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Clinical Trials Search at Vanderbilt-Ingram Cancer Center

Ruxolitinib Phosphate before and after Stem Cell Transplant in Treating Patients with Primary or Secondary Myelofibrosis

This phase II trial studies how well ruxolitinib phosphate before and after stem cell transplant works in treating patients with primary or secondary myelofibrosis. Ruxolitinib phosphate may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as fludarabine and melphalan, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving chemotherapy before a donor stem cell transplant helps stop the growth of cells in the bone marrow, including normal blood-forming cells (stem cells) and cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. The donated stem cells may also replace the patient’s immune cells and help destroy any remaining cancer cells.
Not Available
Phase II
Adults
Not Available
Not Available
Byrne, Michael
National
Vanderbilt University
12-06-2018
Treatment
VICCBMT1863
NCT03427866

Eligibility

18 Years
BOTH
NO
Inclusion Criteria:

Participants must have pathologically confirmed primary myelofibrosis according to World Health Organization (WHO) criteria or secondary myelofibrosis as defined by the International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) criteria * Intermediate-2/ high-risk disease as per Dynamic International Prognostic Scoring System (IPSS) (DIPSS) Plus criteria OR * Intermediate-1 risk disease defined by one of the following unfavorable features known to impact the survival adversely ** Red cell transfusion dependency ** Unfavorable Karyotype ** Platelet count =
Participants must be designated to undergo reduced intensity allogeneic peripheral blood (PB) or bone marrow (BM) hematopoietic stem cell transplantation. Consent will be obtained prior to admission for HCT

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. Matching in the unrelated setting must be at the allele level * At the time of enrollment, donor identification may be ongoing for cohort 1 participants

Eastern Cooperative Oncology Group (ECOG) performance status == 60%)

Life expectancy of greater than 3 months

Able to give informed consent

Off all mycosis fungoides (MF)-directed therapy one week or 4 half-lives, whichever is longer, prior to enrollment, with the exception of ruxolitinib

COHORT I 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

COHORT I ONLY: 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 and/or loss of clinical benefit from ruxolitinib defined as continuing to derive symptom improvement (either from systemic symptoms or spleen discomfort). There is no minimum or maximum time requirement for time on ruxolitinib

COHORT I ONLY: Participants must have splenomegaly (defined by ultrasound or computed tomography [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 and platelets > 25/uL and hemoglobin > 7/dL

COHORT II ONLY: Participants are ineligible for ruxolitinib – do not have splenomegaly or symptoms of myelofibrosis as defined by the MPN-SAF. OR participants failed ruxolitinib as defined by loss of response to therapy and no allergy to ruxolitinib in the past



Exclusion Criteria:

Hypersensitivity to any JAK inhibitor

Prior allogeneic transplant for any hematopoietic disorder

Had accelerated phase or leukemic transformation (>= 10% blasts in peripheral blood [PB] or bone marrow [BM] any time prior to HCT)

Active uncontrolled infection

History of another malignancy within 5-years of date of except history of (h/o) basal cell or squamous cell carcinoma of skin or polycythemia vera or essential thrombocythemia

Aspartate aminotransferase (AST) (serum glutamic-oxaloacetic transaminase [SGOT]), alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase [SGPT]) >= 3 x institutional upper limit of normal (ULN)

Alkaline phosphatase >= 3 x institutional upper limit of normal (ULN)

Direct bilirubin > 2.0 mg/dL

Adequate renal function as defined by calculated creatinine clearance =
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 (left ventricular ejection fraction [LVEF]
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

Not able to take oral medication

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