Study on Ruxolitinib Phosphate and Azacytidine for Treating Myelofibrosis or Myelodysplastic Syndrome/Myeloproliferative Neoplasm

Sponsor: M.D. Anderson Cancer Center

Sponsor score: 0
Inactive

ClinicalTrials ID:NCT01787487

Provider View

This clinical trial is testing the effectiveness of combining two drugs, ruxolitinib phosphate and azacytidine, to treat patients with myelofibrosis or myelodysplastic syndrome/myeloproliferative neoplasm. These drugs may help stop cancer cell growth by blocking necessary enzymes and killing cancer cells.

Patient Parameters

Program Overview

This study is exploring how well the combination of ruxolitinib phosphate and azacytidine works in treating patients with myelofibrosis or myelodysplastic syndrome/myeloproliferative neoplasm. Ruxolitinib phosphate may help stop cancer cells from growing by blocking certain enzymes. Azacytidine, a chemotherapy drug, works by killing cancer cells or stopping them from dividing and spreading. The combination of these drugs may be an effective treatment for these conditions.

Description

The main goal of this study is to see how effective the combination of ruxolitinib phosphate (RUX) and azacytidine (AZA) is in improving the condition of patients with myelofibrosis (MF) and myelodysplastic syndromes/myeloproliferative neoplasms (MDS/MPN). This includes specific types like chronic myelomonocytic leukemia (CMML) and atypical chronic myeloid leukemia (aCML).

The study also aims to determine the safety of this drug combination and explore how quickly patients respond to the treatment and how long the response lasts. Researchers will also look at how the treatment affects anemia and the need for blood transfusions, as well as the impact of genetic mutations and anemia on patient survival.

Patients will be divided into two groups based on their condition. Those with myelofibrosis will take ruxolitinib phosphate orally twice a day and, starting from the fourth treatment cycle, will also receive azacytidine either under the skin or through an IV for five days. This treatment cycle repeats every 28 days for up to 15 cycles unless the disease worsens or side effects become unacceptable.

Patients with myelodysplastic syndromes/myeloproliferative neoplasms will receive the same treatment as those with myelofibrosis. After completing the study treatment, patients will be monitored for 30 days and up to five years.

Eligibility Criteria

Inclusion Criteria

  • Diagnosed with primary myelofibrosis, post polycythemia vera myelofibrosis, or post essential thrombocythemia myelofibrosis needing treatment, including those who have been treated before and relapsed, or newly diagnosed with intermediate or high risk.
  • Diagnosed with myelodysplastic syndrome/myeloproliferative neoplasm, unclassifiable, requiring treatment.
  • Able to understand and sign an informed consent form approved by an Institutional Review Board (IRB).
  • Have an ECOG performance status of 0 to 2, indicating full activity or some restriction in physically strenuous activity but ambulatory.
  • Direct bilirubin level of 2 mg/dL or less.
  • Liver enzyme levels (SGPT) of 2.5 times the upper limit of normal or less, or 5 times if related to liver infiltration by MF or MDS/MPN.
  • Creatinine level of 2.5 mg/dL or less.
  • Platelet count of at least 50 x 10^9/L.
  • Absolute neutrophil count (ANC) of at least 1.0 x 10^9/L.

Exclusion Criteria

  • Use of any other standard or experimental drug or therapy within 14 days of starting the study, except for certain allowed medications.
  • Previous treatment with ruxolitinib phosphate or azacytidine.
  • Serious psychological or psychiatric conditions preventing informed consent.
  • Pregnant or breastfeeding women.
  • Individuals of childbearing potential unwilling to use effective contraception during the study and follow-up.
  • Any condition posing unacceptable risk or affecting data interpretation.
  • Known HIV positive or active hepatitis A, B, or C infection.
  • Active cancer other than the study's focus, except for certain skin cancers or in situ cervical or breast cancer. Indolent cancers like prostate cancer treated with curative intent may be eligible.

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