EMMPACT
Research type
Research Study
Full title
A Randomized, Double-Blind, Placebo-Controlled, Dose Ranging Study to Evaluate the Safety, Tolerability, and Efficacy of AXA1125 in Subjects With Non-Cirrhotic, Non-Alcoholic Steatohepatitis and Fibrosis (EMMPACT)
IRAS ID
301806
Contact name
Kosh Agarwal
Contact email
Sponsor organisation
Axcella Health, Inc.
Eudract number
2021-000685-15
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
150562.1, IND
Duration of Study in the UK
2 years, 0 months, 0 days
Research summary
Non-alcoholic fatty liver disease (NAFLD) is the most common cause of abnormal liver enzymes worldwide, affecting 20% to 30% of the general population in the United States. Approximately 20% of patients with NAFLD develop non-alcoholic steatohepatitis (NASH). NASH causes a build-up of fat and inflammation (a response to injury) in the liver, accompanied by varying amounts of scarring (fibrosis). Over time, fibrosis can get worse and lead to extensive scarring of the liver (cirrhosis). Patients with NASH have an increased risk of liver failure, liver cancer, and heart disease. Currently, there is no approved treatment for patients with NASH, although there are a number of drugs in development. Given the multiple pathways involved in the pathogenesis of NASH, it is likely that the effective management of the disease may require combination treatments or treatments with a multimodal action. Supplementation of amino acids (AAs) (individually or in combinations of a few AAs) has been previously shown to impact specific aspects of NASH pathophysiology, including insulin resistance, build-up of fat within the liver and inflammation. AXA1125 is an orally active mixture of 5 Amino Acids (ie, leucine, isoleucine, valine, arginine, and glutamine) and an AA derivative N-acetyl L-cysteine (Nac) with complementary modes of action and the potential to impact core metabolic drivers of NASH while simultaneously modulating pathways related to inflammation and fibrosis. This is a randomised, double-blind placebo-controlled study to assess the safety of the study drug AXA1125 and how well it works in improving metabolism (how the body breaks down substances like fats and sugars), reduces injury to the liver, and reduces scarring in the liver (fibrosis) in people with non-cirrhotic non-alcoholic steatohepatitis (NASH) and fibrosis. Participation in this study is expected to be 60 weeks.
REC name
East Midlands - Leicester Central Research Ethics Committee
REC reference
21/EM/0198
Date of REC Opinion
4 Oct 2021
REC opinion
Further Information Favourable Opinion