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

Status: Recruiting
Phase: Phase 2
Diagnosis: Leukemia/MDS
NCT ID: NCT01546038 (View complete trial on ClinicalTrials.gov)
DFCI Protocol ID:


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.


Conducting Institutions:
Dana-Farber Cancer Institute, Brigham and Women's Hospital, Massachusetts General Hospital

Overall PI:
Daniel DeAngelo, MD, Dana-Farber Cancer Institute

Site-responsible Investigators:
Eyal Attar, MD, Massachusetts General Hospital

Massachusetts General Hospital: Cancer Trials Call Center, 877-789-6100
Dana-Farber Cancer Institute: Ilene Galinsky, 617-632-3902, igalinsky@partners.org

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.
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