US-based biopharmaceutical firm Ocera Therapeutics has enrolled the first patient in its multi-centre Phase IIb trial ‘STOP-HE’ or OCR002-HE209 trial designed to treat acute hepatic encephalopathy (HE).
The trial is designed to assess the efficacy, safety and pharmacokinetics of OCR-002, or ornithine phenylacetate, in hospitalised patients with liver cirrhosis and an acute episode of HE.
Hepatic encephalopathy is a serious problem of liver failure and decompensated cirrhosis in the setting of chronic liver disease, where increase of ammonia concentration in the blood and brain can lead to deterioration of neurocognitive function, ranging from subtle abnormalities to frank (overt) disorientation, stupor, coma and death.
The Phase IIb trial is a randomised double-blind, controlled study of OCR-002 plus current standard of care versus standard of care alone.
Around 200 patients will be enrolled in the trial, which will be carried out at around 40 centres in the US and Canada.
The trial is expected to be completed in early 2015, and will assess the time to achievement of meaningful improvement in HE symptoms.
Center for Liver Disease and Transplantation director Robert Brown and professor of medicine and paediatrics Frank Cardile said: "HE is a serious disorder that leads to approximately 150,000 hospitalisations per year in the United States and for which the best available treatments are still suboptimal."
OCR-002 is an ammonia scavenger that is intended to reduce the concentration of ammonia in the blood when given as a continuous intravenous infusion.
The US Food and Drug Administration (FDA) has also granted orphan drug designation to OCR-002 for the treatment of hyperammonemia and resultant HE in patients with acute liver failure or acute on chronic liver disease.
Image: Abdominal computed tomography of a three year old child showing liver cirrhosis in transverse section. Photo: courtesy of Christaras A.