A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Safety, Tolerability, and Efficacy of Investigational Cilofexor in Non-Cirrhotic Subjects With Primary Sclerosing Cholangitis
The primary objective of this study is to evaluate whether investigational cilofexor reduces the risk of fibrosis progression among non-cirrhotic adults with primary sclerosing cholangitis (PSC).
Key Inclusion Criteria:
- Male or female 18-75 years old;
- Diagnosis of large duct PSC;
- Liver biopsy at screening that is deemed acceptable for interpretation and demonstrates stage F0 - F3 fibrosis in the opinion of the central reader;
- Individual has the following laboratory parameters at the screening visit, as determined by the central laboratory:
- Platelet count ≥ 150,000/mm^3,
- Estimated glomerular filtration rate (eGFR) ≥ 30 milliliter/minute (mL/min), as calculated by the Cockcroft-Gault equation;
- ALT ≤ 8 x upper limit of the normal range (ULN);
- Total bilirubin < 2 mg/dL, unless the individual is known to have Gilbert's syndrome or hemolytic anemia;
- International normalized ratio (INR) ≤ 1.4, unless due to therapeutic anticoagulation;
- Negative anti-mitochondrial antibody
Key Exclusion Criteria:
- Current or prior history of any of the following:
- Liver transplantation
- Cholangiocarcinoma or hepatocellular carcinoma (HCC)
- Ascending cholangitis within 30 days of screening
- Presence of a percutaneous drain or biliary stent
- Other causes of liver disease
- Current or prior history of unstable cardiovascular disease
- Current moderate to severely active inflammatory bowel disease (IBD)
Note: Other protocol defined Inclusion/Exclusion criteria may apply
For additional information, visit ClinicalTrials.gov (NCT03890120): https://clinicaltrials.gov/ct2/show/NCT03890120
The PRIMIS study will help evaluate the safety and effectiveness of investigational cilofexor in patients with PSC.
The study is divided into several phases:
- Screening Period (about 2 months): Tests will be performed to determine if you can enroll in the study and adjustments may be made to current medications.
- Study Treatment Period (about 22 months): You will be randomly assigned (like the flip of a coin) to study treatment with either the investigational drug (cilofexor) or placebo (an inactive substance). You will return to the study clinic every 1-3 months for health checks and study procedures.
Follow up period: one last visit about 4 weeks after the Study Treatment Period ends to check on your health and any potential side effects.
Disclaimer: Cilofexor is an investigational agent and its safety and efficacy have not been established.
Description of Treatment or Intervention (Mechanism of Action)
100 mg tablet administered orally once daily