SHS_610031 KOWK CNOV 15130

A Multi-Part, Randomized, Double-Blind, Placebo Controlled Study to Assess the Safety, Tolerability and Efficacy of LJN452 in Patients with Primary Biliary Cirrhosis

A multi-part study to assess safety, tolerability and efficacy of LJN452 in patients with primary biliary cirrhosis.

Inclusion Criteria:

•Diagnosis of PBC as demonstrated by the presence of ≥ 2 of the following 3 diagnostic criteria:
•History of Alkaline Phosphatase elevated above upper limit of normal for at least 6 months
•Positive anti-mitochondrial antibodies (AMA) titer or if AMA negative or in low titer (<1:80) PBC specific antibodies (anti- GP210 and/or anti-SP100 and/or antibodies against the major M2 components (PDC-E2, 2-oxo-glutaric acid dehydrogenase complex)
•Previous liver biopsy consistent with PBC
•At least 1 of the following markers of disease severity: ALP ≥ 1.67× upper limit of normal (ULN) OR total bilirubin > ULN but < 2× ULN
•Subjects must weigh at least 40 kg to participate in the study, and must have a body mass index (BMI) within the range of 18 - 35 kg/m^2. BMI = Body weight (kg) / [Height (m)]^2.
•Able to communicate well with the investigator, to understand and comply with the requirements of the study.

Exclusion Criteria:

•Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception during dosing and for 5 days following end of treatment.
•History or presence of other concomitant liver diseases
•Clinically significant hepatic decompensation.
•History of any venous thromboembolism, TIA, intracranial hemorrhage, neoplasm, arteriovenous malformation, vasculitis, bleeding disorder, coagulation disorders or screening blood tests that indicate altered coagulability (e.g., platelet count, aPTT, PTT or TT tests).
•History of conditions that may cause increases in ALP (e.g., Paget's disease).
•Use of other investigational drugs at the time of enrollment, or within 5 half-lives of enrollment
Phase II
NCT02516605
All Other
Hepatic
Kris Kowdley, M.D.
Novartis
Brook Quist
  • Swedish Medical Center