AK-US-001-0107
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 Non-Invasively Diagnosed Nonalcoholic Steatohepatitis (NASH)/Nonalcoholic Fatty Liver Disease (NAFLD)
IRAS ID
1008071
Contact name
Chris Ayscue-Tse
Contact email
Sponsor organisation
Akero Therapeutics, Inc.
Research summary
This study is taking place in approximately 260 sites worldwide with 480 patients being enrolled and a possible 100 patients being enrolled to the open-label rollover study. Nonalcoholic steatohepatitis (NASH) is the most severe form of nonalcoholic fatty liver disease (NAFLD), making it a global health burden. It is characterised by excessive accumulation of fat in the liver and inflammation. Although there are no presenting symptoms of NAFLD, symptoms may include fatigue and mild jaundice. There are no approved therapies for treating NASH. Current standard of care is weight loss through lifestyle modifications, e.g. avoidance of sugar and alcohol in the diet. Although diet and exercise are effective in maintaining the symptoms of NASH, adhering to a modified lifestyle is poor, highlighting the unmet need to develop therapies that slow or halt the progression of NASH. The purpose of this study is to see if study treatment, Exfruxifermin (EFX) is effective in treating NASH/NAFLD by improving energy metabolism in the liver. After Screening assessments, eligible participants will be randomly assigned to 1 of 2 treatment groups; a placebo group and an experimental group that will receive EFX, 50mg. Participants in the open-label rollover study will receive EFX 50 mg. All groups will receive subcutaneous injections once weekly. The study is anticipated to last 68 weeks, including a 12-week screening period, 52-week treatment period and a 30-day follow up visit. After Screening, site visits for safety and disease-related assessments will be performed for up to 52 weeks. Participants need to return to study site 30 days after last dose of study treatment. This study includes blood sampling and Dual-energy X-ray Absorptiometry (DXA) Scans. An external Data Monitoring Committee (DMC) will review the progress of the study. Participants’ true study duration may be longer or shorter due to deviance from assumptions on participant enrolment and other aspects of study conduct.
REC name
South Central - Berkshire Research Ethics Committee
REC reference
23/SC/0313
Date of REC Opinion
18 Dec 2023
REC opinion
Further Information Favourable Opinion