Multicentre, Randomized, Double Blind, Placebo-Controlled Study to Ev

• Research type

  Research Study

• Full title

  A Study to Evaluate the Efficacy and Safety of GFT505 once daily on Steatohepatitis in Patients with Non-Alcoholic Steatohepatitis (NASH). A Multicentre, Randomized, Double Blind, Placebo-Controlled study, with an adaptive design to allow for initial GFT505 80mg dosing versus placebo, followed by a second phase including GFT505 120mg dose, after review of 6-month safety analysis of the 80mg data on at least 50% of patients.

• IRAS ID

  112730

• Contact name

  Quentin Anstee

• Sponsor organisation

  GENFIT

• Eudract number

  2012-000295-42

• ISRCTN Number

  N/A

• Clinicaltrials.gov Identifier

  N/A

• Research summary

  Non-alcoholic fatty liver disease (NAFLD) is one cause of a fatty liver, occurring when fat is deposited in the liver not due to excessive alcohol use. It is related to insulin resistance and the metabolic syndrome. Over a long period, the disease progression may lead to an advanced condition called Non-alcoholic Steatohepatitis (NASH). NASH is the most extreme form of NAFLD, is characterized by inflammation of the liver with concurrent fat accumulation in the liver and may progress to cirrhosis. Common conditions associated with fatty liver and NASH include obesity, type 2 diabetes mellitus, high levels of triglyceride (fat) in the blood and high blood pressure. Today NAFLD is one of the most common forms of liver disease and NASH is believed to be a frequent cause of unexplained cirrhosis (at least in Western societies). So far it is not known what the precise prevalence of NASH in the British population is, but studies in the United States (US) suggest that up to 90% of obese individuals have fatty liver, and around 25% of them have NASH. Since the present prevalence of obesity in the United Kingdom is around 30%, the expected prevalence of fatty liver is around 20-27% including two thirds not related to alcohol or virus. Up to now there is no specific treatment for NASH and patients are mainly provided with lifestyle and dietary recommendations. GFT505 is a new liver targeted investigational medicinal product. It was developed for the treatment of NASH and reduction of the multiple cardiometabolic risk factors associated with the metabolic syndrome and type 2 Diabetes. In addition to lifestyle and dietary recommendations, GFT505 may benefit patients and therefore, improve the medical management of their disease. In this study, the primary objective is to demonstrate the efficacy and safety of GFT505 compared to placebo in reversing NASH. A total of 270 patients from 75 centers in Europe and US will be enrolled in the study. After a maximum of 16 weeks screening period, the suitable patients will be randomly divided into two groups, and will receive either the placebo or GFT505 at a dose of 80mg. When 45 patients will have received GFT505 80mg for 6 months, the safety data will be analyzed by a Data Safety Monitoring Board. If the analysis are satisfactory, GFT505 at a dose of 120mg will be introduced and new patients will be enrolled and will receive either the placebo, GFT505 80 mg or GFT505 120mg. At the end of the study 90 patients will have received GFT505 80mg, 90 will have received GFT505 120 mg and 90 will have received the placebo. The patients will receive the treatment for up to 52 weeks and will then be followed-up during 3 months. The whole study duration is 82 weeks maximum.

• REC name

  North East - Newcastle & North Tyneside 1 Research Ethics Committee

• REC reference

  12/NE/0342

• Date of REC Opinion

  2 Jan 2013

• REC opinion

  Further Information Favourable Opinion