Refametinib in Combination With Sorafenib in RAS Mutant Hepatocellular Carcinoma (HCC)
Status:
Completed
Trial end date:
2017-02-08
Target enrollment:
Participant gender:
Summary
This is a study to investigate the potential clinical benefit of refametinib when given in
combination with sorafenib as first line treatment 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 partial response according to an objective criteria to
evaluate tumor size based on contrast enhancement [modified response evaluation criteria in
solid tumors (mRECIST)] assessed by external independent radiologists.
Refametenib 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 in combination with sorafenib 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 in combination with sorafenib improves the response rate in this patient
population compared to historical results observed with the sorafenib only.