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Myelodysplastic/Myeloproliferative NeoplasmPhase 2NCT07228624

Ruxolitinib Before, During and After Hematopoietic Cell Transplant in Older Patients With Myelofibrosis and Myelodysplastic Syndrome/Myeloproliferative Neoplasm Overlap Syndromes

A Phase 2 study testing Ruxolitinib, Allogeneic Hematopoietic Stem Cell Transplantation and Busulfan in people with Myelodysplastic/Myeloproliferative Neoplasm and Primary Myelofibrosis, run by Fred Hutchinson Cancer Center at 1 site 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
Search all trials

Trial summary

This phase II trial tests the effect of adding ruxolitinib to standard graft versus host disease (GVHD) prevention in treating older patients with myelofibrosis (MF) or myelodysplastic syndrome/myeloproliferative neoplasm (MDS/MPN) overlap syndromes before, during, and after a donor (allogeneic) hematopoietic cell… Read more about this study

Who is it for?
Ages 18 to 75 years
What is being tested?
Ruxolitinib, Allogeneic Hematopoietic Stem Cell Transplantation and Busulfan
Could I receive a placebo?
No, everyone receives an active treatment
How many people
50 participants
Where
1 siteUnited States
Study dates
4 Feb 2026to 1 Sep 2030
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 to 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • PART 1 JAK INHIBITOR ADMINISTRATION: Age 18-75 years
  • Patients > 75 must be considered an HCT candidate, meet all protocol criteria and have comorbidity score =< 3 and Karnofsky performance status (KPS) > or = to 90. Patients. > 75 who do not meet these criteria may be presented at PCC for consensus exception
  • PART 1 JAK INHIBITOR ADMINISTRATION: Disease criteria
  • Diagnosis of primary or secondary MF as defined by the 2022 World Health Organization classification system or the International Consensus Classification for Myeloid and Acute Leukemias
  • Diagnosis of an MDS/MPN overlap syndrome as defined by the 2022 World Health Organization
  • PART 1 JAK INHIBITOR ADMINISTRATION: Ability to understand and the willingness to sign a written informed consent document
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  • PART 1 JAK INHIBITOR ADMINISTRATION: Patient must be a potential HCT candidate as assessed by the consenting physician
  • PART 1 JAK INHIBITOR ADMINISTRATION: Patient must be agreeable to taking a JAK-inhibitor (ruxolitinib preferred) for at least 8 consecutive weeks immediately prior to conditioning and be willing to take ruxolitinib 5mg BID starting from day -4 prior to and continuing until 12 months post-transplant as tolerated followed by a 6-month taper.
  • PART 2 ALLOGENEIC STEM CELL TRANSPLANT: Meeting criteria for Part 1 at time of initiation of JAK-inhibitor, including the ability to understand and willingness to sign a written informed consent. Patients arriving to our institution for HCT 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 their medical records
  • PART 2 ALLOGENEIC STEM CELL TRANSPLANT: Received a JAK-inhibitor for at least 8 weeks immediately prior to conditioning and be willing to take Rux from day -4 at the 5mg BID dose until 12 months post-transplant as tolerated followed by a 6 month taper
  • PART 2 ALLOGENEIC STEM CELL TRANSPLANT: Performance status score
  • Karnofsky ≥ 70 or > 90 for patients > 75 years old
  • PART 2 ALLOGENEIC STEM CELL TRANSPLANT: HCT-CI Score < 8; if patient is > 75 years old HCT-CI < 3
  • PART 2 ALLOGENEIC STEM CELL TRANSPLANT: Calculated creatinine clearance using the Cockcroft-Gault formula or 24-hour urine creatinine clearance must be > 60 ml/min
  • PART 2 ALLOGENEIC STEM CELL TRANSPLANT: Total serum bilirubin must be < 3mg/dL unless the elevation is thought to be due to Gilbert's disease or hemolysis
  • PART 2 ALLOGENEIC STEM CELL TRANSPLANT: Transaminases must be < 3 x the upper limit of normal
  • PART 2 ALLOGENEIC STEM CELL TRANSPLANT: 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
  • PART 2 ALLOGENEIC STEM CELL TRANSPLANT: Diffusion capacity of lung for carbon monoxide (DLCO) corrected > 60% normal. Patient may not be on oxygen
  • PART 2 ALLOGENEIC STEM CELL TRANSPLANT: Left ventricular ejection fraction > 40%
  • DONOR: Human leukocyte antigen (HLA)-matched sibling donor
  • DONOR: 10 of 10 HLA-matched unrelated donor
  • DONOR: 9 of 10 allele or antigen mismatched unrelated donor
  • DONOR: Peripheral blood is preferred over bone marrow
  • DONOR: ABO matched donors should be selected if possible
  • DONOR: Matched unrelated donors may be preferred over siblings if the unrelated donor is < 30 years and the sibling is > 60 years. However, sibling donors < 70 should be preferred over mismatched unrelated donors

Who cannot join

  • PART 1 JAK INHIBITOR ADMINISTRATION: Contraindication to receiving ruxolitinib including patients who have known hypersensitivity to JAK inhibitors and excipients
  • PART 1 JAK INHIBITOR ADMINISTRATION: History of prior allogeneic transplant
  • PART 1 JAK INHIBITOR ADMINISTRATION: Leukemic transformation (> 20% blasts)
  • PART 1 JAK INHIBITOR ADMINISTRATION: Uncontrolled viral, bacterial, or fungal infection despite being on therapy
  • PART 1 JAK INHIBITOR ADMINISTRATION: History of HIV infection
  • PART 1 JAK INHIBITOR ADMINISTRATION: History of untreated tuberculosis (TB)
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  • PART 1 JAK INHIBITOR ADMINISTRATION: Pregnant or breastfeeding
  • PART 1 JAK INHIBITOR ADMINISTRATION: Patients with history of myocardial infarction (MI), cerebrovascular accident (CVA) or unprovoked pulmonary embolism (PE)/deep vein thrombosis (DVT) in the past 6 months
  • PART 1 JAK INHIBITOR ADMINISTRATION: Secondary malignancy in last 5 years with > 20% risk of relapse
  • PART 2 ALLOGENEIC STEM CELL TRANSPLANT: Contraindication to receiving ruxolitinib including patients who have known hypersensitivity to JAK inhibitors and excipients
  • PART 2 ALLOGENEIC STEM CELL TRANSPLANT: History of prior allogeneic transplant
  • PART 2 ALLOGENEIC STEM CELL TRANSPLANT: Leukemic transformation (> 20% blasts)
  • PART 2 ALLOGENEIC STEM CELL TRANSPLANT: Uncontrolled viral or bacterial infection at the time of transplant data review and consent conference
  • PART 2 ALLOGENEIC STEM CELL TRANSPLANT: Active or recent (prior 6 month) invasive fungal infection without infectious disease (ID) consult and approval
  • PART 2 ALLOGENEIC STEM CELL TRANSPLANT: History of HIV infection
  • PART 2 ALLOGENEIC STEM CELL TRANSPLANT: History of untreated TB
  • PART 2 ALLOGENEIC STEM CELL TRANSPLANT: Requiring supplemental oxygen
  • PART 2 ALLOGENEIC STEM CELL TRANSPLANT: Pregnant or breastfeeding
  • PART 2 ALLOGENEIC STEM CELL TRANSPLANT: Secondary malignancy in last 5 years with > 20% risk of relapse
  • PART 2 ALLOGENEIC STEM CELL TRANSPLANT: Patients with a history of MI, CVA, or unprovoked PE/DVT in the past 6 months
  • PART 2 ALLOGENEIC STEM CELL TRANSPLANT: Patients without an HLA-identical sibling donor, 10 of 10 HLA-matched or 9 of 10 mismatched unrelated donor

Trial locations and contacts

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

Rachel Salit, MD contact

206-667-1317

rsalit@fredhutch.org

United States1 site, 1 recruiting

  • Fred Hutch/University of Washington Cancer Consortium

    Seattle, Washington, 98109

    Rachel Salit, MD, 206-667-1317, rsalit@fredhutch.org

    Rachel Salit, MD

    Recruiting

Other trials in United States

About this study

Official title: Peri-Hematopoietic Cell Transplantation Ruxolitinib in Patients With Myelofibrosis and Myelodysplastic Syndrome/Myeloproliferative Neoplasm Overlap Syndromes

This phase II trial tests the effect of adding ruxolitinib to standard graft versus host disease (GVHD) prevention in treating older patients with myelofibrosis (MF) or myelodysplastic syndrome/myeloproliferative neoplasm (MDS/MPN) overlap syndromes before, during, and after a donor (allogeneic) hematopoietic cell transplant (HCT). Allogeneic HCT is a procedure in which a person receives blood-forming stem cells (cells from which all blood cells develop) from a genetically similar, but not identical donor. Giving chemotherapy, such as cytoxan and busulfan or fludarabine and melphalan, before a donor transplant helps kill cancer cells in the body and helps make room in the patient's bone marrow for new blood-forming cells (stem cells) to grow. However, sometimes the transplanted cells from a donor can attack the body's normal cells (called GVHD). Giving standard prevention (prophylaxis) therapies, such as tacrolimus and methotrexate, after the transplant may stop this from happening. Methotrexate, a type of antifolate, is in a class of medications called antimetabolites. Methotrexate stops cells from using folic acid to make deoxyribonucleic acid and may kill cancer cells. Tacrolimus is used to help reduce the risk of rejection by the body of organ and bone marrow transplants. Ruxolitinib, a type of Janus-associated kinase (JAK) inhibitor, blocks a protein called JAK, which may help keep abnormal blood cells or cancer cells from growing. It may also lower the body's immune response and prevent the development of GVHD. Giving ruxolitinib before, during and after allogeneic HCT in addition to standard GVHD prophylaxis may be safe, tolerable and effective in preventing GVHD and improving outcomes in older patients with MF or MDS/MPN overlap syndrome.

Read the detailed description

OUTLINE:

PART 1: Patients receive ruxolitinib or an alternate JAK-inhibitor for at least 8 weeks prior to the start of HCT conditioning. Starting on day -4, patients receive 5 mg of ruxolitinib orally (PO) or via nasogastric tube twice daily (BID) for 12 months then PO via nasogastric tube once daily (QD) until 18 months. Patients receive high intensity conditioning with cyclophosphamide intravenously (IV) on days -7 and -6 and busulfan IV over 3 hours on days -5 to -2 or reduced intensity conditioning with fludarabine IV over 30 minutes on days -6 to -2 and melphalan IV over 15-30 minutes on days -3 and -2.

PART 2: Patients receive stem cells infusion on day 0. Starting on day -3, patients receive tacrolimus IV over 1-2 hours or PO every 12 hours for at least 100 days. Patients also receive methotrexate IV on days 1, 3, 6, and 11.

Additionally, patients undergo urine sample collection, echocardiography (ECHO), chest computed tomography (CT) and pulmonary function testing prior to conditioning and blood sample collection throughout the study. Patients may also undergo bone marrow biopsy and aspiration, and/or spleen ultrasound or CT at multiple time points before and after transplant.

After completion of study treatment, patients are followed at days 28, 100, 180, and at 9 months, 1 year, 18 months and 2 years.

Medical subject headings
Myelodysplastic-Myeloproliferative Diseases, Primary Myelofibrosis

What participants receive

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

  • RuxolitinibDrug

    Also called INCB 018424, INCB-018424, INCB-18424, INCB18424

    Given PO or via nasogastric tube

  • Allogeneic Hematopoietic Stem Cell TransplantationProcedure or surgery

    Also called Allogeneic Hematopoietic Cell Transplantation, HSC, HSCT, Stem Cell Transplantation, Allogeneic

    Given infusion

  • BusulfanDrug

    Also called 1, 4-Bis[methanesulfonoxy]butane, BUS, Busilvex, Bussulfam

    Given IV

  • Computed TomographyProcedure or surgery

    Also called CAT, CAT Scan, Computed Axial Tomography, Computerized Axial Tomography

    Undergo CT

  • CyclophosphamideDrug

    Also called (-)-Cyclophosphamide, 2H-1,3,2-Oxazaphosphorine, 2-[bis(2-chloroethyl)amino]tetrahydro-, 2-oxide, monohydrate, Asta B 518, B 518

    Given IV

  • Echocardiography TestProcedure or surgery

    Also called EC, Echocardiography

    Undergo echocardiography

  • FludarabineDrug

    Also called Fluradosa

    Given IV

  • JAK InhibitorDrug

    Also called JAK inhibitors, Janus Kinase Inhibitor

    Given JAK inhibitor

  • MelphalanDrug

    Also called Alanine Nitrogen Mustard, CB-3025, L-PAM, L-Phenylalanine Mustard

    Given IV

  • MethotrexateDrug

    Also called Abitrexate, Alpha-Methopterin, Amethopterin, Brimexate

    Given IV

  • TacrolimusDrug

    Also called FK 506, FK-506, FK506, Fujimycin

    Given IV or PO

  • Biospecimen CollectionProcedure or surgery

    Also called Biological Sample Collection, Biospecimen Collected, Specimen Collection

    Undergo urine and blood sample collection

  • Bone Marrow AspirationProcedure or surgery

    Undergo bone marrow biopsy and aspiration

  • Bone Marrow BiopsyProcedure or surgery

    Also called Biopsy of Bone Marrow

    Undergo bone marrow biopsy and aspiration

Study groups in NCT07228624
Study groupRoleWhat they receive
Treatment (ruxolitinib)ExperimentalPART 1: Patients receive ruxolitinib or alternate JAK-inhibitor for at least 8 weeks prior to the start of HCT conditioning. Starting on day -4, patients receive ruxolitinib 5mg PO or via nasogastric tube BID for 12 months then PO via nasogastric tube QD until 18 months. Patients receive high intensity conditioning with cyclophosphamide IV on days -7 and -6 and busulfan IV over 3 hours on days -5 to -2 or reduced intensity conditioning with fludarabine IV over 30 minutes on days -6 to -2 and melphalan IV over 15-30 minutes on days -3 and -2. PART 2: Patients receive stem cells infusion on day 0. Starting on day -3, patients receive tacrolimus IV over 1-2 hours or PO every 12 hours for at least 100 days. Patients also receive methotrexate IV on days 1, 3, 6, and 11.
Receives: Drug: Ruxolitinib; Procedure: Allogeneic Hematopoietic Stem Cell Transplantation; Drug: Busulfan; Procedure: Computed Tomography; Drug: Cyclophosphamide; Procedure: Echocardiography Test; Drug: Fludarabine; Drug: JAK Inhibitor; Drug: Melphalan; Drug: Methotrexate; Drug: Tacrolimus; Procedure: Biospecimen Collection; Procedure: Bone Marrow Aspiration; Procedure: Bone Marrow Biopsy

What is being measured

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

  1. Incidence of grade II-IV graft versus host disease (GVHD) requiring systemic immune suppression

    Will be estimated as a simple proportion, and the upper bound of the one-sided 95% confidence interval for the estimated proportion will be estimated using the Clopper-Pearson method. The exact binomial test will be used to compare the observed probability to the benchmark of 65%.

    Measured: Up to day-100

Secondary outcomes (9)

  1. Incidence of grade III-IV acute GVHD

    Will be estimated as simple proportions and informally compared to the results from the historical cohort.

    Measured: Up to day-100

  2. Incidence of any-grade chronic GVHD

    Will be treated as time-to-event endpoints, with specified point estimates.

    Measured: Up to 1 and 2 years

  3. Incidence of moderate-to-severe chronic GVHD

    Will be treated as time-to-event endpoints, with specified point estimates.

    Measured: Up to 1 and 2 years

  4. Non-relapse mortality (NRM)

    Will be estimated as simple proportions and informally compared to the results from the historical cohort.

    Measured: At day-100

  5. NRM

    Will be treated as time-to-event endpoints, with specified point estimates.

    Measured: At 1 year

  6. Remission status

    Will be estimated as simple proportions and informally compared to the results from the historical cohort.

    Measured: At day-100

  7. Relapse rate

    Will be treated as time-to-event endpoints, with specified point estimates.

    Measured: At 1 and 2 years

  8. Overall survival

    Will be treated as time-to-event endpoints, with specified point estimates.

    Measured: Up to 1 year and by end of study, assessed up to 2 years

  9. Incidence of graft failure

    Will be estimated as simple proportions and informally compared to the results from the historical cohort.

    Measured: Up to day-100

Timeline

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

  1. First posted14 Nov 2025
  2. Study start4 Feb 2026
  3. Primary completion1 Sep 2030 (estimated)
  4. Study completion1 Sep 2030 (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
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
50 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07228624
Sponsor's protocol ID
RG1125655
Other IDs
NCI-2025-07728, FHIRB0020989
Status last verified
Oct 2026
First submitted
13 Nov 2025
Last updated
19 Oct 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Ruxolitinib, Allogeneic Hematopoietic Stem Cell Transplantation and Busulfan in people with Myelodysplastic/Myeloproliferative Neoplasm and Primary Myelofibrosis, run by Fred Hutchinson Cancer Center at 1 site 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 25 inclusion and 21 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 19 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site 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 Rachel Salit, MD (206-667-1317, rsalit@fredhutch.org), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of grade II-IV graft versus host disease (GVHD) requiring systemic immune suppression (time frame: Up to day-100). 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 NCT07228624.

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 19 Oct 2026.

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