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9L-14-6: A Randomized, Double-Blind Phase 1b/2 Study of PF-04449913 in Combination with Azacitidine in Patients with Previously Untreated Intermediate-2 or High-Risk Myelodysplastic Syndrome, Acute Myeloid Leukemia with 20-30% Blasts and Multi-Lineage Dysplasia, or Chronic Myelomonocytic Leukemia

Description

PF-04449913 is a novel small molecule inhibitor of the Sonic Hedgehog (Hh) Pathway which is currently under development for the treatment of hematologic malignancies and solid tumors.This multi-center randomized (1:1), double-blind, placebo-controlled Phase 1b/2 study is designed to compare the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of PF-04449913 or placebo when combined with azacitidine in patients with previously untreated Intermediate-2 or High-Risk Myelodysplastic Syndrome (MDS), Acute Myeloid Leukemia (AML) with 20-30% blasts and multi-lineage dysplasia, and Chronic Myelomonocytic Leukemia (CMML).This clinical study includes two components: (a) a Phase 1b safety lead-in and (b) a randomized Phase 2.Primary Objective is to assess the safety and tolerability of PF-04449913 when administered in combination with azacitidinePrimary endpoint is adverse events as characterized by type, frequency, severity, timing, seriousness and relationship to study therapy

Phase

N/A

Inclusion and Exclusion Criteria

  • Patient must meet all the following inclusion criteria to be eligible for enrollment into the study:
  • Morphologically confirmed diagnosis of one of the following:
  • MDS according to the WHO 2008 classification and bone marrow blasts > or = 5%
  • AML with 20-30 % BM blasts and multi lineage dysplasia, according to WHO 2008 classification and WBC < 20x109/L
  • CMML according to the WHO 2008 classification (Appendix 1) and BM blasts between 10% 19% and WBC < 13x109/L
  • MDS patients must have Intermediate 2 (1.5 to 2.0 points) or High Risk (≥2.5 points) disease according to the International Prognostic Scoring System 1997 (IPSS).
  • MDS patients must have normal levels of vitamin B12 within the institutional range of normal as determined within 28 days of study entry.
  • AML patients with 20- 30% BM blasts and multi lineage dysplasia, must have stable blast counts per Investigator's judgment.
  • Clinical indication for treatment with azacitidine for MDS, AML or CMML.

  • Patients with any of the following may not be included in the study:
  • Patients with AML who are candidates for standard induction chemotherapy as first line treatment.
  • Therapy related (secondary to radiation or chemotherapy) MDS or AML.
  • Prior hypomethylating agents or cytotoxic chemotherapy for MDS, AML or CMML (prior immunosuppressive therapy and hydroxyurea are permitted provided that treatment is stopped within 8 and 2 weeks from study entry, respectively).
  • Previous hematopoietic stem cell transplant.
  • Prior treatment with a licensed or experimental smoothened inhibitor (SMOi) and/or hypomethylating agent (HMA).

Sites

Please contact Gangothri Namasivayam to learn more about where you can participate in this trial. Please use the contact form on the right side.

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