A Multi-Center, Placebo-Controlled Randomized Pilot of the Effect of Sorafenib on Portal Pressure in Patients With Cirrhosis, Portal Hypertension and Hepatocellular Carcinoma Treated With Ablative Therapy and/or Transarterial Chemoembolization
This is a pilot proof-of-concept study that investigates the effect of sorafenib on portal
pressure, as determined by the hepatic venous pressure gradient (HVPG), in patients with
liver cirrhosis, portal hypertension and unresectable hepatocellular carcinoma (HCC) that
has successfully responded to radiofrequency ablation and/or transarterial
chemoembolization, and have obtained a complete response.
The primary end-point of the study is the change in HVPG observed from baseline to three
months after starting treatment with sorafenib. Secondary end-point is safety of sorafenib.
The trial is structured as a randomized double blind placebo controlled study. After a
three-month period of therapy with sorafenib or placebo (double-blind phase), patients will
be given open-label sorafenib for an additional 3-month period (open-label phase). A total
of 44 patients will be randomized (in the initial phase) on a 1:1 ratio to sorafenib or
placebo. Patients will be followed monthly and HCC follow-up will be according to standards
of care. The study will be sponsored by Onyx, who will also provide the treatment medication
(sorafenib and placebo)
Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator), Primary Purpose: Treatment
Change in HVPG from baseline
Change in HVPG three months after initiation of sorafenib.
Guadalupe Garcia-Tsao, MD
United States: Institutional Review Board
HIC # 1002006266
|Yale Cancer Center||New Haven, Connecticut 06520-8028|
|The University of Texas Health Science Center at Houston||Houston, Texas 77030|