Safety, Tolerability, Pharmacokinetics and Efficacy of EYP001a in Patients With Nonalcoholic Steatohepatitis (NASH)

Enyo Pharma

Status and phase: Completed; Phase 2

Conditions: Non-alcoholic Steatohepatitis

Treatments: Drug: Vonafexor; Other: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT03812029

EYP001-202

2018-003119-22 (EudraCT Number)

Details and patient eligibility

About

The purpose of this study is to assess the effects of EYP001a (Vonafexor) with respect to safety, tolerability, pharmacokinetics and on markers of liver inflammation in patients with NASH

Full description

This is a 2-part, randomized, double-blind, multicenter, placebo-controlled study to evaluate the safety and efficacy of Vonafexor in patients with NASH who likely have stage F2 to F3 fibrosis at approximately 50 global clinical sites. Overall, approximately 114 eligible patients will be enrolled: 24 patients in Part A (Safety Run-in Cohort), followed by 90 patients in Part B.

In Part A, 24 patients will be randomized on Day 1 to 1 of 4 parallel treatment groups: 100 mg Vonafexor twice daily (BID), 200 mg Vonafexor once daily (QD), 400 mg Vonafexor QD, or placebo BID. In Part B, 90 patients will be randomized on Day 1 to 1 of 3 parallel treatment groups: 100 mg Vonafexor QD, 200 mg Vonafexor QD, or placebo QD.

Enrollment: 120 patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Written informed consent
• Suspected diagnosis of NASH, evidenced by elevated alanine aminotransferase (ALT), liver stiffness compatible with F2 or F3 fibrosis and Liver Fat Content (LFC) ≥10% as measured by MRI
• Women of childbearing potential and male patients with female partners must agree to use a dual method of contraception

Exclusion criteria

• Evidence of worsening liver injury
• Previous diagnosis of other forms of non-NASH liver disease
• Use of Vitamin E, glitazones, glucagon-like Peptide-1 receptor agonists, ursodeoxycholic acid, or obeticholic acid within 90 days prior to screening
• History of cirrhosis or liver decompensation
• Known history of alcohol abuse or daily heavy alcohol consumption
• Pregnant or breastfeeding women
• Type 1 diabetes mellitus and uncontrolled type 2 diabetes mellitus
• Patients with contraindications to MRI imaging

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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120 participants in 5 patient groups, including a placebo group

Vonafexor 100 mg BID

Experimental group

Description:

Oral dose twice daily for 12 weeks (84 days)

Treatment:

Drug: Vonafexor

Vonafexor 200 mg QD

Experimental group

Description:

Oral dose once daily for 12 weeks (84 days)

Treatment:

Drug: Vonafexor

Vonafexor 400 mg QD

Experimental group

Description:

Oral dose once daily for 12 weeks (84 days)

Treatment:

Drug: Vonafexor

Placebo

Placebo Comparator group

Description:

Oral dose twice daily for 12 weeks (84 days)

Treatment:

Other: Placebo

Vonafexor 100 mg QD

Experimental group

Description:

Oral dose once daily for 12 weeks (84 days)

Treatment:

Drug: Vonafexor