Safety, Tolerability, and Efficacy of Cilofexor in Non-Cirrhotic Adults With Primary Sclerosing Cholangitis

• Register: ClinicalTrials.gov
• Last refreshed on: 7 August 2023
• Main ID: NCT03890120
• Date of registration: 25/03/2019
• Prospective Registration: Yes
• Primary sponsor: Gilead Sciences
• Public title: Safety, Tolerability, and Efficacy of Cilofexor in Non-Cirrhotic Adults With Primary Sclerosing Cholangitis PRIMIS
• Scientific title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Safety, Tolerability, and Efficacy of Cilofexor in Non-Cirrhotic Subjects With Primary Sclerosing Cholangitis
• Date of first enrolment: March 27, 2019
• Target sample size: 419
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/ct2/show/NCT03890120
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Double (Participant, Investigator).
• Phase: Phase 3

Countries of recruitment

Australia, Austria, Belgium, Canada, Denmark, Finland, France, Germany, Israel, Italy, Japan, New Zealand, Spain, Switzerland, United Kingdom, United States

Contacts

• Name: Gilead Study Director
• Affiliation: Gilead Sciences

Key inclusion & exclusion criteria

Key Inclusion Criteria:

- 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:

- Cirrhosis

- 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 severe inflammatory bowel disease (IBD) (including ulcerative
colitis, Crohn's disease, and indeterminate colitis)

Note: Other protocol defined Inclusion/Exclusion criteria may apply



Age minimum: 18 Years
Age maximum: 75 Years
Gender: All

Health Condition(s) or Problem(s) studied

Primary Sclerosing Cholangitis

Intervention(s)

Drug: Placebo

Drug: Cilofexor

Primary Outcome(s)

Proportion of Participants With Progression of Liver Fibrosis at Blinded Phase Week 96 [Time Frame: Blinded Phase Week 96]

Secondary Outcome(s)

Change From Baseline in Primary Sclerosing Cholangitis (PSC) Symptoms - Module 1 based on disease-specific Patient Reported Outcome (PSC-PRO) at Blinded Phase Week 96 [Time Frame: Baseline; Blinded Phase Week 96]

Change From Baseline in Serum Concentrations of Bile Acids at Blinded Phase Week 96 [Time Frame: Baseline; Blinded Phase Week 96]

Change From Baseline in Serum Concentrations of Alanine Aminotransferase (ALT) at Blinded Phase Week 96 [Time Frame: Baseline; Blinded Phase Week 96]

Change From Baseline in Enhanced Liver Fibrosis (ELF™ ) Test Score at Blinded Phase Week 96 [Time Frame: Baseline; Blinded Phase Week 96]

Composite Endpoint Measuring the Proportion of Participants With = 25% Relative Reduction in Serum ALP Concentration From Baseline and No Worsening of Fibrosis According to the Ludwig Classification at Blinded Phase Week 96 [Time Frame: Baseline; Blinded Phase Week 96]

Percentage of Participants Experiencing Serious Adverse Events (SAE [Time Frame: Up to 4 years]

Change From Baseline in Liver Stiffness by FibroScan® at Blinded Phase Week 96 [Time Frame: Baseline; Blinded Phase Week 96]

Change From Baseline in Serum Concentrations of Alkaline Phosphatase (ALP) at Blinded Phase Week 96 [Time Frame: Baseline; Blinded Phase Week 96]

Proportion of Participants With Fibrosis Improvement According to the Ludwig Classification at Blinded Phase Week 96 [Time Frame: Blinded Phase Week 96]

Percentage of Participants Experiencing Adverse Events (AEs) [Time Frame: Up to 4 years]

Secondary ID(s)

2019-000204-14

GS-US-428-4194

jRCT2080224728

Source(s) of Monetary Support

Please refer to primary and secondary sponsors