF-directed therapy at the time of enrollment, with the exception of ruxolitinib, one week or 4 half-lives (effective), whichever is longer, prior to the first dose of study treatment * No allergy to ruxolitinib in the past * For patients already receiving ruxolitinib at the time of enrollment, patients should be treated with ruxolitinib for a sufficient time to optimize spleen response or symptoms, at the discretion of the treating provider, prior to enrollment. Patients who have had prior splenectomy are eligible. Exclusion Criteria: * Prior history of progressive multifocal leukoencephalopathy (PML) * Concomitant receipt of St. John's Wort * Hypersensitivity to any JAK inhibitor, including ruxolitinib, fedratinib, or any other JAK inhibitor * Prior allogeneic transplant for any hematopoietic disorder * Had accelerated phase or leukemic transformation (≥10% blasts in PB or BM any time prior to HCT) * Patients with uncontrolled infection (patients with stable controlled infections such as hepatitis B or HIV patients with undetectable viral load on antiviral treatment would be eligible). Patients who are actively ill and require hospitalization to treat an infection will be excluded. * History of another malignancy within 5-years of date of enrollment except those who have received definitive treatment. Definitive treatment will be defined as the use of surgery, chemotherapy or radiation for the treatment of a malignancy, which susbsquently has no evidence of disease after 2 years or \<10% probably of recurrence after 1 year. In addition, patients with history of the following are eligible: * basal cell or squamous cell carcinoma of skin * Polycythemia Vera or Essential Thrombocythemia * ductal carcinoma in situ (DCIS) * superficial bladder cancer * prostatic intraepithelial neoplasia (PIN) * Patients without normal organ function defined as follows: * AST (SGOT), ALT (SGPT) and Alkaline Phosphatase ≥ 3 × institutional Upper Limit of Normal (ULN) * Direct bilirubin \>2.0 mg/dL * Calculated creatinine clearance ≤60 mL/min (Cockcroft-Gault formula) * Note: patients with CrCl ≤60 mL/min but with normal creatinine (within institutional normal ranges) and no other evidence of inadequate renal function are eligible. * Have current or a history of congestive heart failure New York Heart Association (NYHA) class 3 or 4, or any history of documented diastolic or systolic dysfunction (LVEF \< 40%, as measured by MUGA scan or echocardiogram) * Pregnancy at the time of enrollment * Unable to give informed consent * Have an uncontrolled intercurrent illness including, but not limited to, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements. * Not able to take oral medication

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Massachusetts General Hospital

    Boston, Massachusetts, 02214, United States

  • Memorial Sloan Kettering Cancer Center

    New York, New York, 10065, United States

  • The Ohio State University Wexner Medical Center

    Columbus, Ohio, 43210, United States

  • Vanderbilt University

    Nashville, Tennessee, 37235, United States

  • Washington University

    St Louis, Missouri, 63130, United States

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