A phase 1 study of oral ridaforolimus in pediatric patients with advanced solid tumors.

ABSTRACT:

Purpose

Ridaforolimus is an investigational, potent, selective mTOR inhibitor. This study was conducted to determine the recommended phase 2 dose (RP2D), maximum tolerated dose, safety, pharmacokinetics, and antitumor activity of oral ridaforolimus in children with advanced solid tumors.

Experimental design

In this phase 1, multicenter, open-label study in children aged 6 to <18 years with advanced solid tumors, ridaforolimus was administered orally for 5 consecutive days/week in 28-day cycles until progression, unacceptable toxicity, or consent withdrawal. Dose started at 22 mg/m2 and increased to 28 mg/m2 and 33 mg/m2, followed by expansion at the RP2D.

Results

Twenty patients were treated; 18 were evaluable for dose-limiting toxicities. One dose-limiting toxicity (

SUBMITTER: Pearson AD 

PROVIDER: S-EPMC5356695 | biostudies-literature | 2016 Dec

REPOSITORIES: biostudies-literature