A Phase III, open-label, single arm study to investigate the efficacy and safety of elafibranor
80 mg in adult Japanese participants with Primary Biliary Cholangitis (PBC).
A study of elafibranor in adult Japanese participants with Primary Biliary Cholangitis (PBC)
Allan Richard
Ipsen Bioscience, Inc
4-1-3 Kyutaro-cho, Chuuou-ku, Osaka-city, Osaka, 541-0056, Japan
+81-6-4560-2001
Japan-Chiken@iconplc.com
Clinical trial contact
ICON Clinical Research GK
4-1-3 Kyutaro-cho, Chuuou-ku, Osaka-city, Osaka, 541-0056, Japan
+81-6-4560-2001
Japan-Chiken@iconplc.com
Recruiting
Nov. 22, 2024
18
Interventional
single arm study
open(masking not used)
uncontrolled control
single assignment
treatment purpose
-Must have provided written informed consent and agree to comply with the study protocol.
-Japanese male or female participants aged 18 to 75 years inclusive at Screening Visit 1 (SV1)
-PBC diagnosis as described in the study protocol
-ALP >=1.67xULN (mean value based on samples collected at SV1 and SV2).
-TB <=2xULN at SV1 and SV2.
-Must have at least 4 available values for PBC Worst Itch Numeric Rating Scale (NRS) during each of the 7-day intervals in the 14 days prior to visit (V)1, for a total of at least 8 values for PBC Worst Itch NRS in the last 14 days prior to V1.
-Participants taking UDCA for at least 12 months (stable dose >=3 months) prior to screening, or unable to tolerate UDCA treatment (no UDCA for >=3 months) prior to screening (per country standard-of-care dosing).
-If on colchicine, must be on a stable dose for >=3 months prior to screening.
-Medications for management of pruritus (for example, cholestyramine, rifampicin, naltrexone, sertraline or nalfurafine hydrochloride) must be on a stable dose for >=3 months prior to screening.
-Participants taking statins or ezetimibe must be on a stable dose for >=2 months prior to screening.
-Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
-History or presence of other concomitant liver disease
-Participants with known cirrhosis who have a Child-Pugh B or C classification.
-Participants with cirrhosis with Child-Pugh A classification are allowed.
-History or presence of clinically significant hepatic decompensation
-Medical conditions that may cause non-hepatic increases in ALP (for example, Paget's disease) or which may diminsh life expectancy to <2 years, including known cancers.
-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.
-Participant has a positive test for human immunodeficiency virus (HIV) Type 1 or 2 at screening, or participant is known to have tested positive for HIV.
-Evidence of any other unstable or untreated clinically significant immunological, endocrine, haematologic, gastrointestinal, neurological, or psychiatric disease as evaluated by the investigator; other clinically significant medical conditions that are not
well controlled.
-History of alcohol abuse, defined as consumption of more than 30 g pure alcohol per day for men, and more than 20 g pure alcohol per day for women, or other substance abuse within 1 year prior to SV1.
-For female participants: known pregnancy, or has a positive serum pregnancy test, or breastfeeding.
-Administration of the following medications are prohibited as specified below:
a. 1 month prior to screening: fibrates.
b. 2 months prior to screening: glitazones.
c. For participants with previous exposure to obeticholic acid (OCA), OCA should be discontinued 3 months prior to screening.
d. 3 months prior to screening: azathioprine, cyclosporine (systemic), methotrexate, mycophenolate, pentoxifylline, budesonide and other systemic corticosteroids (parenteral and oral chronic administration only); potentially hepatotoxic drugs (including alpha-methyldopa, sodium valproate/valproic acid isoniazid, or nitrofurantoin).
e. 12 months prior to screening: antibodies or immunotherapy directed against interleukins (ILs) or other cytokines or chemokines.
-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 screening; for participants with previous exposure to seladelpar, seladelpar should be discontinued 3 months prior to screening.
-Participants with previous exposure to elafibranor.
-SV1 or SV2 value ALT and/or AST >5xULN.
-For participants with aminotransferases or TB >ULN at SV1, variability (between SV1 and SV2) of aminotransferases or TB >40%.
-SV1 value albumin <3.0 g/dL.
-Severely advanced participants according to Rotterdam criteria (TB >ULN and albumin <LLN).
-SV1 international normalised ratio (INR) >1.3 due to altered hepatic function.
-SV1 creatine phosphokinase (CPK) >2xULN.
-SV1 serum creatinine >1.5 mg/dL.
-Significant renal disease, including nephritic syndrome, chronic kidney disease (defined as participants with markers of kidney failure damage or estimated glomerular filtration rate (eGFR) <60 mL/min/1.73 m2) calculated by modification of diet in renal disease study (MDRD).
-SV1 platelet count <150x103/ micro L.
-Alpha-fetoprotein (AFP) >20 ng/mL with 4-phase liver computerised tomography (CT) or magnetic resonance imaging (MRI) imaging suggesting presence of liver cancer.
-Known hypersensitivity to the investigational product or to any of the formulation excipients of the elafibranor tablet.
-Mental instability or incompetence, such that the validity of informed consent or ability to be compliant with the study is uncertain.
18age old over
75age old under
Both
Primary Biliary Cholangitis (PBC)
80 mg of Elafibranor administered orally once a day. The participant will be treated for 52 weeks during the treatment period followed by a variable treatment extension period of 2 to 5 years (the maximum treatment duration anticipated would be 6 years). The primary endpoint will be evaluated at the end of the treatment period on week 52.
Response to treatment at Week 52 defined as ALP <1.67xULN and TB <=ULN and ALP decrease >=15%
Ipsen Bioscience, Inc (ICCC: ICON Clinical Research GK)
Teine Keijinkai Hospital Institutional Review Board
