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

Akero Therapeutics

Status and phase: Completed; Phase 2

Conditions: NASH - Nonalcoholic Steatohepatitis

Treatments: Drug: EFX; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT03976401

AK-US-001-0101

Details and patient eligibility

About

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.

Enrollment: 110 patients

Sex: All

Ages: 18 to 80 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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).

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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110 participants in 6 patient groups, including a placebo group

EFX Dose 1

Experimental group

Description:

Main Study

Treatment:

Drug: EFX

EFX Dose 2

Experimental group

Description:

Main Study

Treatment:

Drug: EFX

EFX Dose 3

Experimental group

Description:

Main Study

Treatment:

Drug: EFX

Placebo

Placebo Comparator group

Description:

Main Study

Treatment:

Drug: Placebo

EFX Dose (Cohort C)

Experimental group

Treatment:

Drug: EFX

Placebo (Cohort C)

Placebo Comparator group

Treatment:

Drug: Placebo