Efficacy and safety of oral GKT137831
Research type
Research Study
Full title
A Double-Blind, Randomized, Placebo-Controlled Clinical Trial to Assess the Efficacy and Safety of Oral GKT137831 in Patients with Primary Biliary Cholangitis Receiving Ursodeoxycholic Acid and with Persistently Elevated Alkaline Phosphatase
IRAS ID
227127
Contact name
Michael Heneghan
Contact email
Sponsor organisation
Genkyotex SA
Eudract number
2016-004599-23
Clinicaltrials.gov Identifier
Duration of Study in the UK
1 years, 3 months, 0 days
Research summary
This Clinical Trial is being conducted to study patients with Primary Biliary Cholangitis (PBC) who are taking a stable dose of Ursodeoxycholic Acid (UDCA) treatment, and have persistently high levels of a liver enzyme called Alkaline Phosphatase (ALP). A total of 90 participants will participate in this study in different centers which may be located in USA, Canada, United Kingdom, Belgium, Israel, Italy, Greece and Spain.
GKT137831 is a “NOX inhibitor”, this means it is a molecule which inhibits the enzyme NADPH oxidase (NOX), which means that it decreases the activity of the enzyme. An enzyme is a protein in the body which facilitates or accelerates certain processes in the body. NOX produces substances such as superoxide or hydrogen peroxide and others, which are called free radicals. Those free radicals are – when present in small quantities – involved in normal cellular functions. However, they become toxic if they are present in excess amounts. The study drug blocks the activity of a subset of NOX enzymes which could be involved in the progression of primary biliary cholangitis.
Primary biliary cholangitis , also sometimes known as primary biliary cirrhosis, is a liver disease caused when the body’s immune system attacks structures in the liver, including the bile ducts. When the bile ducts are damaged, bile and other toxins build up in the liver (cholestasis) and over time damage the liver tissue in combination with the ongoing immune system-related damage. The disease may progress to chronic cholestasis, secondary inflammation and scarring (fibrosis), and may lead to liver cirrhosis and liver failure.
It is hoped that giving GKT137831 in combination with UDCA will reduce cholestasis, secondary inflammation and fibrosis in patients with PBC.REC name
Yorkshire & The Humber - Leeds East Research Ethics Committee
REC reference
17/YH/0186
Date of REC Opinion
28 Jun 2017
REC opinion
Further Information Favourable Opinion