Safety, Tolerability, Pharmacokinetics and Efficacy of EYP001a in Patients With Nonalcoholic Steatohepatitis (NASH)
- Registration Number
- NCT03812029
- Lead Sponsor
- Enyo Pharma
- Brief Summary
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
- Detailed 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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 120
- 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
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Vonafexor 100 mg QD Vonafexor Oral dose once daily for 12 weeks (84 days) Vonafexor 100 mg BID Vonafexor Oral dose twice daily for 12 weeks (84 days) Vonafexor 200 mg QD Vonafexor Oral dose once daily for 12 weeks (84 days) Vonafexor 400 mg QD Vonafexor Oral dose once daily for 12 weeks (84 days) Placebo Placebo Oral dose twice daily for 12 weeks (84 days)
- Primary Outcome Measures
Name Time Method Analysis of Absolute Change From Baseline in Percentage of Fat in the Liver as Assessed by Magnetic Resonance Imaging - Proton Density Fat Fraction (MRI-PDFF) 12 weeks The liver fat percentage was assessed by MRI-PDFF, which is an established method that enables the quantification of fat content in the liver; the value of liver fat is expressed in percentage and ranges from 0 to 100% with higher values representing higher liver fat level.
- Secondary Outcome Measures
Name Time Method Analysis of Change From Baseline in Body Weight 12 weeks Analysis of Change From Baseline in Glomerular Filtration rate_Part B 12 weeks Analysis of Change From Baseline in Corrected T1 (CT1) 12 weeks Analysis of Percent Change (Relative) From Baseline in Percentage of Fat in the Liver as Assessed by Magnetic Resonance Imaging - Proton Density Fat Fraction (MRI-PDFF) 12 weeks The liver fat percentage was assessed by MRI-PDFF, which is an established method that enables the quantification of fat content in the liver; the value of liver fat is expressed in percentage and ranges from 0 to 100% with higher values representing higher liver fat level.
Analysis of Change From Baseline in Gamma Glutamyltranspeptidase (GT) 12 weeks Analysis of Change From Baseline in Waist Circumference 12 weeks Analysis of Change From Baseline in Glomerular Filtration rate_Part A 12 weeks For Part A, as per ICH, analysis were performed as defined in the SAP: data from the 3 vonafexor treatment groups were pooled and compared with the placebo group.
Analysis of Change From Baseline in Alanine Aminotransferase (ALT) 12 weeks Analysis of Change From Baseline in Waist to Hip ratio_Part B 12 weeks
Trial Locations
- Locations (42)
ENYO PHARMA Investigative site 0413
🇺🇸Baton Rouge, Louisiana, United States
ENYO PHARMA Investigative site 0422
🇺🇸Baltimore, Maryland, United States
ENYO PHARMA Investigative site 0103
🇧🇪Gent, Belgium
ENYO PHARMA Investigative site 0407
🇺🇸Snellville, Georgia, United States
ENYO PHARMA Investigative site 0423
🇺🇸Savannah, Georgia, United States
ENYO PHARMA Investigative site 0302
🇬🇧Cambridge, United Kingdom
ENYO PHARMA Investigative site 0205
🇫🇷Villejuif, France
ENYO PHARMA Investigative site 0418
🇺🇸Lakewood Ranch, Florida, United States
ENYO PHARMA Investigative site 0404
🇺🇸Marrero, Louisiana, United States
ENYO PHARMA Investigative site 0421
🇺🇸Rapid City, South Dakota, United States
ENYO PHARMA Investigative site 0304
🇬🇧Belfast, United Kingdom
ENYO PHARMA Investigative site 0206
🇫🇷Paris, France
ENYO PHARMA Investigative site 0101
🇧🇪Edegem, Belgium
ENYO PHARMA Investigative site 0429
🇵🇷San Juan, Puerto Rico
ENYO PHARMA Investigative site 0202
🇫🇷Pessac, France
ENYO PHARMA Investigative site 0305
🇬🇧London, United Kingdom
ENYO PHARMA Investigative site 0301
🇬🇧Nottingham, United Kingdom
ENYO PHARMA Investigative site 0207
🇫🇷Toulouse, France
ENYO PHARMA Investigative site 0411
🇺🇸Columbus, Ohio, United States
ENYO PHARMA Investigative site 0409
🇺🇸Indianapolis, Indiana, United States
ENYO PHARMA Investigative site 0105
🇧🇪Brussels, Belgium
ENYO PHARMA Investigative site 0402
🇺🇸Ocoee, Florida, United States
ENYO PHARMA Investigative site 0424
🇺🇸North Little Rock, Arkansas, United States
ENYO PHARMA Investigative site 0403
🇺🇸Athens, Georgia, United States
ENYO PHARMA Investigative site 0419
🇺🇸Port Orange, Florida, United States
ENYO PHARMA Investigative site 0203
🇫🇷Limoges, France
ENYO PHARMA Investigative site 0412
🇺🇸Jackson, Mississippi, United States
ENYO PHARMA Investigative site 0405
🇺🇸Arlington, Texas, United States
ENYO PHARMA Investigative site 0201
🇫🇷Angers, France
ENYO PHARMA Investigative site 0417
🇺🇸Edinburg, Texas, United States
ENYO PHARMA Investigative site 0104
🇧🇪Gent, Belgium
ENYO PHARMA Investigative site
🇫🇷Créteil, France
ENYO PHARMA Investigative site 0204
🇫🇷Lyon, France
ENYO PHARMA Investigative site 0303
🇬🇧London, United Kingdom
ENYO PHARMA Investigative site 0420
🇺🇸Orlando, Florida, United States
ENYO PHARMA Investigative site 0414
🇺🇸Kansas City, Missouri, United States
ENYO PHARMA Investigative site 0406
🇺🇸Durham, North Carolina, United States
ENYO PHARMA Investigative site 0416
🇺🇸Austin, Texas, United States
ENYO PHARMA Investigative site 0410
🇺🇸San Antonio, Texas, United States
ENYO PHARMA Investigative site 0401
🇺🇸Charleston, South Carolina, United States
ENYO PHARMA Investigative site 0408
🇺🇸Charleston, South Carolina, United States
ENYO PHARMA Investigative site 0415
🇺🇸San Antonio, Texas, United States