Phase 1 Program

SCCA Clinical Studies: Phase 1 Program

PF-04449913 w/ Chemotherapy for AML or MDS
A Phase 1B Study To Evaluate The Safety And Preliminary Efficacy Of PF-04449913, An Oral Hedgehog Inhibitor, In Combination With Intensive Chemotherapy, Low Dose Ara-C Or Decitabine In Patients With Acute Myeloid Leukemia Or High Risk Myelodysplastic Syndrome
Status Conditions Phase Study ID
Closed Acute Myeloid Leukemia
High-risk Myelodysplastic Syndrome
Phase I NCT01546038

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.

Vivian G. Oehler, MD
Seattle Cancer Care Alliance 800-804-8824  
Eligibility Criteria (must meet the following to participate in this study)
  • 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 approved prior targeted therapy (eg, Azacytidine or decitabine) 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
Exclusions (conditions that would prevent participation in this study)
  • AML M3 Acute Promyelocytic Leukemia (APL) or patients with a t(9:22) cytogenetic translocation.
  • Patients in whom, at the time of study entry, a stem cell transplant is planned within the next 6 months.
  • Patients with known active uncontrolled central nervous system (CNS) leukemia.
Last Updated
July 23, 2014
See this trial at
Access protocol and consent forms at Fred Hutchinson Cancer Research Center
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