Study of Cilofexor in Adults With Primary Sclerosing Cholangitis (PRIMIS)

Gilead Sciences

Status and phase: Terminated; Phase 3

Conditions: Primary Sclerosing Cholangitis

Treatments: Drug: Placebo; Drug: Cilofexor

Study type: Interventional

Funder types: Industry

Identifiers

NCT03890120

GS-US-428-4194

jRCT2080224728 (Registry Identifier)

2019-000204-14 (EudraCT Number)

Details and patient eligibility

About

The primary objective of this study is to evaluate whether cilofexor reduces the risk of fibrosis progression among non-cirrhotic adults with primary sclerosing cholangitis (PSC).

Enrollment: 419 patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

Inclusion and 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
  • Alanine transaminase (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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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419 participants in 4 patient groups, including a placebo group

Cilofexor 100 mg (Blinded Phase)

Experimental group

Description:

Participants received cilofexor 100 mg tablet, orally, once daily for up to 100.3 weeks.

Treatment:

Drug: Cilofexor

Placebo (Blinded Phase)

Placebo Comparator group

Description:

Participants received placebo to match cilofexor 100 mg tablet, orally, once daily for up to 98.1 weeks.

Treatment:

Drug: Placebo

Cilofexor From Cilofexor 100 mg (OLE Phase)

Experimental group

Description:

Participants who received cilofexor in blinded phase and had entered the open-label extension (OLE) phase received open-label cilofexor 100 mg tablet, orally, once daily for up to 44.7 weeks.

Treatment:

Drug: Cilofexor

Cilofexor From Placebo (OLE Phase)

Experimental group

Description:

Participants who received placebo in blinded phase and had entered the OLE phase received open-label cilofexor 100 mg tablet, orally, once daily for up to 45.0 weeks.

Treatment:

Drug: Cilofexor