B1371012 Phase 1B/2 study of patients with MDS,AML,MLD OR CMML

• Research type

  Research Study

• Full title

  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

• IRAS ID

  163350

• Contact name

  Paresh Vyas

• Contact email

  paresh.vyas@imm.ox.ac.uk

• Sponsor organisation

  Pfizer Inc, 235 East 42nd Street, New York, 10017

• Eudract number

  2014-001345-24

• Duration of Study in the UK

  3 years, 5 months, 18 days

• Research summary

  Summary of Research
  The study medication, PF-04449913, is in a class of medications known as hedgehog inhibitors. These medications slow down or stop a pathway in cells called the hedgehog pathway. The hedgehog pathway regulates the growth of stem cells that form blood cells. PF-04449913 is designed to act on the hedgehog pathway to kill cancerous cells in leukaemia, and to correct the problems with the maturation and growth of young blood cells in the bone marrow that cause myelodysplastic disorders. This multi-centre randomised (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 randomised Phase 2.

  Approximately 10 people will be enrolled into the Lead-in Phase of this study, and about 160 people will be enrolled into the Randomised Phase. The study is being done at about 92 different research sites in 13 countries.

  Summary of Results
  A Combination Study of PF-04449913 (Glasdegib) and Azacitidine In Untreated MDS, AML and CMML Patients (BRIGHT 1012) | Pfizer

• REC name

  North East - Newcastle & North Tyneside 1 Research Ethics Committee

• REC reference

  14/NE/1240

• Date of REC Opinion

  16 Jan 2015

• REC opinion

  Further Information Favourable Opinion