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

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: A Multicenter, Open-Label, Pilot Study of Alisertib (MLN8237), a Novel Inhibitor of Aurora Kinase A, in Adult Patients With Relapsed/Refractory Acute Megakaryoblastic Leukemia

Brief Summary: 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 Summary:

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.


Sponsor: Northwestern University

Current Primary Outcome: Incidence of adverse events [ Time Frame: Up to 6 months after the last dose of treatment ]

The number, frequency, and severity of adverse events will be recorded every cycle for each population. All patients who receive at least 1 dose of alisertib will be considered evaluable for this endpoint.


Original Primary Outcome: Same as current

Current Secondary Outcome: Response to treatment [ Time Frame: Baseline to up to 6 months after the last dose of treatment ]

Serial blood and/or bone marrow samples will be collected at specific timepoints for each disease to determine response to alisertib treatment.


Original Secondary Outcome: Same as current

Information By: Northwestern University

Dates:
Date Received: August 18, 2015
Date Started: October 2015
Date Completion: March 2020
Last Updated: April 13, 2017
Last Verified: April 2017