A Phase 2 Study of AZD6244 in Relapsed or Refractory AML
I. To determine the response rate (includes complete response-CR, complete response with
incomplete count recovery CRi, partial response-PR, and minor response-MR) to AZD6244
I. To determine the effects of AZD6244 in AML samples on p-ERK and evaluate the potential
utility of p-ERK inhibition as a surrogate marker of biologic activity.
II. To correlate the effects of AZD6244 with the presence (or absence) of mutated RAS or
FLT-3 at baseline.
III. To assess the safety profile of AZD6244 in patients with AML.
Patients receive selumetinib orally (PO) twice daily (BID) on days 1 -28. Treatment repeats
every 4 weeks in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed for 52 weeks.
Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Response rate (CR, CR with incomplete count recovery, partial response, and minor response) to AZD6244
A Simon, optimal two-stage design will be employed in cohort A. For Cohort B, we will determine the response rate and provide an exact 90% confidence interval using the binomial distribution. The response rate in these patients will be compared to that observed in the main cohort using Fisherâs exact test.
Up to 52 weeks
University of Chicago Comprehensive Cancer Center
United States: Food and Drug Administration
|University of Chicago Comprehensive Cancer Center||Chicago, Illinois 60637-1470|