PF-04449913 For Patients With Acute Myeloid Leukemia at High Risk of Relapse After Donor Stem Cell Transplant
|Adult Acute Lymphoblastic Leukemia in Remission Adult Acute Myeloid Leukemia in Remission Recurrent Adult Acute Lymphoblastic Leukemia Recurrent Adult Acute Myeloid Leukemia||Drug: PF-04449913||Phase 2|
|Study Design:||Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
|Official Title:||A Phase 2 Study of PF-04449913 for the Treatment of Acute Myeloid Leukemia Patients With High Risk of Post-Allogeneic Stem Cell Transplantation Relapse|
- Relapse-free survival [ Time Frame: 1 year ]Kaplan-Meier estimates will be used to measure relapse-free survival
- Remission duration [ Time Frame: Up to 5 years ]
- Number of patients with adverse events (AE) [ Time Frame: 30 days ]Subjects will be evaluated for AEs at each visit with the NCI-CTCAE version 4.03 used as a guide for the grading of severity.
- Overall survival of all patients [ Time Frame: 1 year ]
|Study Start Date:||April 11, 2013|
|Estimated Study Completion Date:||April 2019|
|Estimated Primary Completion Date:||April 2018 (Final data collection date for primary outcome measure)|
Beginning 80 days after allogeneic stem cell transplant, patients receive PF-04449913 (100mg) orally once daily on days 1-28. Treatment repeats every 28 days for up to 1 year in the absence of disease progression or unacceptable toxicity.
100mg given orally
Other Name: Hedgehog inhibitor
Disease relapse is the most common cause of death after allogeneic stem cell transplantation for acute myeloid leukemia. Patients at high risk for relapse may benefit from a novel, biologically rational therapeutic intervention to prevent this outcome. PF-04449913 is a small molecule inhibitor of the hedgehog (Hh) pathway that inhibits the protein Smoothened (SMO). Aberrant Hh signaling may contribute to the survival and expansion of the leukemia stem cell, and inhibiting the Hh pathway can eliminate these cells. Therefore, targeting Hh may be a logical intervention in the post-transplantation setting for those with high risk of relapse. The investigators propose a phase 2 study of PF-04449913 in patients with acute myeloid leukemia who have received an allogeneic stem cell transplantation and are at high risk of relapse.
This is an open label, phase 2 study employing PF-04449913 in acute myeloid leukemia patients who received an allogeneic stem cell transplantation and are at high risk of relapse. Patients will receive consecutive 28-day cycles of PF-04449913 at 100 mg/day, beginning on post-transplantation day 80 +/- 10 days, after their routine post-transplant bone marrow biopsy. Treatment will continue for up to one year or until they experience toxicity or disease relapse. 50 patients will be required for a 90% power to detect a 20% difference in one-year relapse-free survival.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01841333
|Contact: Emily A Denoncourt, RN, BSN, OCNfirstname.lastname@example.org|
|United States, Colorado|
|University of Colorado Cancer Center||Recruiting|
|Aurora, Colorado, United States, 80045|
|Contact: Emily Denoncourt, RN, BSN, OCN 720-848-0566 email@example.com|
|Principal Investigator: Dan A Pollyea, MD, MS|
|United States, Ohio|
|Ohio State University||Not yet recruiting|
|Columbus, Ohio, United States, 43210|
|Contact: Steve Devine, MD 614-293-5655|
|Principal Investigator: Steve Devine, MD|
|Principal Investigator:||Daniel A Pollyea, MD, MS||University of Colorado, Denver|