EVALUATION OF RUXOLITINIB AND AZACYTIDINE COMBINATION AS A THERAPY FOR PATIENTS WITH MYELOFIBROSIS AND MYELODYSPLASTIC SYNDROME/ MYELOPROLIFERATIVE NEOPLASM
The goal of this clinical research study is to learn if the combination of ruxolitinib and azacytidine can help to control myelofibrosis and myeloproliferative neoplasm.
Disease Group: Leukemia
Treatment Agent: Ruxolitinib
Treatment Location: Only at MDACC
Primary Objectives: To determine the efficacy of the combination of RUX with AZA in patients with MF (primary myelofibrosis, post polycythemia vera myelofibrosis, or post essential thrombocythemia myelofibrosis (PMF, post- PV MF, or post - ET MF) in achieving objective improvements in disease status To determine the efficacy of the combination of RUX + AZA in patients with MDS/MPN including chronic myelomonocytic leukemia (CMML), atypical chronic myeloid leukemia (BCR-ABL1 negative:aCML),and myelodysplastic syndromes/myeloproliferative neoplasms, unclassifiable (MDS/MPN-U), in achieving objective improvements in disease status Secondary Objectives: To determine the safety of the RUX + AZA combination in patients with MF and MDS/MPN Exploratory Objectives: To explore time to response (TTR) and duration of response (DOR) To explore the effect of the combination on anemia and transfusion dependence in patients with MF and MDS/MPN To explore the impact of baseline mutational profile on IWG-MRT response and overall survival in patients with MF and MDS/MPN To explore the impact of baseline anemia on overall survival in patients with MF and MDS/MPN
IRB Review and Approval Date: 03/13/2013
Recruitment Status: Open
Projected Accrual: 01
1) Patients with a diagnosis of primary myelofibrosis (PM), post
polycythemia vera myelofibrosis (PPV MF), or post essential
thrombocythemia myelofibrosis (PET MF) requiring therapy, including
those previously treated and relapsed or refractory, or if newly
diagnosed, with intermediate or high risk according to International
Working Group (IWG-MRT) criteria (appendix G).
2) Patients with a diagnosis of Myelodysplastic syndrome/myeloproliferative neoplasm, unclassifiable (MDS/MPN-U)} that requires therapy.
3) Understanding and voluntarily signing an IRB-approved informed consent form.
4) Age>/=18 years at the time of signing the informed consent.
5) ECOG performance status 0 to 2.
6) Adequate liver function (direct bilirubin of </= 2mg/dL, SGPT </= 2.5 x ULN or 5 x ULN if related to MF or MDS/MPN associated liver infiltration): if total bilirubin is </=2 , fractionation is not required for eligibility determination.
7) Creatinine </= 2.5 mg/dL
8) Platelets >/= 50 x 10^9/L
9) Absolute neutrophil count (ANC) >/= 1.0 x 10^9/L
1) For the MF and MDS/MPN-U arms (arms 1 & 2), use of any other
standard drug except hydroxyurea (see section 4.5 for guidelines for
allowed hydroxyurea use) anagrelide, growth factors, revlimid,
clofarabine, etc) or experimental drug or therapy within 14 days of
starting study therapy.
2) Patients previously treated with RUX or AZA (Only applicable for the MF and MDS/MPN arms).
3) Any serious psychological condition or psychiatric illness that would prevent the subject from signing the informed consent document, in the investigator opinion.
4) Pregnant or lactating females
5) Subjects of childbearing potential who are unwilling to take appropriate precautions (from screening through follow-up) to avoid becoming pregnant or fathering a child. Females of non-childbearing potential are defined as women who (a) are 55 years of age with history of amenorrhea for 1 year, OR (b) are surgically sterile for at least 3 months. For females of childbearing potential, or for males, pregnancy must be avoided by taking appropriate precautions. These precautions and the methods of contraception should be communicated to the subjects and their understanding confirmed.
6) Any condition, which significantly places the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study.
7) Known positive for HIV or with known active infectious hepatitis, type A, B or C.
8) Patients with active malignancy of other type than required for this study are not eligible with the exception of currently treated basal cell, squamous cell carcinoma of the skin, or carcinoma "in situ" of the cervix or breast. Patients with malignancies with indolent behavior such as prostate cancer treated with radiation or surgery can be enrolled in the study as long as they have a reasonable expectation to have been cured with the treatment modality received.
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