Refametinib in combination with sorafenib in RAS mutant HCC
Research type
Research Study
Full title
A prospective, single-arm, multicenter, uncontrolled, open-label Phase II trial of refametinib (BAY 86-9766) in combination with sorafenib as first line treatment in patients with RAS mutant Hepatocellular Carcinoma (HCC)
IRAS ID
131723
Contact name
Paul Ross
Contact email
Sponsor organisation
Bayer plc
Eudract number
2013-000241-39
ISRCTN Number
n/a
Clinicaltrials.gov Identifier
n/a
Research 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.REC name
East of England - Cambridge Central Research Ethics Committee
REC reference
13/EE/0227
Date of REC Opinion
16 Aug 2013
REC opinion
Favourable Opinion