A Study To Evaluate PF-04449913 With Chemotherapy In Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome

Status: Complete


This is a study to evaluate PF-04449913 (an inhibitor of the Hedgehog pathway) in Acute Myeloid Leukemia and high-risk Myelodysplastic Syndrome in combination with standard agents used to treat these diseases.

Eligibility Criteria

Inclusion Criteria

  • Patients with AML or RAEB 2 High Risk MDS who are newly diagnosed according to the WHO 2008 Classification and previously untreated.
  • Patients with AML (arising from an antecedent hematologic disease [AHD]) or MDS who may have had one prior regimen with commercially available agents for the treatment of their prior hematologic disease. The patients may not have had a prior therapy for their AML.
  • AML patients include de novo AML, AML evolving from MDS or other AHD and AML after previous cytotoxic therapy or radiation (secondary AML)
  • For a diagnosis of AML, a bone marrow blast count of 20% or more is required.
  • For a diagnosis of high-risk Myelodysplastic Syndrome RAEB 2 the patient must have 10-19% bone marrow blasts
  • Adequate Organ Function
  • ECOG Performance Status 0, 1, or 2

Exclusion Criteria

  • AML M3 Acute Promyelocytic Leukemia (APL) or patients with a t(9:22) cytogenetic translocation.
  • Patients with known active uncontrolled central nervous system (CNS) leukemia.

Locations & Contacts


University of Chicago Comprehensive Cancer Center
Status: Active
Name Not Available


Fred Hutch / University of Washington Cancer Consortium
Status: Active
Name Not Available

Trial Phase & Type

Trial Phase

Phase II

Trial Type


Lead Organization

Lead Organization

Trial IDs

Primary ID B1371003
Secondary IDs NCI-2012-01145, 2012-000684-24
Clinicaltrials.gov ID NCT01546038