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    Summary
    EudraCT Number:2019-003400-12
    Sponsor's Protocol Code Number:GFT505E-218-1
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2019-10-17
    Trial results View results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    H.4 THIRD COUNTRY IN WHICH THE TRIAL WAS FIRST AUTHORISED
    Expand All   Collapse All
    A. Protocol Information
    A.2EudraCT number2019-003400-12
    A.3Full title of the trial
    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)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Elafibranor, PK and Safety in Children and Adolescents 8 to 17 Years of Age With Non Alcoholic Steatohepatitis (NASH)
    A.4.1Sponsor's protocol code numberGFT505E-218-1
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT03883607
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/237/2016
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorGENFIT
    B.1.3.4CountryFrance
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportGENFIT
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationGENFIT
    B.5.2Functional name of contact pointClinical Trials Contact Point
    B.5.3 Address:
    B.5.3.1Street Address885 Avenue Eugène Avinée
    B.5.3.2Town/ cityLoos
    B.5.3.3Post code59120
    B.5.3.4CountryFrance
    B.5.4Telephone number+33320 164 000
    B.5.6E-mailclinicaltrial@genfit.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameElafibranor
    D.3.2Product code GFT505
    D.3.4Pharmaceutical form Coated tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNELAFIBRANOR
    D.3.9.1CAS number 824932-88-9
    D.3.9.3Other descriptive nameGFT505
    D.3.9.4EV Substance CodeSUB187548
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number80
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNELAFIBRANOR
    D.3.9.1CAS number 824932-88-9
    D.3.9.3Other descriptive nameGFT505
    D.3.9.4EV Substance CodeSUB187548
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number120
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Non-Alcoholic Steato-Hepatitis (NASH)
    E.1.1.1Medical condition in easily understood language
    Non alcoholic steatotic hepatic
    E.1.1.2Therapeutic area Not possible to specify
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 22.0
    E.1.2Level PT
    E.1.2Classification code 10053219
    E.1.2Term Non-alcoholic steatohepatitis
    E.1.2System Organ Class 10019805 - Hepatobiliary disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess the pharmacokinetics of elafibranor and its active metabolite GFT1007, following once daily oral administration of two dose levels of elafibranor (80 mg and 120 mg) to children and adolescents, 8 to 17 years of age (inclusive).
    E.2.2Secondary objectives of the trial
    To assess changes in serum alanine aminotransferase (ALT), aspartate aminotransferase (AST), gamma-glutamyl transferase (GGT), and alkaline phosphatase (ALP) and other liver markers;
    To assess changes in markers of glucose homeostasis (homeostatic model assessment for insulin resistance (HOMA-IR) and fasting insulin);
    To assess changes in serum lipid parameters;
    To assess changes in body weight and Body Mass Index (BMI) z-score;
    To assess changes in waist circumference;
    To assess changes in inflammatory markers;
    To assess the change in the pediatric quality of life (PedsQLTM) score as completed by the children/adolescent and the parent/legal guardian;
    To assess the safety and tolerability profile of two dose levels of elafibranor (80 mg and 120 mg) in children and adolescents, 8 through 17 years of age;
    To explore the relationship between plasma concentrations of elafibranor (PK) and selected pharmacodynamic (PD) markers.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    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.
    E.4Principal exclusion criteria
    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 than 140 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 m^2 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:
    1. Abnormal hemoglobin (with the exception of females with a documented history of a low hemoglobin during menstruation);
    2. White blood cell count less than 3,500 cells/mm^3 of blood;
    3. Platelet count less than 150,000 cells/mm^3 of blood;
    4. Direct bilirubin greater than 0.3 mg/dL;
    5. 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;
    6. Serum albumin less than 3.5 g/dL;
    7. International normalized ratio (INR) greater than 1.4;

    Has evidence of chronic liver disease other than NASH, defined by any one of the following:
    1. Biopsy consistent with histological evidence of autoimmune hepatitis;
    2. Serum hepatitis A antibody positive;
    3. Serum hepatitis B surface antigen positive;
    4. Serum hepatitis C antibody positive;
    5. Serum hepatitis E antibody positive;
    6. History of or current positive Anti-Mitochondrial Antibody Test;
    7. Known or current Iron/total iron binding capacity ratio (transferrin saturation) greater than 45% with histological evidence of iron overload;
    8. Known or current Alpha-1-antitrypsin phenotype/genotype ZZ or SZ;
    9. 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.
    E.5 End points
    E.5.1Primary end point(s)
    Plasma concentrations of elafibranor and its active metabolite GFT1007
    E.5.1.1Timepoint(s) of evaluation of this end point
    Day 1 at pre-dose; Day 29 and 30 at pre-dose; 0.5, 1, 1.5, 2, 4, 6, 8, 24 hours post dose; Day 85 24 hours after lase dose
    E.5.2Secondary end point(s)
    Pharmacodynamic
    • Change from baseline in serum alanine aminotransferase (ALT), aspartate aminotransferase (AST), gamma-glutamyl transferase (GGT), and alkaline phosphatase (ALP) and other liver markers;
    • Change from baseline in glucose homeostasis makers (Homeostatic Model Assessment of Insulin Resistance [HOMA-IR] and fasting insulin), as a continuous measure;
    • Change from baseline in serum lipid parameters;
    • Change from baseline in body weight and BMI z-score;
    • Change from baseline in waist circumference;
    • Change from baseline in inflammatory markers;
    • Change from baseline in pediatric quality of life (PedsQL™) score.

    Safety
    • Incidence and severity of treatment emergent adverse events (TEAEs) and their relationship to study drug;
    • Incidence of clinically meaningful changes from baseline in safety laboratory parameters, physical examination, electrocardiogram (ECG) and vital signs.
    E.5.2.1Timepoint(s) of evaluation of this end point
    From Day 1 to Day 85
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy No
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Yes
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 Will this trial be conducted at a single site globally? No
    E.8.4 Will this trial be conducted at multiple sites globally? Yes
    E.8.6 Trial involving sites outside the EEA
    E.8.6.2Trial being conducted completely outside of the EEA Yes
    E.8.6.3Specify the countries outside of the EEA in which trial sites are planned
    United States
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    February 2021 (Final data collection date for primary outcome measure)
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months9
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 20
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 8
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 12
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 20
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    None
    G. Investigator Networks to be involved in the Trial
    H.4 Third Country in which the Trial was first authorised
    H.4.1Third Country in which the trial was first authorised: United States
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