Refametinib(BAY86-9766) in RAS Mutant Hepatocellular Carcinoma (HCC)
Status:
Completed
Trial end date:
2014-10-08
Target enrollment:
Participant gender:
Summary
This is a study to investigate the potential clinical benefit of refametinib in patients with
unresectable or metastatic HCC carrying a RAS mutation. The study will be conducted in 2
stages. Approximately 95 patients (15 at Stage 1/ 80 at Stage 2) will be accrued to this
study to receive treatment. Stage 2 of the trial will only be conducted if at least 5 out of
15 patients at Stage 1 show at least confirmed partial response (PR) according to modified
response evaluation criteria in solid tumors (mRECIST) assessed by central image review.
Refametinib is an oral (i.e. taken by mouth) protein kinase inhibitor. A kinase inhibitor
targets certain key proteins that are essential for the survival of the cancer cell. By
specifically targeting these proteins, refametinib may stop cancer growth. The growth of the
tumor may be decreased by preventing these specific proteins from functioning.
The primary endpoint (the most meaningful result to be tracked) of this study is based on the
rate of response, i.e. the disease getting smaller. The aim is to show that the therapy with
refametinib improves the response rate in this RAS mutation patient population.