NCT01756118

A Phase I, Dose-finding Study of BEZ235 in Adult Patients With Relapsed or Refractory Acute Leukemia

A Phase I, Dose-finding Study of the Oral, Dual Phosphatidylinositol 3(PI3)-Kinase / Mammilian Target of Rapamycin (mTOR) Inhibitor BEZ235 in Adult Patients With Relapsed or Refractory Acute Leukemia

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 24 (actual)
• Sponsor: Goethe University · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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

Conditions

• Acute Lymphoblastic Leukemia
• Leukemia, Myelocytic, Acute
• Chronic Myelogenous Leukemia With Crisis of Blast Cells

Interventions

Type	Name	Description
DRUG	BEZ235	A minimum of 3 dose levels

Timeline

• Start date: 2012-06-01
• Primary completion: 2017-12-01
• Completion: 2020-02-07
• First posted: 2012-12-24
• Last updated: 2023-02-16

Locations

1 site across 1 country: Germany