Texas Clinical Research Institute | Arlington, TX
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About
The participants of this study will have confirmed Primary Biliary Cholangitis (PBC) and cirrhosis (scarring of the liver).
PBC is a slowly progressive disease, characterised by damage to the bile ducts in the liver, leading to a build-up of bile acids which causes further damage.
The liver damage in PBC may lead to cirrhosis. PBC may also be associated with multiple symptoms. Many patients with PBC may require liver transplant or may die if the disease progresses and a liver transplant is not done.
This study will compare a daily dose of elafibranor (the study drug) to a daily dose of placebo (a dummy treatment) and will last up to 3.5 years for each participant.
The main aim of this study is to determine if elafibranor is better than placebo in preventing clinical outcome events showing disease worsening (including progression of disease leading to liver transplant or death).
This study will also study the safety of long-term treatment with elafibranor, as well as the impact on symptoms such as itching and tiredness.
Enrollment
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Inclusion and exclusion criteria
Inclusion Criteria :
Exclusion Criteria :
History or presence of other concomitant liver disease including but not limited to:
History or presence of clinically significant hepatic decompensation, including:
Known history of human immunodeficiency virus (HIV) infection or having a positive confirmatory test for HIV type 1 or 2.
Medical conditions that may cause non-hepatic increases in ALP (e.g. Paget's disease).
Evidence of any other unstable or untreated clinically significant immunological, endocrine, hematologic, gastrointestinal, neurological, or psychiatric disease as evaluated by the investigator; other clinically significant conditions that are not well controlled.
Non-hepatic medical conditions that may diminish life expectancy to <2 years, including known cancers.
History of hepatocellular carcinoma.
Alpha-fetoprotein (AFP) >20 ng/mL with 4-phase liver computerised tomography (CT) or magnetic resonance imaging (MRI) imaging suggesting presence of hepatocellular carcinoma.
Known malignancy or history of malignancy within the last 5 years, with the exception of local, successfully treated basal cell carcinoma or in-situ carcinoma of the uterine cervix.
Administration of the following medications is prohibited during the study, and prior to the study as per the timelines specified below: • i) 3 months prior to screening period: fibrates, seladelpar, glitazones, obeticholic acid, azathioprine, cyclosporine, methotrexate, mycophenolate, pentoxifylline, budesonide and other systemic corticosteroids (parenteral and oral chronic administration only); potentially hepatotoxic drugs (including α-methyl-dopa, sodium valproic acid, isoniazid or nitrofurantoin).
Participants who are currently participating in, plan to participate in, or have participated in an investigational drug study or medical device study containing active substance within 30 days or 5 half-lives, whichever is longer, prior to the screening period.
i) If the previous study was for an experimental therapy being studied for potential benefit in PBC, and the potential therapeutic agent was proven to have no beneficial effect in PBC and there are no safety concerns, the participant may enrol after 30 days or 5 half-lives from the last dose of the therapeutic agent, whichever is longer.ii) For therapeutic agents being studied for potential benefit in PBC for which it is still unclear if there may be a potential benefit, participants may enrol after 6 months from the last dose of the therapeutic agent.
Electrocardiogram (ECG) with QT interval corrected by Fridericia's formula (QTcF) >450 msec in males or QTcF >470 msec in females for participants without bundle branch block. For participants with bundle branch block or other intraventricular conduction delay, a longer QTcF >480 msec would be exclusionary.
Total bilirubin (TB) >5x ULN
Alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) >5x ULN at SV1
Creatinine phosphokinase (CPK) >2x ULN.
Platelet count <50,000/μL
International normalised ratio (INR) >1.8 in the absence of anticoagulant therapy.
Estimated glomerular filtration rate (eGFR) <45 mL/min/1.73m2 per the Modification of Diet in Renal Disease (MDRD)-6 Study formula at SV1.
Significant renal disease, including nephritic syndrome, chronic kidney disease (CKD) (defined as participants with evidence of significantly impaired kidney function or underlying kidney injury).
For female participants: known current pregnancy, or has a positive serum pregnancy test, or is breastfeeding.
Regular alcohol intake in excess of the recommended limit of 1 standard drink per day for men or women.
History of alcohol abuse, or other substance abuse within 1 year prior to SV1.
Known hypersensitivity to elafibranor or to any of the excipients of the investigational product(s).
Mental instability or incompetence, such that the validity of informed consent or ability to be compliant with the study is uncertain.
Any other condition that, in the opinion of the investigator, would interfere with study participation or completion, or would put the participant at risk, including a potential participant assessed as being at high risk of noncompliance with the study.
Alkaline phosphatase (ALP) ≥10x ULN.
Albumin <2.8 g/dL due to impaired hepatic function.
Primary purpose
Allocation
Interventional model
Masking
276 participants in 2 patient groups, including a placebo group
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Ipsen Clinical Study Enquiries
Data sourced from clinicaltrials.gov
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