A Study Evaluating the Safety, Tolerability, and Efficacy of Aramchol Meglumine in Primary Sclerosing Cholangitis

A Proof of Concept, Prospective, Randomized, Placebo-controlled, Double-blind, Study to Evaluate the Safety, Tolerability, and Efficacy of Aramchol Meglumine in Patients With Primary Sclerosing Cholangitis

The objectives of this study is to Evaluate the Safety, Tolerability, and Efficacy of Aramchol Meglumine in Patients with Primary Sclerosing Cholangitis

The objectives of this study are to:

• Establish the safety and tolerability of once daily (QD) Aramchol meglumine in patients with PSC
• Examine whether once daily (QD) Aramchol meglumine has any effect on serum alkaline phosphatase
• Provide a comprehensive readout of clinical efficacy following once daily (QD) Aramchol meglumine administration

Inclusion Criteria:

1. Male or female age 18 years and above (inclusive at first screening visit)
2. Established diagnosis of large duct PSC based on abnormal cholangiography as assessed by magnetic resonance cholangiopancreatography (MRCP) or Endoscopic retrograde cholangiopancreatography (ERCP)
3. Alkaline phosphatase > 150 IU/l
4. Stable inflammatory bowel disease therapy > 3months for IBD patients
5. If receiving treatment with Ursodeoxycholic acid (UDCA; ursodiol), therapy is at a dose of <20 mg/kg/day, has been stable for at least 6 months before screening
6. Ability to understand the nature of the study and to sign a written informed consent form (ICF)

Exclusion Criteria:

1. Other causes of liver disease, including secondary sclerosing cholangitis or viral, metabolic, or alcoholic liver disease, as assessed clinically
2. Active Crohn's disease (CDAI > 40) or ulcerative colitis (Mayo IBD score > 4) or active non-hemorrhoidal rectal bleeding
3. Small bowel resection > 100 cm
4. Cirrhosis (clinically evident or by biopsy)
5. Prior hepatic decompensation event
6. Recent (< 6 weeks) acute cholangitis or hospitalization for PSC or IBD
7. Bleeding diathesis or other contraindication for liver biopsy
8. Known GI or hepatobiliary malignancy
9. Prior liver transplantation
10. Prior exposure to study drug
11. Active untreated viral hepatitis or other concomitant liver disease