AK-US-001-0106
Research type
Research Study
Full title
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Safety and Efficacy of Efruxifermin in Subjects with Compensated Cirrhosis Due to Nonalcoholic Steatohepatitis (NASH)/Metabolic Dysfunction-Associated Steatohepatitis (MASH)
IRAS ID
1010406
Contact name
Chris Ayscue-Tse
Contact email
Sponsor organisation
Akero Therapeutics, Inc.
Clinicaltrials.gov Identifier
Research summary
This study is taking place in 330 sites worldwide with 1150 patients who have compensated liver cirrhosis due to nonalcoholic steatohepatitis (NASH)/metabolic dysfunction-associated steatohepatitis (MASH). NASH/MASH is the most severe form of nonalcoholic fatty liver disease (NAFLD)/metabolic dysfunction-associated steatotic liver disease (MASLD) making it a global health burden. This is characterised by excessive accumulation of fat in the liver and inflammation. There are no approved therapies for the treatment of NASH/MASH in the United Kingdom. The current standard of care is weight loss through lifestyle modifications. Diet and exercise are effective in the treatment of NASH/MASH when maintained, but adherence to a modified lifestyle is quite poor, highlighting the unmet need to develop therapies against NASH/MASH. The purpose of this study is see if the compound, efruxifermin (EFX), is effective in treating compensated cirrhosis due to NASH/MASH. The study will have 2 parts, Part 1 and Part 2. The main difference is that Part 1 requires collection of liver biopsies. All participants must provide written consent to joining the study. After Screening, eligible participants will be randomly assigned to 1 of 2 treatment groups; a placebo group and a group that will receive 50 mg of EFX. Both groups will receive study drug via once weekly subcutaneous injections. Participation in the study is anticipated to take 5 years. After Screening, site visits for safety and disease-related assessments will be performed. Participants are required to return to study site 30 days after last dose of study treatment. This study includes blood sampling and DXA scans. An external Data Monitoring Committee (DMC) will review study progress. Participant's true study duration may differ.
REC name
London - Chelsea Research Ethics Committee
REC reference
24/LO/0568
Date of REC Opinion
17 Oct 2024
REC opinion
Further Information Favourable Opinion