A Study of Efruxifermin in Subjects With Histologically Confirmed Nonalcoholic Steatohepatitis (NASH)

A Phase 2a, Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Safety and Efficacy of Efruxifermin in Subjects With Nonalcoholic Steatohepatitis (NASH)

Status

Completed

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT03976401

Enrollment

110

Registered

2019-06-06

Start date

2019-05-28

Completion date

2022-01-10

Last updated

2022-08-04

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

NASH - Nonalcoholic Steatohepatitis

Brief 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.

Interventions

DRUGEFX

Administered by subcutaneous injection

DRUGPlacebo

Administered by subcutaneous injection

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)

Eligibility

Sex/Gender

ALL

Age

18 Years to 80 Years

Healthy volunteers

Inclusion 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).

Design outcomes

Primary

Measure	Time frame	Description
Main: Absolute Change From Baseline in Hepatic Fat Fraction Assessed by MRI-PDFF at Week 12.	12 weeks	Main study. ANCOVA multiple imputation with treatment group and F1 fibrosis score (F1 vs F2-3) as factors and baseline hepatic fat fraction measured by MRI-PDFF as a covariate.

Secondary

Measure	Time frame	Description
Main: Percent Change From Baseline in Hepatic Fat Fraction Measured by MRI-PDFF at Week 12.	12 weeks	Main study. ANCOVA multiple imputation with treatment group and F1 fibrosis score (F1 vs F2-3) as factors and baseline hepatic fat fraction measured by MRI-PDFF as a covariate.
Main: Percent Change From Baseline in Hepatic Fat Fraction Measured by MRI-PDFF at Week 22-24.	22-24 weeks	Main study. ANCOVA multiple imputation with treatment group and F1 fibrosis score (F1 vs F2-3) as factors and baseline hepatic fat fraction measured by MRI-PDFF as a covariate.
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.	12 weeks	Main Study. The analyses included the treatment group and F1 fibrosis score (F1 vs F2-3) as factors and baseline hepatic fat fraction measured by MRI-PDFF as a covariate.
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.	22-24 weeks	Main study: Responders were defined for subjects with ≥ 30% relative fat reduction on MRI-PDFF at Week 12 and required to return between Weeks 22 - 24. Fisher's exact test was used for the analysis using the Full Analysis Set with missing values imputed as non-responders and repeated on Liver Biopsy Evaluable Analysis Set without imputation.
Main: Absolute Change From Baseline in Hepatic Fat Fraction Assessed by MRI-PDFF at Week 22-24.	22-24 weeks	Main study. Included subjects with ≥30% relative fat reduction on MRI-PDFF at Week 12 that were required to return between Weeks 22 - 24. ANCOVA model with treatment group and F1 fibrosis score (F1 vs F2-3) as factors and baseline hepatic fat fraction as a covariate were performed.
Cohort C: Change From Baseline in Liver Stiffness as Evaluated by FibroScan at Week 16	16 weeks	Cohort C: ANCOVA model with treatment group as a factor and baseline liver stiffness as evaluated by FibroScan® as a covariate using the Full Analysis Set. Missing values at Week 16 were imputed using the last-observed-carried-forward (LOCF) method.
Cohort C: Change From Baseline in Non-invasive Biomarkers Including Pro-C3 at Week 12, 16, and 20.	12, 16, and 20 weeks	Cohort C. ANCOVA model with treatment group as a factor and baseline liver stiffness as evaluated by FibroScan® as a covariate using the Full Analysis Set. Missing values were imputed using the last-observed-carried-forward (LOCF) method.
Cohort C: Change From Baseline in Non-invasive Biomarkers Including Liver Fibrosis by ELF Test Score at Week 12 and 16.	12 and 16 weeks	Cohort C. The enhanced liver fibrosis (ELF) score describes the severity of liver fibrosis where a score of \<7.7 indicates no or mild fibrosis, a score of ≥7.7 to \<9.8 indicates moderate fibrosis, and a score of ≥9.8 indicates severe fibrosis. A change from baseline with a negative value indicates a decrease in severity of liver fibrosis. An ANCOVA model with treatment group as a factor and baseline liver stiffness as evaluated by FibroScan® as a covariate using the Full Analysis Set was used. Missing values were imputed using the last-observed-carried-forward (LOCF) method.
Main: Change From Baseline in ALT at Week 12, 16, and 20.	12, 16, and 20 weeks	ANCOVA model with treatment group, baseline hepatic fat fraction (\<15% vs ≥15%), and F1 fibrosis score (F1 vs F2-3) as factors and baseline value as a covariate.

Countries

Puerto Rico, United States

Participant flow

Participants by arm

Arm	Count
Main EFX 28 mg Main Study EFX: Administered by subcutaneous injection	19
Main EFX 50 mg Main Study EFX: Administered by subcutaneous injection	20
Main EFX 70 mg Main Study EFX: Administered by subcutaneous injection	20
Main Placebo Main Study Placebo: Administered by subcutaneous injection	21
Cohort C EFX 50 mg Cohort C EFX: Administered by subcutaneous injection	20
Cohort C Placebo Cohort C Placebo: Administered by subcutaneous injection	10
Total	110

Withdrawals & dropouts

Period	Reason	FG000	FG001	FG002	FG003	FG004	FG005
Overall Study	Adverse Event	2	0	4	1	1	0
Overall Study	Lost to Follow-up	1	2	0	0	0	0
Overall Study	Physician Decision	0	0	1	0	0	0
Overall Study	Protocol Violation	0	1	0	0	0	0
Overall Study	Withdrawal by Subject	0	1	0	0	0	1
Overall Study	Withdrew Consent	0	0	0	1	0	0

Baseline characteristics

Characteristic	Main EFX 28 mg	Total	Cohort C Placebo	Cohort C EFX 50 mg	Main Placebo	Main EFX 70 mg	Main EFX 50 mg
Age, Continuous	50.4 years STANDARD_DEVIATION 12.4	54.4 years STANDARD_DEVIATION 12.3	57.1 years STANDARD_DEVIATION 14.4	61.1 years STANDARD_DEVIATION 10	52.4 years STANDARD_DEVIATION 9.57	53.0 years STANDARD_DEVIATION 13.22	52.6 years STANDARD_DEVIATION 14.19
Ethnicity (NIH/OMB) Hispanic or Latino	12 Participants	53 Participants	5 Participants	8 Participants	10 Participants	7 Participants	11 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	7 Participants	57 Participants	5 Participants	12 Participants	11 Participants	13 Participants	9 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants
Race (NIH/OMB) Asian	0 Participants	1 Participants	0 Participants	0 Participants	1 Participants	0 Participants	0 Participants
Race (NIH/OMB) Black or African American	0 Participants	5 Participants	0 Participants	1 Participants	1 Participants	1 Participants	2 Participants
Race (NIH/OMB) More than one race	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants	1 Participants	0 Participants	1 Participants	0 Participants	0 Participants	0 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants
Race (NIH/OMB) White	19 Participants	103 Participants	10 Participants	18 Participants	19 Participants	19 Participants	18 Participants
Region of Enrollment United States	19 participants	110 participants	10 participants	20 participants	21 participants	20 participants	20 participants
Sex: Female, Male Female	10 Participants	65 Participants	3 Participants	16 Participants	15 Participants	11 Participants	10 Participants
Sex: Female, Male Male	9 Participants	45 Participants	7 Participants	4 Participants	6 Participants	9 Participants	10 Participants

Adverse events

Event type	EG000 affected / at risk	EG001 affected / at risk	EG002 affected / at risk	EG003 affected / at risk	EG004 affected / at risk	EG005 affected / at risk
deaths Total, all-cause mortality	0 / 19	0 / 19	0 / 20	0 / 21	0 / 20	0 / 10
other Total, other adverse events	18 / 19	17 / 19	19 / 20	16 / 21	19 / 20	8 / 10
serious Total, serious adverse events	1 / 19	0 / 19	1 / 20	0 / 21	0 / 20	1 / 10

Outcome results

Primary

Main: Absolute Change From Baseline in Hepatic Fat Fraction Assessed by MRI-PDFF at Week 12.

Main study. ANCOVA multiple imputation with treatment group and F1 fibrosis score (F1 vs F2-3) as factors and baseline hepatic fat fraction measured by MRI-PDFF as a covariate.

Time frame: 12 weeks

Population: Full Analysis Set (all randomized subjects)

Arm	Measure	Value (LEAST_SQUARES_MEAN)	Dispersion
Main Study EFX 28 mg	Main: Absolute Change From Baseline in Hepatic Fat Fraction Assessed by MRI-PDFF at Week 12.	-12.32 Absolute percentage of hepatic fat	Standard Error 1.04
Main Study EFX 50 mg	Main: Absolute Change From Baseline in Hepatic Fat Fraction Assessed by MRI-PDFF at Week 12.	-13.44 Absolute percentage of hepatic fat	Standard Error 1.04
Main Study EFX 70 mg	Main: Absolute Change From Baseline in Hepatic Fat Fraction Assessed by MRI-PDFF at Week 12.	-14.14 Absolute percentage of hepatic fat	Standard Error 1.04
Main Study Placebo	Main: Absolute Change From Baseline in Hepatic Fat Fraction Assessed by MRI-PDFF at Week 12.	-0.29 Absolute percentage of hepatic fat	Standard Error 0.98

Secondary

Cohort C: Change From Baseline in Liver Stiffness as Evaluated by FibroScan at Week 16

Cohort C: ANCOVA model with treatment group as a factor and baseline liver stiffness as evaluated by FibroScan® as a covariate using the Full Analysis Set. Missing values at Week 16 were imputed using the last-observed-carried-forward (LOCF) method.

Time frame: 16 weeks

Population: Full Analysis Set (all randomized subjects)

Arm	Measure	Value (LEAST_SQUARES_MEAN)	Dispersion
Main Study EFX 28 mg	Cohort C: Change From Baseline in Liver Stiffness as Evaluated by FibroScan at Week 16	-37.6 KPa	Standard Error 8.6
Main Study EFX 50 mg	Cohort C: Change From Baseline in Liver Stiffness as Evaluated by FibroScan at Week 16	12.1 KPa	Standard Error 12

Secondary

Cohort C: Change From Baseline in Non-invasive Biomarkers Including Liver Fibrosis by ELF Test Score at Week 12 and 16.

Cohort C. The enhanced liver fibrosis (ELF) score describes the severity of liver fibrosis where a score of \<7.7 indicates no or mild fibrosis, a score of ≥7.7 to \<9.8 indicates moderate fibrosis, and a score of ≥9.8 indicates severe fibrosis. A change from baseline with a negative value indicates a decrease in severity of liver fibrosis. An ANCOVA model with treatment group as a factor and baseline liver stiffness as evaluated by FibroScan® as a covariate using the Full Analysis Set was used. Missing values were imputed using the last-observed-carried-forward (LOCF) method.

Time frame: 12 and 16 weeks

Population: Full Analysis Set (all randomized subjects)

Arm	Measure	Group	Value (LEAST_SQUARES_MEAN)	Dispersion
Main Study EFX 28 mg	Cohort C: Change From Baseline in Non-invasive Biomarkers Including Liver Fibrosis by ELF Test Score at Week 12 and 16.	Change from Baseline to Week 12	-0.3 Score on a scale	Standard Error 0.2
Main Study EFX 28 mg	Cohort C: Change From Baseline in Non-invasive Biomarkers Including Liver Fibrosis by ELF Test Score at Week 12 and 16.	Change from Baseline to Week 16	-0.4 Score on a scale	Standard Error 0.1
Main Study EFX 50 mg	Cohort C: Change From Baseline in Non-invasive Biomarkers Including Liver Fibrosis by ELF Test Score at Week 12 and 16.	Change from Baseline to Week 12	0.3 Score on a scale	Standard Error 0.2
Main Study EFX 50 mg	Cohort C: Change From Baseline in Non-invasive Biomarkers Including Liver Fibrosis by ELF Test Score at Week 12 and 16.	Change from Baseline to Week 16	0.3 Score on a scale	Standard Error 0.2

Secondary

Cohort C: Change From Baseline in Non-invasive Biomarkers Including Pro-C3 at Week 12, 16, and 20.

Cohort C. ANCOVA model with treatment group as a factor and baseline liver stiffness as evaluated by FibroScan® as a covariate using the Full Analysis Set. Missing values were imputed using the last-observed-carried-forward (LOCF) method.

Time frame: 12, 16, and 20 weeks

Population: Full Analysis Set (all randomized subjects)

Arm	Measure	Group	Value (LEAST_SQUARES_MEAN)	Dispersion
Main Study EFX 28 mg	Cohort C: Change From Baseline in Non-invasive Biomarkers Including Pro-C3 at Week 12, 16, and 20.	Change from Baseline to Week 12	-8.4 ug/L	Standard Error 1.1
Main Study EFX 28 mg	Cohort C: Change From Baseline in Non-invasive Biomarkers Including Pro-C3 at Week 12, 16, and 20.	Change from Baseline to Week 16	-9.0 ug/L	Standard Error 1.2
Main Study EFX 28 mg	Cohort C: Change From Baseline in Non-invasive Biomarkers Including Pro-C3 at Week 12, 16, and 20.	Change from Baseline to Week 20	-5.2 ug/L	Standard Error 1.2
Main Study EFX 50 mg	Cohort C: Change From Baseline in Non-invasive Biomarkers Including Pro-C3 at Week 12, 16, and 20.	Change from Baseline to Week 12	-2.8 ug/L	Standard Error 1.5
Main Study EFX 50 mg	Cohort C: Change From Baseline in Non-invasive Biomarkers Including Pro-C3 at Week 12, 16, and 20.	Change from Baseline to Week 16	-3.4 ug/L	Standard Error 1.7
Main Study EFX 50 mg	Cohort C: Change From Baseline in Non-invasive Biomarkers Including Pro-C3 at Week 12, 16, and 20.	Change from Baseline to Week 20	-4.9 ug/L	Standard Error 1.7

Secondary

Main: Absolute Change From Baseline in Hepatic Fat Fraction Assessed by MRI-PDFF at Week 22-24.

Main study. Included subjects with ≥30% relative fat reduction on MRI-PDFF at Week 12 that were required to return between Weeks 22 - 24. ANCOVA model with treatment group and F1 fibrosis score (F1 vs F2-3) as factors and baseline hepatic fat fraction as a covariate were performed.

Time frame: 22-24 weeks

Population: Subjects from Full Analysis Set with 30% or greater reduction in hepatic fat fraction at Week 12, who returned for MRI-PDFF

Arm	Measure	Value (LEAST_SQUARES_MEAN)	Dispersion
Main Study EFX 28 mg	Main: Absolute Change From Baseline in Hepatic Fat Fraction Assessed by MRI-PDFF at Week 22-24.	-9.50 Absolute percentage of hepatic fat	Standard Error 1.49
Main Study EFX 50 mg	Main: Absolute Change From Baseline in Hepatic Fat Fraction Assessed by MRI-PDFF at Week 22-24.	-8.99 Absolute percentage of hepatic fat	Standard Error 1.49
Main Study EFX 70 mg	Main: Absolute Change From Baseline in Hepatic Fat Fraction Assessed by MRI-PDFF at Week 22-24.	-12.72 Absolute percentage of hepatic fat	Standard Error 1.5
Main Study Placebo	Main: Absolute Change From Baseline in Hepatic Fat Fraction Assessed by MRI-PDFF at Week 22-24.	-14.45 Absolute percentage of hepatic fat	Standard Error 4.08

Secondary

Main: Change From Baseline in ALT at Week 12, 16, and 20.

ANCOVA model with treatment group, baseline hepatic fat fraction (\<15% vs ≥15%), and F1 fibrosis score (F1 vs F2-3) as factors and baseline value as a covariate.

Time frame: 12, 16, and 20 weeks

Population: Full Analysis Set (all randomized subjects)

Arm	Measure	Group	Value (LEAST_SQUARES_MEAN)	Dispersion
Main Study EFX 28 mg	Main: Change From Baseline in ALT at Week 12, 16, and 20.	Change from Baseline to Week 12	-24.46 U/L	Standard Error 3.76
Main Study EFX 28 mg	Main: Change From Baseline in ALT at Week 12, 16, and 20.	Change from Baseline to Week 20	-17.57 U/L	Standard Error 4.42
Main Study EFX 28 mg	Main: Change From Baseline in ALT at Week 12, 16, and 20.	Change from Baseline to Week 16	-23.46 U/L	Standard Error 4.87
Main Study EFX 50 mg	Main: Change From Baseline in ALT at Week 12, 16, and 20.	Change from Baseline to Week 12	-30.47 U/L	Standard Error 3.59
Main Study EFX 50 mg	Main: Change From Baseline in ALT at Week 12, 16, and 20.	Change from Baseline to Week 20	-21.20 U/L	Standard Error 4.17
Main Study EFX 50 mg	Main: Change From Baseline in ALT at Week 12, 16, and 20.	Change from Baseline to Week 16	-31.99 U/L	Standard Error 4.59
Main Study EFX 70 mg	Main: Change From Baseline in ALT at Week 12, 16, and 20.	Change from Baseline to Week 16	-32.52 U/L	Standard Error 4.65
Main Study EFX 70 mg	Main: Change From Baseline in ALT at Week 12, 16, and 20.	Change from Baseline to Week 12	-32.29 U/L	Standard Error 3.59
Main Study EFX 70 mg	Main: Change From Baseline in ALT at Week 12, 16, and 20.	Change from Baseline to Week 20	-28.70 U/L	Standard Error 4.29
Main Study Placebo	Main: Change From Baseline in ALT at Week 12, 16, and 20.	Change from Baseline to Week 12	-5.94 U/L	Standard Error 3.38
Main Study Placebo	Main: Change From Baseline in ALT at Week 12, 16, and 20.	Change from Baseline to Week 20	-4.29 U/L	Standard Error 3.96
Main Study Placebo	Main: Change From Baseline in ALT at Week 12, 16, and 20.	Change from Baseline to Week 16	-3.17 U/L	Standard Error 4.42

Secondary

Main: Percent Change From Baseline in Hepatic Fat Fraction Measured by MRI-PDFF at Week 12.

Main study. ANCOVA multiple imputation with treatment group and F1 fibrosis score (F1 vs F2-3) as factors and baseline hepatic fat fraction measured by MRI-PDFF as a covariate.

Time frame: 12 weeks

Population: Full Analysis Set (all randomized subjects)

Arm	Measure	Value (LEAST_SQUARES_MEAN)	Dispersion
Main Study EFX 28 mg	Main: Percent Change From Baseline in Hepatic Fat Fraction Measured by MRI-PDFF at Week 12.	-63.21 Percent change from baseline	Standard Error 5.01
Main Study EFX 50 mg	Main: Percent Change From Baseline in Hepatic Fat Fraction Measured by MRI-PDFF at Week 12.	-70.92 Percent change from baseline	Standard Error 4.97
Main Study EFX 70 mg	Main: Percent Change From Baseline in Hepatic Fat Fraction Measured by MRI-PDFF at Week 12.	-72.26 Percent change from baseline	Standard Error 5.04
Main Study Placebo	Main: Percent Change From Baseline in Hepatic Fat Fraction Measured by MRI-PDFF at Week 12.	-0.29 Percent change from baseline	Standard Error 4.55

Secondary

Main: Percent Change From Baseline in Hepatic Fat Fraction Measured by MRI-PDFF at Week 22-24.

Main study. ANCOVA multiple imputation with treatment group and F1 fibrosis score (F1 vs F2-3) as factors and baseline hepatic fat fraction measured by MRI-PDFF as a covariate.

Time frame: 22-24 weeks

Population: Subjects from Full Analysis Set with 30% or greater reduction in hepatic fat fraction at Week 12, who returned for MRI-PDFF

Arm	Measure	Value (LEAST_SQUARES_MEAN)	Dispersion
Main Study EFX 28 mg	Main: Percent Change From Baseline in Hepatic Fat Fraction Measured by MRI-PDFF at Week 22-24.	-49.80 Percent change from baseline	Standard Error 7.64
Main Study EFX 50 mg	Main: Percent Change From Baseline in Hepatic Fat Fraction Measured by MRI-PDFF at Week 22-24.	-43.37 Percent change from baseline	Standard Error 7.52
Main Study EFX 70 mg	Main: Percent Change From Baseline in Hepatic Fat Fraction Measured by MRI-PDFF at Week 22-24.	-63.70 Percent change from baseline	Standard Error 7.62
Main Study Placebo	Main: Percent Change From Baseline in Hepatic Fat Fraction Measured by MRI-PDFF at Week 22-24.	-69.87 Percent change from baseline	Standard Error 20.78

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.

Main study: Responders were defined for subjects with ≥ 30% relative fat reduction on MRI-PDFF at Week 12 and required to return between Weeks 22 - 24. Fisher's exact test was used for the analysis using the Full Analysis Set with missing values imputed as non-responders and repeated on Liver Biopsy Evaluable Analysis Set without imputation.

Time frame: 22-24 weeks

Population: Liver Biopsy Evaluable Analysis Set (subjects from Full Analysis Set with Baseline and Week 22-24 liver biopsy results)

Arm	Measure	Value (COUNT_OF_PARTICIPANTS)
Main Study EFX 28 mg	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 Participants
Main Study EFX 50 mg	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 Participants
Main Study EFX 70 mg	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.	11 Participants
Main Study Placebo	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.	1 Participants

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.

Main Study. The analyses included the treatment group and F1 fibrosis score (F1 vs F2-3) as factors and baseline hepatic fat fraction measured by MRI-PDFF as a covariate.

Time frame: 12 weeks

Population: Full Analysis Set (all randomized subjects)

Arm	Measure	Value (COUNT_OF_PARTICIPANTS)
Main Study EFX 28 mg	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 Participants
Main Study EFX 50 mg	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.	17 Participants
Main Study EFX 70 mg	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.	15 Participants
Main Study Placebo	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.	2 Participants

Source: ClinicalTrials.gov · Data processed: Feb 22, 2026

A Study of Efruxifermin in Subjects With Histologically Confirmed Nonalcoholic Steatohepatitis (NASH)

Conditions

Brief summary

Related papers

Interventions

Sponsors

Study design

Eligibility

Inclusion criteria

Exclusion criteria

Design outcomes

Primary

Secondary

Countries

Participant flow

Participants by arm

Withdrawals & dropouts

Baseline characteristics

Adverse events

Outcome results

Main: Absolute Change From Baseline in Hepatic Fat Fraction Assessed by MRI-PDFF at Week 12.

Cohort C: Change From Baseline in Liver Stiffness as Evaluated by FibroScan at Week 16

Cohort C: Change From Baseline in Non-invasive Biomarkers Including Liver Fibrosis by ELF Test Score at Week 12 and 16.

Cohort C: Change From Baseline in Non-invasive Biomarkers Including Pro-C3 at Week 12, 16, and 20.

Main: Absolute Change From Baseline in Hepatic Fat Fraction Assessed by MRI-PDFF at Week 22-24.

Main: Change From Baseline in ALT at Week 12, 16, and 20.

Main: Percent Change From Baseline in Hepatic Fat Fraction Measured by MRI-PDFF at Week 12.

Main: Percent Change From Baseline in Hepatic Fat Fraction Measured by MRI-PDFF at Week 22-24.

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.

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.