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)
Overview
- Phase
- Phase 2
- Intervention
- AXA1125
- Conditions
- Non Alcoholic Steatohepatitis (NASH)
- Sponsor
- Axcella Health, Inc
- Enrollment
- 273
- Locations
- 69
- Primary Endpoint
- Improvement in steatohepatitis
- Last Updated
- 3 years ago
Overview
Brief Summary
This study will compare the effects of AXA1125, an orally active mixture of amino acids, compared to placebo, on improving fat and inflammation (steatohepatitis) as well as fibrosis in subjects with non alcoholic steatohepatitis (NASH). as well as the safety and tolerability of AXA1125. Subjects will take one of two different doses of AXA1125 or a placebo twice daily, and a liver biopsy will be done at the beginning and end of the 48-week study.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Willing to participate in the study and provide written informed consent.
- •Male and female adults aged \> 18 years.
- •Must have NASH and fibrosis on a liver biopsy sample
- •If a historical liver biopsy is used for Screening, obtained within 6 months prior to Screening;
- •Subjects may have a diagnosis of T2DM
Exclusion Criteria
- •History or presence of liver disease (other than NAFLD or NASH)
- •History or presence of cirrhosis and/or history or presence of hepatic decompensation
Arms & Interventions
AXA1125 22.6g
22.6 g AXA1125 administered orally BID with or without food
Intervention: AXA1125
AXA1125 33.9g
33.9 g AXA1125 administered orally BID with or without food
Intervention: AXA1125
Placebo
Matching Placebo administered orally BID with or without food
Intervention: Placebo
Outcomes
Primary Outcomes
Improvement in steatohepatitis
Time Frame: Baseline to Week 48
2-point improvement from baseline to Week 48 in the non-alcoholic fatty liver disease (NAFLD) Activity Score (NAS) based on a scale from 0-8 with 0 being no NASH and 8 being the highest score
Secondary Outcomes
- Resolution of NASH without worsening of fibrosis(Baseline to week 48)
- Improvement of fibrosis by one stage without worsening of NASH(Baseline to week 48)
- Incidence of study drug emergent adverse events (AEs) and serious adverse events (SAEs)(Baseline to week 48)
- Change from baseline in liver stiffness as measured by vibration controlled transient elastography (Fibroscan™)(Baseline to week 48)
- Change from baseline in hepatic fat as measured by MRI(Baseline to week 48)
- Change from baseline in measures of glucose control as determined by glycated hemoglobin (HbA1c)(Baseline to week 48)