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Primary MyelofibrosisPhase 2NCT04384692

Peritransplant Ruxolitinib for Patients With Primary and Secondary Myelofibrosis

A Phase 2 study testing Allogeneic Hematopoietic Stem Cell Transplantation, Busulfan and Cyclophosphamide in people with Primary Myelofibrosis and Secondary Myelofibrosis, run by Fred Hutchinson Cancer Center at 1 site in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

This phase II trial studies how well administering ruxolitinib before, during, and after allogeneic hematopoietic stem cell transplantation works in preventing graft versus host disease and improving transplant outcomes in patients with primary and secondary myelofibrosis. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Allogeneic Hematopoietic Stem Cell Transplantation, Busulfan and Cyclophosphamide
Could I receive a placebo?
No, everyone receives an active treatment
How many people
53 participants
Where
1 siteUnited States
Study dates
18 Dec 2020to 23 Apr 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 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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
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  • 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

Who cannot join

  • 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
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  • 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

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Fred Hutch/University of Washington Cancer Consortium

    Seattle, Washington, 98109

Other trials in United States

About this study

This phase II trial studies how well administering ruxolitinib before, during, and after allogeneic hematopoietic stem cell transplantation works in preventing graft versus host disease and improving transplant outcomes in patients with primary and secondary myelofibrosis. Donor hematopoietic stem cell transplantation (HSCT) is currently the only treatment with proven curative potential for myelofibrosis, however, myelofibrosis patients have a high risk for developing graft versus host disease post-transplant. Graft versus host disease is a condition where the transplanted cells from a donor can attack the body's normal cells. Ruxolitinib, a janus-associated kinase (JAK) inhibitor, is known to decrease inflammatory signals, which may reduce spleen size and decrease symptoms such as night sweats and weight loss. Administering ruxolitinib before, during, and after transplant may decrease the incidence and severity of graft versus host disease, increase survival, and improve quality of life in patients with primary and secondary myelofibrosis.

Read the detailed description

OUTLINE:

PART 1: Patients receive ruxolitinib orally (PO) starting 8 weeks prior to hematopoietic stem cell transplantation (HSCT) and continuing until approximately 14 days prior to conditioning regimen, then tapered per the treating clinician until day -4 in the absence of disease progression or unacceptable toxicity. Patients who join a different research study for Part 2 have their collected data and samples from Part 1 carried over to the new protocol.

PART 2: Patients are assigned to either a high (myeloablative) or reduced intensity conditioning regimens per the clinical provider together with the Clinical Coordinators Office (CCO):

MYELOABLATIVE CONDITIONING: Patients receive cyclophosphamide intravenously (IV) on days -7 and -6 and busulfan IV over 3 hours on days -5 to -2. Patients with umbilical cord blood (UCB) as their transplant source also receive fludarabine IV over 30 minutes on days -8 to -6. Treatment continues in the absence of disease progression or unacceptable toxicity.

REDUCED INTENSITY CONDITIONING: Patients receive fludarabine IV over 30 minutes on days -6 to -2 and melphalan IV over 15-30 minutes on days -3 and -2. Patients with UCB as their transplant source also undergo total body irradiation (TBI) on day -1. Treatment continues in the absence of disease progression or unacceptable toxicity.

TRANSPLANT: After completion of conditioning regimen, patients undergo HSCT on day 0.

GVHD PROPHYLAXIS: Patients receive ruxolitinib until approximately 7 months post-transplant and then tapered over 2 months until 9-12 months post HSCT. Patients also receive tacrolimus IV continuously (inpatients) or every 12 hours (outpatients) beginning day -1 (day -3 for patients with UCB as their donor source), then PO twice daily (BID) once therapeutic levels are reached, with a taper beginning on day 56 for patients with related donors, and day 100 for patients with unrelated donors over 4 months in the absence of GVHD. The duration of tacrolimus for patients with GVHD is determined by the attending physician. Patients with related and unrelated donors also receive methotrexate IV on days 1, 3, 6, and 11. Patients with UCB as their transplant source also receive mycophenolate mofetil IV or PO every 8 hours beginning on days 0-30, then tapered until day 40 in the absence of GVHD. All treatment continues in the absence of disease progression or unacceptable toxicity.

Patients undergo computed tomography (CT) scan and may undergo echocardiography on study and bone marrow aspiration and biopsy and blood sample collection and may undergo magneti resonance imaging (MRI) and ultrasound throughout the study.

Patients are followed up at 6 months, 1 year, and 2-5 years after completion of HSCT.

Medical subject headings
Primary Myelofibrosis

What participants receive

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

  • Allogeneic Hematopoietic Stem Cell TransplantationProcedure or surgery

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

    Undergo HSCT

  • BusulfanDrug

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

    Given IV

  • CyclophosphamideDrug

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

    Given IV

  • FludarabineDrug

    Also called Fluradosa

    Given IV

  • 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

  • Mycophenolate MofetilDrug

    Also called CellCept, MMF

    Given IV or PO

  • RuxolitinibDrug

    Also called INCB-18424, INCB18424, Oral JAK Inhibitor INCB18424

    Given PO

  • TacrolimusDrug

    Also called FK 506, Fujimycin, Hecoria, Prograf

    Given IV and PO

  • Total-Body IrradiationRadiation

    Also called TBI, Total Body Irradiation, Whole Body Irradiation, Whole-Body Irradiation

    Undergo TBI

  • Computed TomographyProcedure or surgery

    Also called CAT Scan, Computed Axial Tomography

    Undergo CT scan

  • EchocardiographyProcedure or surgery

    Also called EC

    Undergo echocardiography

  • Bone Marrow Aspiration and BiopsyProcedure or surgery

    Undergo bone marrow aspiration and biopsy

  • Magnetic Resonance ImagingProcedure or surgery

    Also called MRI

    Undergo MRI

  • Ultrasound ImagingProcedure or surgery

    Also called 2-Dimensional Grayscale Ultrasound Imaging

    Undergo ultrasound imaging

Study groups in NCT04384692
Study groupRoleWhat they receive
Treatment (ruxolitinib, conditioning, HSCT, GVHD prophylaxis)ExperimentalSee detailed description.
Receives: Procedure: Allogeneic Hematopoietic Stem Cell Transplantation; Drug: Busulfan; Drug: Cyclophosphamide; Drug: Fludarabine; Drug: Melphalan; Drug: Methotrexate; Drug: Mycophenolate Mofetil; Drug: Ruxolitinib; Drug: Tacrolimus; Radiation: Total-Body Irradiation; Procedure: Computed Tomography; Procedure: Echocardiography; Procedure: Bone Marrow Aspiration and Biopsy; Procedure: Magnetic Resonance Imaging; Procedure: Ultrasound Imaging

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) in myelofibrosis patients

    The probability of grade II-IV acute GVHD observed in this study will be compared to a fixed benchmark, this fixed benchmark derived from the results of a previous study (FH Protocol 9033).

    Measured: Up to day 100

Secondary outcomes (8)

  1. Incidence of grade III-IV GVHD

    Estimated as simple proportions and informally compared to the results from FH Protocol 9033.

    Measured: Up to day 100

  2. Incidence of chronic GVHD

    Measured: At 1 and 2 years

  3. Overall survival rate (OS)

    Measured: At 1 and 2 years

  4. Incidence of primary and secondary graft failure

    Estimated as simple proportions and informally compared to the results from FH Protocol 9033.

    Measured: 6 months

  5. Time to neutrophil (ANC > 500) engraftment

    Measured: Day 100

  6. Time to platelet (> 20,000) engraftment

    Measured: Day 100

  7. Incidence of relapse

    Measured: At 1 year

  8. Non-relapse mortality (NRM)

    Estimated as simple proportions and informally compared to the results from FH Protocol 9033.

    Measured: Day 100 and 1 year

Timeline

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

  1. First posted12 May 2020
  2. Study start18 Dec 2020
  3. Primary completion3 Apr 2026
  4. Study completion23 Apr 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
53 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04384692
Sponsor's protocol ID
RG1006507
Other IDs
NCI-2020-01626, 10093
Status last verified
Apr 2026
First submitted
8 May 2020
Last updated
14 Apr 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 Allogeneic Hematopoietic Stem Cell Transplantation, Busulfan and Cyclophosphamide in people with Primary Myelofibrosis and Secondary 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 19 inclusion and 16 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: under way, not looking for participants, as of the sponsor's last update on 14 Apr 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?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of grade II-IV graft versus host disease (GVHD) in myelofibrosis patients (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 NCT04384692.

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 14 Apr 2026.

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