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 - 75 Years|
- - Participants must have pathologically confirmed primary myelofibrosis according to WHO criteria1 or secondary myelofibrosis as defined by the IWG-MRT criteria.
- - Intermediate-2/ high-risk disease as per Dynamic IPSS (DIPSS) criteria2 (Appendix 1) OR.
- - Intermediate-1 risk disease with one of the following additional unfavorable features known to impact the survival adversely.
- - Red cell transfusion dependency2.
- - Unfavorable Karyotype2.
- - Platelet count ≤100 x 109/L.
- - Age 18-75.
- - Participants must be designated to undergo reduced intensity allogeneic peripheral blood (PB) or bone marrow (BM) hematopoietic stem cell transplantation.
- - Participants who will undergo HCT from the following donor types are eligible: - 5/6 or 6/6 (HLA-A, B, DR) matched related donor.
- - 7/8 or 8/8 (HLA-A, B, DR, C) matched unrelated donor.
- - ECOG performance status ≤2 (Karnofsky ≥60%, see Appendix A) - Life expectancy of greater than 3 months.
- - Able to give informed consent.
- - Off all MF-directed therapy at the time of enrollment, with the exception of ruxolitinib.
- - Additional Criteria for Cohort 1 Only: - Patients are candidates for enrollment in cohort 1 if they have an indication for ruxolitinib based on splenomegaly or symptoms and are either on ruxolitinib already or going to start therapy with ruxolitinib.
- - Patients that are on ruxolitinib may enroll in study as long as they are willing to remain on ruxolitinib during the study and have not lost response to ruxolitinib defined as an increase in >5 cm in spleen size from nadir.
- - Participants must have splenomegaly (defined by ultrasound or CT scan of the abdomen) or symptoms (demonstrated by the presence of 1 symptom score >5 or 2 symptom scores >3) related to myelofibrosis as measured by the myeloproliferative neoplasm symptom assessment form MPN-SAF (see Appendix F) and platelets >25/μL and hemoglobin >7/dL.
- - Additional Criteria Cohort 2 Only: - Participants are ineligible for ruxolitinib - do not have splenomegaly or symptoms of myelofibrosis as defined by the MPN-SAF.
- - Participants failed ruxolitinib as defined by loss of response to therapy and.
- - No allergy to ruxolitinib in the past.
- - Hypersensitivity to any JAK inhibitor.
- - Prior allogeneic transplant for any hematopoietic disorder.
- - Had accelerated phase or leukemic transformation (≥10% blasts in PB or BM any time prior to HCT) - Active uncontrolled infection.
- - History of another malignancy within 5-years of date of except h/o basal cell or squamous cell carcinoma of skin or Polycythemia Vera or Essential Thrombocythemia.
- - Patients without normal organ function defined as follows: - AST (SGOT), ALT (SGPT) and Alkaline Phosphatase ≥ 3 × institutional Upper Limit of Normal (ULN) - Direct bilirubin >2.0 mg/dL.
- - Adequate renal function as defined by calculated creatinine clearance≤60 mL/min (Cockcroft-Gault formula) - Have a chronic or active infection that requires systemic antibiotics, antifungal or antiviral treatment.
- - Have current or a history of congestive heart failure New York Heart Association (NYHA) class 3 or 4, or any history of documented diastolic or systolic dysfunction (LVEF < 40%, as measured by MUGA scan or echocardiogram) - Pregnancy at the time of enrollment.
- - Unable to give informed consent.
- - Have an uncontrolled intercurrent illness including, but not limited to, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
- - Subjects who require therapy with a strong CYP3A4 inhibitor prior to enrollment to this study.
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.
|Massachusetts General Hospital|
The person who is responsible for the scientific and technical direction of the entire clinical study.
|Gabriela Hobbs, MD|
|Principal Investigator Affiliation||Massachusetts General Hospital|
Category of organization(s) involved as sponsor (and collaborator) supporting the trial.
The disease, disorder, syndrome, illness, or injury that is being studied.
This research study is a Phase II clinical trial. Phase II clinical trials test the safety and effectiveness of an investigational drug to learn whether the drug works in treating a specific disease. "Investigational" means that the drug is being studied. The FDA (the U.S. Food and Drug Administration) has approved Ruxolitinib as a treatment option for this disease. This study examines two different cohorts of participants:
- - Cohort 1: Participants who are eligible for Ruxolitinib therapy before transplant, based on their platelet counts.
- - Cohort 2: Participants who are not eligible for Ruxolitinib therapy pre-treatment based on their platelet counts.
- - assessing the efficacy (how well the study drug works) and tolerability of Ruxolitinib before, during, and after HCT.
- - examining the rates of GVHD after HCT when ruxolitinib is administered.
Experimental: Ruxolitinib Eligible pre-HSCT
Ruxolitinib will be taken orally at a fixed dose twice every day Dosing will be continuous, with a new cycle scheduled to start every 28 days. There will be no break in dosing between cycles Ruxolitinib can be administered with or without food.
Experimental: Ruxolitinib Not Eligible pre-HSCT
Ruxolitinib will be taken orally at a fixed dose twice every day after transplant Dosing will be continuous, with a new cycle scheduled to start every 28 days. There will be no break in dosing between cycles Ruxolitinib can be administered with or without food.
Drug: - Ruxolitinib
Ruxolitinib is a medication that blocks certain proteins called tyrosine kinases. Specifically, it blocks tyrosine kinases called JAK2. The JAK2 pathway is over active in the disease, acute myeloid leukemia.
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.