Safety and Tolerability of Cilofexor in Participants With Primary Sclerosing Cholangitis (PSC) and Compensated Cirrhosis

• Status: Completed
• Trial end date: 2021-09-02
• Target enrollment: 0
• Participant gender: All

Summary

The primary objective of this study is to assess the safety and tolerability of escalating doses of cilofexor in participants with primary sclerosing cholangitis (PSC) and compensated cirrhosis.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Gilead Sciences

Criteria

Key Inclusion Criteria:

• Diagnosis PSC based on cholangiogram (magnetic resonance cholangiopancreatography [MRCP], endoscopic retrograde cholangiopancreatography [ERCP], or percutaneous transhepatic cholangiogram [PTC]) or liver biopsy

• Individuals have evidence of cirrhosis based on historical liver biopsy, abdominal imaging (MRI, CT, or Ultrasound), or a screening FibroScan®, ELF™, or FibroTest®.

• Individual has the following laboratory parameters at the Screening visit, as determined by the central laboratory:

• Estimated glomerular filtration rate (eGFR) > 60 mL/min, as calculated by the Cockcroft-Gault equation

• ALT ≤ 5 x ULN

• Total 2 mg/dL, unless the individual is known to have Gilbert's syndrome or hemolytic anemia

• INR ≤ 1.4, unless due to therapeutic anticoagulation

• Platelet count ≥ 75,000/μL. Individuals with evidence of high-risk esophageal or gastric varices in the opinion of the investigator are excluded

• Negative anti-mitochondrial antibody

Key Exclusion Criteria:

• Current or prior history of any of the following

• Decompensated liver disease, including ascites, hepatic encephalopathy (HE), or variceal hemorrhage

• Liver transplantation

• Cholangiocarcinoma or hepatocellular carcinoma (HCC).

• Model for End-stage Liver Disease (MELD) score > 12 at Screening, unless due to an alternate etiology such as therapeutic anticoagulation

• Child-Pugh (CP) score > 6 at Screening, unless due to an alternative etiology such as Gilbert's syndrome or therapeutic anticoagulation

• Current moderate to severely active inflammatory bowel disease (IBD) (including ulcerative colitis, Crohn's disease, and indeterminate colitis).

• Note: Individuals with IBD who currently have an external ostomy bag and/or proctocolectomy are not subject to this exclusion criterion and need not undergo IBD Symptom Severity Assessment.

Note: Other protocol defined Inclusion/Exclusion criteria may apply.