Reduced Intensity Haploidentical Transplantation for the Treatment of Primary or Secondary Myelofibrosis
NCT ID: NCT04370301
Last Updated: 2026-01-12
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
RECRUITING
PHASE2
20 participants
INTERVENTIONAL
2021-02-09
2029-08-31
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
JAK Inhibitor Before Donor Stem Cell Transplant in Treating Patients With Primary or Secondary Myelofibrosis
NCT02251821
Haplo-HSCT for Myelofibrosis
NCT06674382
Reduced Intensity Chemotherapy and Radiation Therapy Before Donor Stem Cell Transplant in Treating Patients With Hematologic Malignancies
NCT02566304
Haplo-identical Transplantation in Patients With Myelofibrosis - A Phase 2 Prospective Multicentric Prospective Study
NCT04728490
Peritransplant Ruxolitinib for Patients With Primary and Secondary Myelofibrosis
NCT04384692
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
COHORT I:
JAK INHIBITOR THERAPY: Patients receive a JAK inhibitor at least 8 weeks prior to the start of hematopoietic cell transplantation (HCT) conditioning through day -4 before transplantation.
CONDITIONING: Patients receive melphalan intravenously (IV) over 1 hour on day -5, fludarabine IV over 30-60 minutes on days -5 to -2, and undergo total-body irradiation (TBI) on day -1 or day -1 and day 0.
TRANSPLANT: Patients receive peripheral blood stem cell infusion on day 0.
GVHD PROPHYLAXIS: Patients then receive cyclophosphamide IV over 3 hours on days 3-4, tacrolimus IV beginning day 5 then orally (PO) for about 6 months, mycophenolate mofetil PO twice daily (BID) or three times daily (TID) beginning day 5 for 6 weeks, and granulocyte colony-stimulating factor (G-CSF) subcutaneously (SC) beginning day 7 until neutrophil recovery is \> 1,500/mm\^3.
COHORT II:
JAK INHIBITOR THERAPY: Patients receive a JAK inhibitor at least 8 weeks prior to the start of HCT conditioning through day -4 before transplantation. Additionally, patients receive a JAK inhibitor following transplantation beginning day +5 through 9-12 months after transplant.
CONDITIONING: Patients receive melphalan IV over 1 hour on day -5, fludarabine IV over 30-60 minutes on days -5 to -2, and undergo TBI on day -1 or day -1 and day 0.
TRANSPLANT: Patients receive peripheral blood stem cell infusion on day 0.
GVHD PROPHYLAXIS: Patients then receive cyclophosphamide IV over 3 hours on days 3-4, tacrolimus IV beginning day 5 then PO for about 6 months, mycophenolate mofetil PO BID or TID beginning day 5 for 6 weeks, and G-CSF SC beginning day 7 until neutrophil recovery is \> 1,500/mm\^3.
All patients undergo magnetic resonance imaging (MRI), computed tomography (CT), bone marrow biopsy and aspiration and blood sample collection throughout the trial. Patients also undergo echocardiography (ECHO) or multigated acquisition scan (MUGA) on the trial.
After completion of study treatment, patients are followed up between day 80-100, at 1 year, and then up to 5 years.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
NON_RANDOMIZED
SINGLE_GROUP
TREATMENT
NONE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Cohort I (JAK inhibitor, conditioning, GVHD prophylaxis)
JAK INHIBITOR THERAPY: Patients receive a JAK inhibitor at least 8 weeks prior to the start of HCT conditioning through day -4 before transplantation.
CONDITIONING: Patients receive melphalan IV over 1 hour on day -5, fludarabine IV over 30-60 minutes on days -5 to -2, and undergo TBI on day -1 or day -1 and day 0.
TRANSPLANT: Patients receive peripheral blood stem cell infusion on day 0.
GVHD PROPHYLAXIS: Patients then receive cyclophosphamide IV over 3 hours on days 3-4, tacrolimus IV beginning day 5 then PO for about 6 months, mycophenolate mofetil PO BID or TID beginning day 5 for 6 weeks, and G-CSF SC beginning day 7 until neutrophil recovery is \> 1,500/mm\^3.
All patients undergo MRI, CT, bone marrow biopsy and aspiration and blood sample collection throughout the trial. Patients also undergo ECHO or MUGA on the trial.
Cyclophosphamide
Given IV
JAK Inhibitor
Given PO
Fludarabine
Given IV
Recombinant Granulocyte Colony-Stimulating Factor
Given SC
Melphalan
Given IV
Mycophenolate Mofetil
Given PO
Peripheral Blood Stem Cell Transplantation
Given IV
Tacrolimus
Given IV and PO
Total-Body Irradiation
Undergo TBI
Computed Tomography
Undergo CT
Magnetic Resonance Imaging
Undergo MRI
Bone Marrow Biopsy
Undergo bone marrow biopsy and aspiration
Bone Marrow Aspiration
Undergo bone marrow biopsy and aspiration
Biospecimen Collection
Undergo blood sample collection
Echocardiography Test
Undergo ECHO
Multigated Acquisition Scan
Undergo MUGA
Cohort II (JAK inhibitor, conditioning, GVHD prophylaxis)
JAK INHIBITOR THERAPY: Patients receive a JAK inhibitor at least 8 weeks prior to the start of HCT conditioning through day -4 before transplantation. Additionally, patients receive a JAK inhibitor following transplantation beginning day 5 through 9-12 months after transplant.
CONDITIONING: Patients receive melphalan IV over 1 hour on day -5, fludarabine IV over 30-60 minutes on days -5 to -2, and undergo TBI on day -1 or day -1 and day 0.
TRANSPLANT: Patients receive peripheral blood stem cell infusion on day 0.
GVHD PROPHYLAXIS: Patients then receive cyclophosphamide IV over 3 hours on days 3-4, tacrolimus IV beginning day 5 then PO for about 6 months, mycophenolate mofetil PO BID or TID beginning day 5 for 6 weeks, and G-CSF SC beginning day 7 until neutrophil recovery is \> 1,500/mm\^3.
All patients undergo MRI, CT, bone marrow biopsy and aspiration and blood sample collection throughout the trial. Patients also undergo ECHO or MUGA on the trial.
Cyclophosphamide
Given IV
JAK Inhibitor
Given PO
Fludarabine
Given IV
Recombinant Granulocyte Colony-Stimulating Factor
Given SC
Melphalan
Given IV
Mycophenolate Mofetil
Given PO
Peripheral Blood Stem Cell Transplantation
Given IV
Tacrolimus
Given IV and PO
Total-Body Irradiation
Undergo TBI
Computed Tomography
Undergo CT
Magnetic Resonance Imaging
Undergo MRI
Bone Marrow Biopsy
Undergo bone marrow biopsy and aspiration
Bone Marrow Aspiration
Undergo bone marrow biopsy and aspiration
Biospecimen Collection
Undergo blood sample collection
Echocardiography Test
Undergo ECHO
Multigated Acquisition Scan
Undergo MUGA
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
Cyclophosphamide
Given IV
JAK Inhibitor
Given PO
Fludarabine
Given IV
Recombinant Granulocyte Colony-Stimulating Factor
Given SC
Melphalan
Given IV
Mycophenolate Mofetil
Given PO
Peripheral Blood Stem Cell Transplantation
Given IV
Tacrolimus
Given IV and PO
Total-Body Irradiation
Undergo TBI
Computed Tomography
Undergo CT
Magnetic Resonance Imaging
Undergo MRI
Bone Marrow Biopsy
Undergo bone marrow biopsy and aspiration
Bone Marrow Aspiration
Undergo bone marrow biopsy and aspiration
Biospecimen Collection
Undergo blood sample collection
Echocardiography Test
Undergo ECHO
Multigated Acquisition Scan
Undergo MUGA
Other Intervention Names
Discover alternative or legacy names that may be used to describe the listed interventions across different sources.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* Diagnosis of primary myelofibrosis (PMF) as defined by the 2016 World Health Organization classification system or diagnosis of secondary 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 the Dynamic International Prognostic Scoring System (DIPSS)-plus scoring system (DIPSS may be used if all data from DIPSS are not available)
* Ability to understand and the willingness to sign a written informed consent document (or legally authorized representative)
* Patient must be a potential hematopoietic stem cell transplant candidate
* Meeting criteria for 1st phase as above, at time of initiation of JAK inhibitor, including ability to understand and willingness to sign a written informed consent (or legally authorized representative). Patients arriving to our institution for transplant and not enrolled in Part 1 may still be enrolled in Part 2 if Part 1 criteria met. These patients will have Part 1 endpoints transcribed from medical records
* Received JAK inhibitor for at least 8 weeks immediately prior to conditioning and be willing to continue until 9-12 months post-transplant as tolerated
* Karnofsky performance status score \>= 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 \> 3 mg/dL, and symptomatic biliary disease will be excluded
* Diffusion capacity of the lung for carbon monoxide (DLCO) corrected \> 60% normal; may not be on supplemental oxygen
* Left ventricular ejection fraction \> 40% OR shortening fraction \> 26%
* Comorbidity Index \< 5 at the time of pre-transplant evaluation
* DONOR: Patients must be screened prior to transplant for donor-specific anti-HLA antibodies (DSA). Patients with DSA will be reviewed by the principal investigator and considered for desensitization treatment
* DONOR: Children are preferred over siblings and parents
* DONOR: Younger donors are preferred over older donors
* DONOR: ABO matched donors are preferred over minor ABO mismatched and over major ABO mismatch donors
Exclusion Criteria
* 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)
* Uncontrolled viral or bacterial infection at the time of study enrollment
* Active or recent (prior 6 month) invasive fungal infection without infectious disease (ID) consult and approval
* Known HIV positivity
* Pregnant or breastfeeding
* Availability of an human leukocyte antigen (HLA)-identical or 1-allele-mismatched related donor or an HLA 10 of 10 matched unrelated donor
18 Years
70 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Fred Hutchinson Cancer Center
OTHER
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Rachel B. Salit
Role: PRINCIPAL_INVESTIGATOR
Fred Hutch/University of Washington Cancer Consortium
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
Fred Hutch/University of Washington Cancer Consortium
Seattle, Washington, United States
Countries
Review the countries where the study has at least one active or historical site.
Central Contacts
Reach out to these primary contacts for questions about participation or study logistics.
Facility Contacts
Find local site contact details for specific facilities participating in the trial.
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
NCI-2020-02422
Identifier Type: REGISTRY
Identifier Source: secondary_id
10441
Identifier Type: OTHER
Identifier Source: secondary_id
RG1006957
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.