Results of the Phase I Trial of RG7112, a Small-Molecule MDM2 Antagonist in Leukemia.

ABSTRACT:

Purpose

RG7112 is a small-molecule MDM2 antagonist. MDM2 is a negative regulator of the tumor suppressor p53 and frequently overexpressed in leukemias. Thus, a phase I study of RG7112 in patients with hematologic malignancies was conducted.

Experimental design

Primary study objectives included determination of the dose and safety profile of RG7112. Secondary objectives included evaluation of pharmacokinetics; pharmacodynamics, such as TP53-mutation status and MDM2 expression; and preliminary clinical activity. Patients were divided into two cohorts: Stratum A [relapsed/refractory acute myeloid leukemia (AML; except acute promyelocytic leukemia), acute lymphoblastic leukemia, and chronic myelogenous leukemia] and Stratum B (relapsed/refractory chronic lymphocytic leukemia/sma

SUBMITTER: Andreeff M 

PROVIDER: S-EPMC4809642 | biostudies-literature | 2016 Feb

REPOSITORIES: biostudies-literature