Phase 2
N=110
A Study of Efruxifermin in Subjects With Histologically Confirmed Nonalcoholic Steatohepatitis (NASH)
NASH - Nonalcoholic Steatohepatitis
Bottom Line
View on ClinicalTrials.gov: NCT03976401 ↗Enrolled (actual)
110
Serious AEs
2.8%
Results posted
Aug 2022
Primary outcome: Primary: Main: Absolute Change From Baseline in Hepatic Fat Fraction Assessed by MRI-PDFF at Week 12. — -12.32; -13.44; -14.14; -0.29 Absolute percentage of hepatic fat
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- EFX (Drug); Placebo (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Akero Therapeutics, Inc
- Primary completion
- Feb 2021
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Main: Absolute Change From Baseline in Hepatic Fat Fraction Assessed by MRI-PDFF at Week 12. |
-12.32; -13.44; -14.14; -0.29 | — |
| SECONDARY Main: Absolute Change From Baseline in Hepatic Fat Fraction Assessed by MRI-PDFF at Week 22-24. |
-9.50; -8.99; -12.72; -14.45 | — |
| SECONDARY Main: Percent Change From Baseline in Hepatic Fat Fraction Measured by MRI-PDFF at Week 12. |
-63.21; -70.92; -72.26; -0.29 | — |
| SECONDARY Main: Percent Change From Baseline in Hepatic Fat Fraction Measured by MRI-PDFF at Week 22-24. |
-49.80; -43.37; -63.70; -69.87 | — |
| SECONDARY Main: Responder: Subjects Who Achieved a Clinically Meaningful Relative Reduction of at Least 30% in Liver Fat Content as Measured by MRI-PDFF at Week 12. |
16; 17; 15; 2 | — |
| SECONDARY Main: Responder Based on NAFLD Activity Score System (NAS): Subjects Who Had a Decrease of ≥2 Points in NAS With at Least a 1-point Reduction in Either Lobular Inflammation or Hepatocellular Ballooning and With no Concurrent Worsening of Fibrosis Stage. |
10; 10; 11; 1 | — |
| SECONDARY Main: Change From Baseline in ALT at Week 12, 16, and 20. |
-24.46; -30.47; -32.29; -5.94; -23.46; -31.99 | — |
| SECONDARY Cohort C: Change From Baseline in Liver Stiffness as Evaluated by FibroScan at Week 16 |
-37.6; 12.1 | — |
| SECONDARY Cohort C: Change From Baseline in Non-invasive Biomarkers Including Pro-C3 at Week 12, 16, and 20. |
-8.4; -2.8; -9.0; -3.4; -5.2; -4.9 | — |
| SECONDARY Cohort C: Change From Baseline in Non-invasive Biomarkers Including Liver Fibrosis by ELF Test Score at Week 12 and 16. |
-0.3; 0.3; -0.4; 0.3 | — |
Summary
This is a multi-center evaluation of efruxifermin (EFX) in a randomized, double-blind, placebo-controlled study administered for 16 weeks in subjects with biopsy proven F1 - F4 NASH.
Eligibility Criteria
Key Inclusion Criteria
- Males and non-pregnant, non-lactating females between 18 - 80 years of age inclusive, based on the date of the screening visit.
- Main Study only: Body mass index (BMI) > 25 kg/m^2 (unless the patient has biopsy-proven NASH documented within the last 2 years).
- Main Study only: Must have confirmation of ≥ 10% liver fat content on magnetic resonance imaging- proton density fat fraction (MRI-PDFF) at screening.
- Main Study only: Biopsy-proven NASH. Must have had a liver biopsy within 180 days of randomization with fibrosis stage 1 to 3 and a non-alcoholic fatty liver disease (NAFLD) activity score (NAS) of ≥ 4 with at least a score of 1 in each of the following NAS components:
- Steatosis (scored 0 to 3),
- Ballooning degeneration (scored 0 to 2), and
- Lobular inflammation (scored 0 to 3)
- Cohort C only: FibroScan® measurement > 13.1 kPa.
- Cohort C only: Cirrhosis due to NASH. Liver biopsy consistent with F4 fibrosis according to the NAS system, confirmed by the central or local reader.
Exclusion Criteria
- Weight gain or loss > 5% in the 3 months prior to randomization or > 10% in the 6 months prior to screening.
- Type 1 and insulin-dependent Type 2 diabetes.
- Poorly controlled hypertension (blood pressure > 160/100).
Data sourced from ClinicalTrials.gov (NCT03976401). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.