Accepts Healthy Volunteers
Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms
An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.
An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.
Searching Both is inclusive of interventional and observational studies.
|Eligible Ages||18 Years and Over|
- - PART 1: JAK INHIBITOR ADMINISTRATION 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.
- - PART 2: ALLOGENEIC STEM CELL TRANSPLANT INCLUSION CRITERIA.
- - 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.
- - Received JAK inhibitor for at least 8 weeks immediately prior to conditioning and be able to continue until day -4 pre-transplant.
- - 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.
- - 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).
- - 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.
- - PART 1: JAK INHIBITOR ADMINISTRATION EXCLUSION CRITERIA.
- - Contraindication to receiving a JAK inhibitor 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) - PART 2: ALLOGENEIC STEM CELL TRANSPLANT EXCLUSION CRITERIA.
- - 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.
This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.
Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.
Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.
Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.
Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.
The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.
|Fred Hutchinson Cancer Center|
The person who is responsible for the scientific and technical direction of the entire clinical study.
|Rachel B. Salit|
|Principal Investigator Affiliation||Fred Hutch/University of Washington Cancer Consortium|
Category of organization(s) involved as sponsor (and collaborator) supporting the trial.
The disease, disorder, syndrome, illness, or injury that is being studied.
|Primary Myelofibrosis, Secondary Myelofibrosis|
OUTLINE: 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. 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 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. After completion of study treatment, patients are followed up between day 80-100, at 1 year, and then up to 5 years.
Experimental: Treatment (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. 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 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.
Drug: - Cyclophosphamide
Drug: - JAK Inhibitor
Drug: - Fludarabine
Biological: - Recombinant Granulocyte Colony-Stimulating Factor
Drug: - Melphalan
Drug: - Mycophenolate Mofetil
Procedure: - Peripheral Blood Stem Cell Transplantation
Drug: - Tacrolimus
Given IV and PO
Radiation: - Total-Body Irradiation
Contact a Trial Team
If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.