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

Official Title

A Phase 1b/2 Study To Evaluate The Safety And 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

Summary:

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.

Trial Description

Primary Outcome:

  • Number of participants with Dose-limiting toxicities (DLT)
  • Percentage of patients with Complete Response rate
  • Overall Survival (OS)
Secondary Outcome:
  • Measure of drug exposure over the dosing interval following repeated dosing
  • Overall Survival (OS)
  • Percentage of patients with disease-specific efficacy for Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS)
  • Percentage of patients with Complete Response rate / Complete Response rate with incomplete blood count recovery
  • QTc Interval
  • Disease-related gene mutation (biomarkers)
  • Changes in analyte levels from baseline to post-treatment (biomarkers)
  • Changes in gene expression levels from baseline to post-treatment (biomarkers)
  • Detectable tumour Gli1 expression (biomarkers)

View this trial on ClinicalTrials.gov

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Resources

Canadian Cancer Society

These resources are provided in partnership with the Canadian Cancer Society