A Phase I, Dose-finding Study of BEZ235 in Adult Patients With Relapsed or Refractory Acute Leukemia
Status:
Unknown status
Trial end date:
2017-12-01
Target enrollment:
Participant gender:
Summary
Primary objectives:
- To establish the maximum tolerated dose (MTD), and the recommended Phase 2 dose (RP2D)
of BEZ235 when administered twice daily (BID) as a single agent in patients with
relapsed or refractory acute leukemia
- To determine the dose-limiting toxicity (DLT)
Secondary objectives:
- Assess the safety and tolerability of daily oral administration of BEZ235 with a BID
schedule
- To describe preliminary anti-leukemic activity of BEZ235 in patients with acute leukemia
- To correlate changes in pharmacodynamic biomarkers with basic pharmacokinetic data
Exploratory objectives:
- To assess pre-treatment phosphatidylinositol 3-kinase (PI3K) pathway-related genes in
blast cells and all other malignant cells derived from blood or bone marrow.
- To assess the pharmacodynamic changes in components of the PI3K-protein kinase B
(AKT)-mTOR pathway in bone marrow following treatment as potential predictive biomarkers
of pharmacodynamic (PD) activity of BEZ235 in association with clinical responses.
- To identify potential resistance mechanisms and biomarkers that may correlate with
efficacy and response from blood and bone marrow samples pre-and post-treatment in case
of resistance