Phase 2 Study of AXA1665 in Liver Cirrhosis and Prior OHE (EMMPOWER)
Research type
Research Study
Full title
A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of AXA1665 in Subjects With Liver Cirrhosis and Prior Overt Hepatic Encephalopathy (EMMPOWER)
IRAS ID
296436
Contact name
Deborah Shawcross
Contact email
Sponsor organisation
Axcella Health, Inc.
Eudract number
2020-005969-13
Clinicaltrials.gov Identifier
Duration of Study in the UK
1 years, 5 months, 5 days
Research summary
Hepatic encephalopathy (HE) is a reversible syndrome of impaired cognitive and neuromuscular function estimated to affect between 30% and 70% of patients with liver cirrhosis. When the liver is damaged, it is unable to remove toxins such as ammonia, which contributes to the pathogenesis. Cognitive impairment ranges from minimal (covert) to overt hepatic encephalopathy (OHE), which is characterised by symptoms such as confusion, disorientation and behavioural changes as well as muscle loss and fatigue. It negatively impacts multiple aspects of the lives of patients, and also has significant burden on caregivers and healthcare systems. This means that early diagnosis and management of OHE is imperative.
This study drug, AXA1665, has been developed by Axcella Health, Inc. as a treatment for reducing the risk of OHE in patients with liver cirrhosis. It is made up of a mixture of 8 different amino acids supplied as a dry powder to be reconstituted in water. AXA1665 is thought to normalise amino acids in the liver and improve ammonia metabolism. A mixture of 2 different amino acids has previously been shown to be beneficial in treatment of HE.
This is a Phase 2, multicentre, randomised, double-blind, placebo-controlled study to evaluate the efficacy and safety of AXA1665 compared to placebo in patients with liver cirrhosis and prior overt hepatic encephalopathy. The primary objective is to assess the efficacy of AXA1665 on neurocognitive function.
Approximately 150 eligible subjects will be randomised into 1 of 2 treatment groups in a 1:1 ratio to receive either placebo or AXA1665. Participants will take the study drug 3 times daily with food for 24 weeks. The total duration for participation is approximately 32 weeks (including screening and follow-up periods). Participants will need the support of a primary caregiver for the entire duration of the study.
REC name
London - Brent Research Ethics Committee
REC reference
21/LO/0492
Date of REC Opinion
23 Sep 2021
REC opinion
Further Information Favourable Opinion