NCT01841333

PF-04449913 For Patients With Acute Myeloid Leukemia at High Risk of Relapse After Donor Stem Cell Transplant

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

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 31 (actual)
• Sponsor: University of Colorado, Denver · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This phase II trial will test whether the Hedgehog signaling pathway inhibitor PF-04449913 can decrease disease relapse in high-risk patients with acute myeloid leukemia after donor stem cell transplant.

Detailed description

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.

Conditions

• Adult Acute Myeloid Leukemia in Remission
• Recurrent Adult Acute Myeloid Leukemia

Interventions

Type	Name	Description
DRUG	PF-04449913	100mg given orally

Timeline

• Start date: 2013-04-29
• Primary completion: 2019-05-08
• Completion: 2020-02-04
• First posted: 2013-04-26
• Last updated: 2022-01-25
• Results posted: 2022-01-25

Locations

2 sites across 1 country: United States