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

Gilead Sciences

Status and phase: Terminated; Phase 1

Conditions: Primary Sclerosing Cholangitis; Compensated Cirrhosis

Treatments: Drug: Cilofexor

Study type: Interventional

Funder types: Industry

Identifiers

NCT04060147

GS-US-428-5443

Details and patient eligibility

About

The primary objective of this study is to assess the safety and tolerability of escalating doses of cilofexor (CILO) in participants with primary sclerosing cholangitis (PSC) and compensated cirrhosis.

Enrollment: 11 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and 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 [magnetic resonance imaging (MRI), computed tomography (CT), or Ultrasound], or a screening FibroScan®, enhanced liver fibrosis (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 milliliter/minute (mL/min), as calculated by the Cockcroft-Gault equation
  • Alanine aminotransferase (ALT) ≤ 5 x upper limit of the normal (ULN)
  • Total 2 milligram/deciliter (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
  • Platelet count ≥ 75,000/microliter (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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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11 participants in 1 patient group

Cilofexor

Experimental group

Description:

Participants will receive escalating doses of cilofexor 30 mg, 60 mg, and 100 mg.

Treatment:

Drug: Cilofexor