A Combination Study of PF-04449913 (Glasdegib) and Azacitidine In Untreated MDS, AML and CMML Patients

Official Title

An Open-label Phase 1b Study Of Pf-04449913 (Glasdegib) In Combination With Azacitidine In Patients With Previously Untreated Higher-risk Myelodysplastic Syndrome, Acute Myeloid Leukemia, Or Chronic Myelomonocytic Leukemia


This multi centre open label Phase 1b study is designed to evaluate the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of glasdegib (PF-04449913) when combined with azacitidine in patients with previously untreated Higher Risk Myelodysplastic Syndrome (MDS), Acute Myeloid Leukemia (AML), or Chronic Myelomonocytic Leukemia (CMML). This clinical study includes two components: (a) a safety lead in cohort (LIC) and (b) an expansion phase with an AML cohort and an MDS cohort.

Trial Description

Primary Outcome:

  • Complete remission (CR) rate
  • Adverse events (Safety lead-in phase)
Secondary Outcome:
  • Response Rate
  • Hematologic Improvement
  • Marrow Complete Response (mCR)
  • Cytogenetic Response
  • Stable Disease
  • QTc interval
  • Duration of CR
  • Time to CR
  • Overall Survival (OS)
  • Complete remission with partial hematologic recovery (AML Expansion cohort)
  • Adverse events (Expansion cohorts)
  • AUC for PF-04449913
  • Cmax for PF-04449913
  • Tmax for PF-04449913
  • AUC for Azacitidine
  • Cmax for Azacitidine
  • Tmax for Azacitidine
  • Ctrough for PF-04449913
  • Complete remission with incomplete hematologic recovery (AML Expansion cohort)
  • Morphologic leukemia free state (AML Expansion cohort)
  • Partial remission (AML Expansion cohort)
  • Stable disease (AML Expansion cohort)
  • Marrow complete remission (MDS Expansion cohort)
  • Partial remission (MDS Expansion cohort)
  • Stable disease (MDS Expansion cohort)
  • Partial or complete cytogenetic response (MDS Expansion cohort)
  • Hematologic improvement (MDS Expansion cohort)

View this trial on ClinicalTrials.gov

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Canadian Cancer Society

These resources are provided in partnership with the Canadian Cancer Society