Ruxolitinib Phosphate and Azacytidine in Treating Patients With Myelofibrosis or Myelodysplastic Syndrome/Myeloproliferative Neoplasm

Launched by M.D. ANDERSON CANCER CENTER · Feb 6, 2013

Keywords

ClinConnect Summary

This clinical trial is looking into how well two medications, ruxolitinib phosphate and azacytidine, work together to treat patients with certain types of blood disorders, specifically myelofibrosis and myelodysplastic syndromes. Ruxolitinib phosphate may help stop cancer cells from growing, while azacytidine can kill these cells or prevent them from multiplying. The goal of this study is to see if combining these two treatments can provide better results for patients than using them separately.

To be eligible for this trial, participants need to have specific diagnoses related to myelofibrosis or myelodysplastic syndromes and must require treatment. This includes patients who have newly diagnosed conditions that are considered intermediate or high risk, as well as those who have had prior treatments that didn’t work. Participants will need to be between 65 and 74 years old, and they should be able to understand the study and provide consent to join. If you join the trial, you will receive regular check-ups and monitoring to see how the treatment affects your condition. It’s also important to note that certain health conditions or treatments within the last two weeks may exclude someone from participating.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 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
• • Patients with a diagnosis of myelodysplastic syndrome/myeloproliferative neoplasm, unclassifiable (MDS/MPN-U) that require therapy
• • Understanding and voluntarily signing an Institutional Review Board (IRB)-approved informed consent form
• • Eastern Cooperative Oncology Group (ECOG) performance status 0 to 2
• • Direct bilirubin of =\< 2 mg/dL
• • Serum glutamate pyruvate transaminase (SGPT) =\< 2.5 x upper limit of normal (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
• • Creatinine =\< 2.5 mg/dL
• • Platelets \>= 50 x 10\^9/L
• • Absolute neutrophil count (ANC) \>= 1.0 x 10\^9/L
• Exclusion Criteria:
• • For the MF and MDS/MPN-U arms (arms 1 \& 2), use of any other standard drug (except hydroxyurea, anagrelide, growth factors, Revlimid, clofarabine, etc) or experimental drug or therapy within 14 days of starting study therapy
• • Patients previously treated with RUX or AZA (only applicable for the MF and MDS/MPN arms)
• • Any serious psychological condition or psychiatric illness that would prevent the subject from signing the informed consent document, in the investigator opinion
• • Pregnant or lactating females
• • 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
• • Any condition which 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
• • Known positive for human immunodeficiency virus (HIV) or with known active infectious hepatitis, type A, B or C
• • 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