NCT01915602

Refametinib in Combination With Sorafenib in RAS Mutant Hepatocellular Carcinoma (HCC)

A Prospective, Single-arm, Multicenter, Uncontrolled, Open-label Phase II Trial of Refametinib (BAY86-9766) in Combination With Sorafenib as First Line Treatment in Patients With RAS Mutant Hepatocellular Carcinoma (HCC)

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 14 (actual)
• Sponsor: Bayer · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Carcinoma, Hepatocellular

Interventions

Type	Name	Description
DRUG	Refametinib (BAY86-9766)	Patients will receive refametinib 50 mg (2x20 mg + 1x10mg capsules or 50 mg tablets) bid
DRUG	Sorafenib (BAY43-9006)	Patients will receive sorafenib 400 mg (2 x 200 mg tablets) bid.

Timeline

• Start date: 2013-09-27
• Primary completion: 2015-07-29
• Completion: 2017-02-08
• First posted: 2013-08-05
• Last updated: 2021-04-08

Locations

79 sites across 21 countries: United States, Austria, Belgium, China, Czechia, France, Germany, Hong Kong, Hungary, Israel, Italy, Japan, New Zealand, Singapore, South Korea, Spain, Switzerland, Taiwan, Thailand, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study