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Report for Clinical Trial NCT01756118

Developed by Shray Alag, 2020.
SNP Clinical Trial Gene

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

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

NCT01756118 Acute Lymphoblastic Leukemia Leukemia, Myelocytic, Acute Chronic Myelogenous Leukemia With Crisis of Blast Cells
MeSH:Leukemia Precursor Cell Lymphoblastic Leukemia-Lymphoma Leukemia, Myeloid Leukemia, Myelogenous, Chronic, BCR-ABL Positive Leukemia, Myeloid, Acute
HPO:Acute megakaryocytic leukemia Acute myeloid leukemia Chronic myelogenous leukemia Leukemia Myeloid leukemia

1 Interventions

Name: BEZ235

Description: A minimum of 3 dose levels
Type: Drug
Group Labels: 1


Primary Outcomes

Measure: Recommended Phase 2 dose (RP2D) of BEZ235 in acute lymphoblastic leukemia (ALL), acute myeloid leukemia (AML) and chronic myeloid leukemia in blastic phase (CML-BP)

Time: 6 months after inclusion of first patient

Measure: Incidence of dose-limiting toxicity (DLT)

Time: 6 months after inclusion of first patient

Secondary Outcomes

Measure: Adverse events as graded by NCI Common Terminology Criteria for Adverse Events (CTCAE) v4.

Time: 1.5 years

Measure: Investigator-reported best overall response (BOR) as determined from the response categories during treatment period.

Time: 1.5 years

Measure: Time to progression

Time: 1.5 years

Measure: Remission duration

Time: 1.5 years

Measure: Biomarker evaluation

Time: 1.5 years

Measure: BEZ235 and metabolite levels in plasma

Time: 1.5 years

Measure: Clinical laboratory tests as graded by NCI CTCAE v4.

Time: 1.5 years

Purpose: Treatment

Allocation: N/A

Single Group Assignment

There is one SNP


1 T315I

If a point mutation threonine 315 to isoleucine (T315I) bcr-abl mutation has been identified, prior treatment with a second TKI is not required. --- T315I ---

or: 3. Patients with Philadelphia chromosome and/or bcr-abl positive B-precursor ALL or prior CML-BP with presence of minimal residual disease (MRD) and the presence of T315I mutation or a high-resistance mutation shown to be unresponsive to approved thyrosine kinase inhibitors (TKI). --- T315I ---

HPO Nodes

Protein Mutations 0