SHS_610049 KOWK CGNF 17014

A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study to Evaluate the Efficacy and Safety of Elafibranor at Doses of 80 mg and 120mg After 12 Weeks of Treatment in Patients With Primary Biliary Cholangitis and Inadequate Response to Ursodeoxycholic Acid

Inclusion Criteria:

Definite or probable PBC diagnosis as demonstrated by the presence of at least 2 of the following 3 diagnostic factors:
History of elevated ALP levels for at least 6 months prior to Day 0 (randomization visit)
Positive Anti-Mitochondrial Antibodies (AMA) titers (> 1/40 on immunofluorescence or M2 positive by enzyme-linked immunosorbent assay (ELISA) or positive PBC-specific antinuclear antibodies
Liver biopsy consistent with PBC
ALP ≥ 1.67x upper limit of normal (ULN)
Taking UDCA for at least 12 months (stable dose for ≥ 6 months) prior to screening visit
Contraception: Females participating in this study must be of non-childbearing potential or must be using highly efficient contraception for the full duration of the study and for 1 month after the end of treatment.

Exclusion Criteria:

History or presence of other concomitant liver diseases
Screening CPK > ULN
Screening ALT or AST > 5 ULN
Screening total bilirubin > 2 ULN
Screening serum creatinine > 1.5 mg/dl
Significant renal disease, including nephritic syndrome, chronic kidney disease (defined as patients with markers of kidney damage or estimated glomerular filtration rate [eGFR] of less than 60 mL/min/1.73 m2).
Patients with moderate or severe hepatic impairment (defined as Child-Pugh B/C)
Platelet count <150 X 10 3/microliter
Albumin <3.5 g/dL
Presence of clinical complications of PBC or clinically significant hepatic decompensation
If female: known pregnancy, or has a positive urine pregnancy test (confirmed by a positive serum pregnancy test), or lactating
Known history of human immunodeficiency virus (HIV) infection
Medical conditions that may cause non-hepatic increases in ALP (e.g., Paget's disease)
Phase II
All Other
Kris Kowdley, M.D.
Brook Quist
  • Swedish Medical Center