Non Alcoholic Steatohepatitis (NASH)
Conditions
Keywords
Steatosis, Lobular inflammation, Ballooning, Liver biopsy, Liver fat, Liver stiffness, NASH, Aminio Acids, Fibrosis
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.
Interventions
DRUGAXA1125
AXA1125 administered BID with or without food
DRUGPlacebo
Matching Placebo administered BID with or without food
Sponsors
Axcella Health, Inc
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Improvement in steatohepatitis | 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
| Measure | Time frame | Description |
|---|---|---|
| Resolution of NASH without worsening of fibrosis | Baseline to week 48 | The proportion of subjects with resolution of NASH with no worsening of fibrosis defined as a post-treatment ballooning score of 0 and an inflammation score of 0 or 1 on liver biopsy. |
| Improvement of fibrosis by one stage without worsening of NASH | Baseline to week 48 | The proportion of subjects who have at least a 1-stage improvement in fibrosis and no worsening of NASH based on liver biopsy 0 being no fibrosis and 4 being the worse. |
| 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 | — |
Countries
Australia, Canada, France, Poland, Puerto Rico, United Kingdom, United States
Contacts
Primary ContactMargaret Koziel, MD
Outcome results
None listed