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

• Register: ClinicalTrials.gov
• Last refreshed on: 11 October 2021
• Main ID: NCT04060147
• Date of registration: 15/08/2019
• Prospective Registration: Yes
• Primary sponsor: Gilead Sciences
• Public title: Safety and Tolerability of Cilofexor in Participants With Primary Sclerosing Cholangitis (PSC) and Compensated Cirrhosis
• Scientific title: A Proof-of-Concept, Open-Label Study Evaluating the Safety and Tolerability of Cilofexor in Subjects With Primary Sclerosing Cholangitis (PSC) and Compensated Cirrhosis
• Date of first enrolment: October 17, 2019
• Target sample size: 11
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT04060147
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: Gilead Study Director
• Affiliation: Gilead Sciences

Key inclusion & exclusion 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.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Primary Sclerosing Cholangitis

Compensated Cirrhosis

Intervention(s)

Drug: CILO

Primary Outcome(s)

Percentage of Participants Experiencing Laboratory Abnormalities [Time Frame: First dose date up to Week 12 plus 30 days]

Percentage of Participants Experiencing Treatment-Emergent Adverse Events [Time Frame: First dose date up to Week 12 plus 30 days]

Secondary ID(s)

GS-US-428-5443

Source(s) of Monetary Support

Please refer to primary and secondary sponsors