Spots Global Cancer Trial Database for LDE225 in Patients With Advanced or Metastatic Hepatocellular Carcinoma and Child-Pugh A/B7 Cirrhosis

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Trial Identification

Brief Title: LDE225 in Patients With Advanced or Metastatic Hepatocellular Carcinoma and Child-Pugh A/B7 Cirrhosis

Official Title: Phase Ib Study of Single Agent LDE225, an Oral Hedgehog Inhibitor, as Second-Line Therapy in Patients With Advanced or Metastatic Hepatocellular Carcinoma and Child-Pugh A/B7 Cirrhosis

Study ID: NCT02151864

Conditions

Hepatocellular Carcinoma

Cirrhosis

Interventions

LDE225

Study Description

Brief Summary: The purpose of this study is to test the safety and determine the maximum safe dose of an experimental drug called LDE225 (hedgehog inhibitor) in people with liver cancer. We have identified hedgehog dysregulation as a novel mechanism for hepatocarcinogenesis and hepatic fibrosis/cirrhosis. Therefore, we hypothesize that the hedgehog inhibitor may be an ideal drug target for treating both hepatocellular carcinoma (HCC) and Child-Pugh A cirrhosis (CPA).

Detailed Description: This is an open-label, single institution, Phase I, 3+3 dose escalation study of LDE225 in patients with advanced or metastatic hepatocellular carcinoma and Child-Pugh A Cirrhosis who are intolerant to sorafenib. The investigational treatment cycle (21 days) will consist of daily oral LDE225. Dose escalation will be performed in serial patient cohorts. Up to six patients can be studied at each dose level. Safety and clinical data will be tabulated and the decision to open the next cohort level will be the responsibility of the principal investigator. Dose escalation will be based on the dose-limiting toxicities encountered through Day 42 of investigational treatment. Patients will be dosed on a flat scale of daily dosing of LDE225 prescribed by the dose cohort.