Summary

for people ages 8-17 (full criteria)
at San Diego, California and other locations
study started
estimated completion
Jeffrey Schwimmer, MD

Description

Summary

The study is being conducted in order to assess the pharmacokinetics and the safety of elafibranor following once daily administration of two dose levels of elafibranor (80 mg and 120mg) during 3 months in children and adolescent population (8 to 17 years of age) with NASH.

Official Title

An Open Label, Randomized, Multicenter Study to Assess the Pharmacokinetic and Pharmacodynamic Profile and the Safety and Tolerability of Two Dose Levels of Elafibranor (80 mg and 120 mg) in Children and Adolescents, 8 to 17 Years of Age, With Nonalcoholic Steatohepatitis (NASH)

Keywords

Non Alcoholic Steatohepatitis Pediatric Pharmacokinetics Fatty Liver Non-alcoholic Fatty Liver Disease Elafibranor 80mg Elafibranor 120mg

Eligibility

You can join if…

Open to people ages 8-17

  • Are male or female between 8 and 17 years of age (inclusive) at the time of Screening Visit (when consent for study participation is given) and at the time of Randomization;
  • Diagnosis of non-alcoholic steatohepatitis (NASH) confirmed by histological evaluation (with or without fibrosis) from a liver biopsy obtained within 24 months prior to Randomization;
  • Has an alanine aminotransferase (ALT) level greater than 50 IU/L, at Screening;
  • Has a Homeostasis Model Assessment of Insulin Resistance (HOMA-IR) greater than or equal to 5, at Screening;
  • Has a Body Mass Index z-score (BMI z-score) (also referred to as BMI-for-age percentile) greater than or equal to 85th percentile for age and gender at Screening;
  • Has a Hemoglobin A1C (HbA1c) less than or equal to 8.5%. If the patient has Type 2 diabetes and is taking anti-diabetic therapy (e.g., metformin or insulin), treatment must have been started at least 3 months prior to Screening and the dose must be stable for at least 3 months prior to Screening and should remain stable through Randomization;
  • Sexually active female participants of childbearing potential must agree to utilize a highly effective method of contraception per the Clinical Trial Facilitation Group Guidelines from Screening through 30 days after the last dose of study drug (1 month after the end of treatment), and agree to monthly pregnancy testing during the study up to and including end of study.

Other inclusion criteria may apply

You CAN'T join if...

  • Has history of bariatric surgery or planned surgery during the study period;
  • Has known history of heart disease;
  • Has uncontrolled hypertension evidenced by sustained elevation in systolic blood pressure greater than140 mmHg or diastolic blood pressure greater than 90 mmHg despite treatment with antihypertensive therapy, prior to Randomization;
  • Has a known history of Type 1 diabetes;
  • Has a known history of acquired immunodeficiency syndrome or positive screening for human immunodeficiency virus antibodies at Screening Visit;
  • Has a documented weight loss of more than 5% during the 6-month period prior to Randomization;
  • Has a history of renal disease defined as an estimated glomerular filtration rate (eGFR) less than 90 mL/min/1.73 m2 using the Schwartz Bedside GFR Calculator for Children or present at Screening Visit;

  • History of, significant alcohol consumption or inability to reliably quantify alcohol intake, and/or use of illicit drugs.
  • Has clinical and/or historical evidence of cirrhosis, included by not limited to:
  • Abnormal hemoglobin (with the exception of females with a documented history of a low hemoglobin during menstruation);
  • White blood cell count less than 3,500 cells/mm3 of blood;

  • Platelet count less than150,000 cells/mm3 of blood;

  • Direct bilirubin greater than 0.3 mg/dL;
  • Total bilirubin greater than 1.3 mg/dL unless the patient has a diagnosis of Gilbert disease in which case direct bilirubin, reticulocyte count and haemoglobin must be normal;
  • Serum albumin less than 3.5 g/dL;
  • International normalized ratio (INR) greater than 1.4;
  • Has evidence of chronic liver disease other than NASH, defined by any one of the following:
  • Biopsy consistent with histological evidence of autoimmune hepatitis;
  • Serum hepatitis A antibody positive;
  • Serum hepatitis B surface antigen positive;
  • Serum hepatitis C antibody positive;
  • Serum hepatitis E antibody positive;
  • History of or current positive Anti-Mitochondrial Antibody Test;
  • Known or current Iron/total iron binding capacity ratio (transferrin saturation) greater than 45% with histological evidence of iron overload;
  • Known or current Alpha-1-antitrypsin phenotype/genotype ZZ or SZ;
  • Diagnosis of Wilson's disease;
  • Has AST and/or ALT greater than 8 fold the upper limit of normal;
  • Is pregnant, lactating or is planning to become pregnant during the study;

Other exclusion criteria may apply

Locations

  • University of California accepting new patients
    San Diego California 92103 United States
  • Columbia University accepting new patients
    New York New York 10032 United States

Lead Scientist

  • Jeffrey Schwimmer, MD
    Professor Of Clinical, Pediatrics. Authored (or co-authored) 121 research publications

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Genfit
ID
NCT03883607
Phase
Phase 2
Study Type
Interventional
Last Updated