Can a cancer drug make stem cell transplants safer for myelofibrosis patients?
NCT ID NCT04384692
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests whether giving the drug ruxolitinib before, during, and after a donor stem cell transplant can prevent graft-versus-host disease (GVHD) in people with myelofibrosis. GVHD is a serious complication where donor cells attack the patient's body. The trial enrolls 53 adults with intermediate- or high-risk myelofibrosis. Ruxolitinib is already used to reduce symptoms like enlarged spleen and night sweats, and researchers hope it will also lower GVHD risk and improve survival.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- ruxolitinib
- What this could lead to
- If successful, this approach could reduce severe graft-versus-host disease and improve survival and quality of life for myelofibrosis patients undergoing stem cell transplant.
- What could go wrong
- This is a small, early-phase (Phase 2) trial with only 53 participants, so results may not apply broadly. Ruxolitinib can cause side effects like infections or blood count changes, and the transplant itself carries serious risks.
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
-
53 people
The number who actually took part.
- Started
-
Dec 2020
- Expected to finish
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Apr 2030
An estimate. End dates often move.
- 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
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18 years and older
- 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 Hide 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: * Age \>= 18 years * JAK INHIBITOR ADMINISTRATION INCLUSION: (PART I) * Disease criteria: * Diagnosis of primary myelofibrosis (PMF) as defined by the 2016 World Health Organization classification system or diagnosis of secondary myelofibrosis (MF) as defined by the International Working Group (IWG) for Myeloproliferative Neoplasms Research and Treatment criteria * Patients meeting the criteria for intermediate-1, intermediate-2 or high-risk disease by Dynamic International Prognostic Scoring System (DIPSS) or DIPSS plus * Ability to understand and the willingness to sign a written informed consent document * Patient must be a potential hematopoietic stem cell transplant candidate as assessed by the consenting physician * Patient must be willing to start ruxolitinib within a 6-month time period * ALLOGENEIC STEM CELL TRANSPLANT INCLUSION: (PART II) * Meeting criteria for part 1, as above, at time of initiation of ruxolitinib, including the ability to understand and willingness to sign a written informed consent. Patients arriving to our institution for transplant and not enrolled in part 1 may still be enrolled in part 2 if part 1 criteria are met. These patients will have part 1 endpoints transcribed from medical records * Received ruxolitinib for at least 8 weeks immediately prior to conditioning and be willing to continue until 9-12 months post-transplant as tolerated * Performance status score: Karnofsky \>= 70 * Calculated creatinine clearance using the Cockcroft-Gault formula or 24 hour (hr) urine creatinine clearance must be \> 60 ml/min * Total serum bilirubin must be \< 3 mg/dL unless the elevation is thought to be due to Gilbert's disease or hemolysis * Transaminases must be \< 3 x the upper limit of normal * Patients with clinical or laboratory evidence of liver disease will be evaluated for the cause of liver disease, its clinical severity in terms of liver function, and the degree of portal hypertension. Patients with fulminant liver failure, cirrhosis with evidence of portal hypertension or bridging fibrosis, alcoholic hepatitis, hepatic encephalopathy, or correctable hepatic synthetic dysfunction evidenced by prolongation of the prothrombin time, ascites related to portal hypertension, bacterial or fungal abscess, biliary obstruction, chronic viral hepatitis with total serum bilirubin \> 3mg/dL, and symptomatic biliary disease will be excluded * Diffusion capacity of lung for carbon monoxide (DLCO) corrected \> 60% normal. May be not be on supplemental oxygen * Left ventricular ejection fraction \> 40% OR shortening fraction \> 26% * Comorbidity index \< 5 at the time of pre-transplant evaluation Exclusion Criteria: * JAK INHIBITOR ADMINISTRATION EXCLUSION: (PART I) * Contraindication to receiving ruxolitinib including: * Patients who have known hypersensitivity to JAK inhibitors * Clinical or laboratory evidence of significant renal or hepatic impairment including cirrhosis * Active uncontrolled infection * Known human immunodeficiency virus (HIV) positivity * Women who are pregnant or trying to conceive * Caution should be used in patients with platelets \< 100 though adjustments in dose can be made to accommodate anyone with platelets \> 50 * History of prior allogeneic transplant * Leukemic transformation (\> 20% blasts) * ALLOGENEIC STEM CELL TRANSPLANT EXCLUSION: (PART II) * Uncontrolled viral or bacterial infection at the time of transplant data review and consent conference * Active or recent (prior 6 month) invasive fungal infection without infectious disease (ID) consult and approval * History of HIV infection * Pregnant or breastfeeding * Patients without a human leukocyte antigen (HLA)-identical sibling donor, 10 of 10 HLA-matched or 9 of 10 allele mismatched unrelated donor, or umbilical cord blood units that meet transplant criteria
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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
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Fred Hutch/University of Washington Cancer Consortium
Seattle, Washington, 98109, United States
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