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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

Study Evaluating the Safety, Tolerability and Efficacy of Cilofexor in Non-Cirrhotic Subjects with Primary Sclerosing Cholangitis

Kamiya Makoto

Gilead Sciences, K.K.

1-9-2, Marunouchi, Chiyoda-ku, Tokyo

ClinicalTrialGSJ@gilead.com

Gilead Sciences, K.K.

1-9-2, Marunouchi, Chiyoda-ku, Tokyo

+81-3-6837-0730

JPClinicalOperations@gilead.com

completed

Aug. 28, 2019

Interventional

randomized controlled study, double blind, placebo control, paralle assignment

treatment purpose

3

1) Dignosis of large duct PSC
2) Liver biopsy at screening that is deemed acceptable for interpretation and demonstrates stage F0 - F3 fibrosis (according to the Ludwig classification) in the opinion of the central reader
3) Individual has the following laboratory parameters at the screening visit, as determined by the central laboratory:
a.Platelet count > =150,000/mm3
b.Estimated glomerular filtration rate (eGFR) > =30 milliliter/minute (mL/min), as calculated by the Cockcroft-Gault equation
c.ALT <= 8 x upper limit of the normal range (ULN)
d.Total bilirubin < 2 mg/dL, unless the individual is known to have Gilbert's syndrome or hemolytic anemia
e.International normalized ratio (INR) <= 1.4, unless due to therapeutic anticoagulation
f.Negative anti-mitochondrial antibody

1)Current or prior history of any of the following:
a.Cirrhosis
b.Liver transplantation
c.Cholangiocarcinoma or hepatocellular carcinoma (HCC)
d.Ascending cholangitis within 30 days of screening
2)Presence of a percutaneous drain or biliary stent
3)Other causes of liver disease
4)Current or prior history of unstable cardiovascular disease
5)Current moderate to severe inflammatory bowel disease (IBD)

Both

Primary Sclerosing Cholangitis

Experimental: Blinded Phase: Cilofexor
Cilofexor 100mg tablet administered orally once daily for 96 weeks

Experimental: Blinded Phase: Placebo
Placebo tablet administered orally once daily for 96 weeks

Experimental: Open-Label Phase: Cilofexor
Cilofexor 100mg tablet administered orally once daily for 96 weeks

Proportion of participants with progression of liver fibrosis at blinded phase week 96.
Progression of liver fibrosis was defined as having a >=1-stage increase in fibrosis according to the Ludwig classification.

1. Changes from baseline in serum concentrations of ALP, ALT, and bile acids at Blinded Study Phase Week 96
2. The proportion of participants with >= 25% relative reduction in serum ALP concentration from baseline (biochemical response) and no worsening of fibrosis according to the Ludwig classification (histologic response) at Blinded Study Phase Week 96
3. The proportion of participants with fibrosis improvement (according to the Ludwig classification) at Blinded Study Phase Week 96
4. Changes from baseline in noninvasive markers of fibrosis, including liver stiffness by FibroScan and ELF test score, at Blinded Study Phase Week 96
5. Change from baseline in PSC Symptoms - Module 1 based on the disease-specific PSC-PRO at Blinded Study Phase Week 96

+81-23-628-5840

June. 04, 2019