Last updated on October 2016

Alisertib in Treating Patients With Myelofibrosis or Relapsed or Refractory Acute Megakaryoblastic Leukemia


Brief description of study

The purpose of this study is to evaluate the safety of alisertib and its effect, bad and/or good, on acute megakaryoblastic leukemia (AMKL) or myelofibrosis (MF). The study drug, alisertib, is an investigational drug. An investigational drug is one that has not been approved by the U.S. Food and Drug Administration (FDA). Alisertib has shown evidence in the lab that it may have an effect on a type of cell that produces platelets. This cell is called a megakaryocyte and it is known to be defective (doesn't work well) in both AMKL and MF.

Detailed Study Description

PRIMARY OBJECTIVES: I. Determine the safety profile of alisertib in patients with acute megakaryoblastic leukemia (AMKL) and in patients with myelofibrosis (MF). SECONDARY OBJECTIVES: I. Determine preliminary efficacy of alisertib in both populations. TERCIARY OBJECTIVES: I. Describe pharmacodynamics (PD) effects of alisertib in peripheral blood and/or bone marrow samples. II. Evaluate the relationship between biomarker expression levels and response to alisertib. III. Evaluate reduction in splenomegaly by palpation (MF arm only). IV. Evaluate improvement in MF symptoms (MF arm only), as assessed by the Myeloproliferative Neoplasm Symptom Assessment form (MPN-SAF). V. Assess change in bone marrow fibrosis in patients in the MF arm. OUTLINE: Patients receive alisertib orally (PO) twice daily (BID) on days 1-7. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity. After completion of study treatment, patients are followed up at approximately 30 days and 6 months.

Clinical Study Identifier: NCT02530619

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Ronan T. Swords, MD

University of Miami Miller School of Medicine-Sylvester Cancer Center
Miami, FL United States
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Brady L. Stein, MD

Northwestern University
Chicago, IL United States
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Naseema Gangat, MD

Mayo Clinic
Rochester, MN United States
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