A Study of the Safety and Effectiveness of Experimental Cilofexor for Primary Sclerosing Cholangitis (scarring of bile ducts)

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Age: 18 to 70 years old
Gender:
Any
Healthy Volunteers: No
Keywords: Primary Sclerosing Cholangitis, liver, PSC
Type: Drug study, Phase 3
Target:
2 Participants
Investigator:
Description
We invite you to take part in a research study because you have primary sclerosing cholangitis (PSC). PSC is a chronic disease which causes scarring of the bile ducts. When the bile ducts are damaged, the ducts become hard and narrow and this can block the flow of bile which can cause damage to the liver over time.

This study will test an experimental drug named GS-9674 (also referred to as cilofexor). It will be used for the treatment of primary sclerosing cholangitis (PSC).

PSC can be without symptoms, especially early in the disease. Some symptoms of PSC can include fatigue (feeling tired), itching, and jaundice (yellowing of eyes and skin). The symptoms of PSC are related to the blockage of bile flow from the scarred bile ducts and a build-up of bile acids. Patients with PSC can develop cirrhosis (severe scarring of the liver). This can lead to liver failure. PSC is also a major risk factor for the development of cancer of the bile ducts (cholangiocarcinoma).

The only effective treatment for PSC is liver transplantation. This involves removing the damaged liver and replacing it with a healthy one from a donor. Experimental GS-9674 is being developed to reduce bile acids. Elevated bile acids contribute to symptoms of PSC including inflammation and fibrosis (scarring) in the liver.
This study requires

The study team will make sure you qualify to take part in the study. Qualified participants will receive detailed information about the study. This may include a list of study-related tests and procedures.

Who can participate

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 severely active inflammatory bowel disease (IBD) Note: Other protocol defined Inclusion/Exclusion criteria may apply
Benefits and risks of participating
BENEFITS:

We cannot promise any benefits to you as a result of taking part in this research study.


RISKS:

The study team will discuss the risks associated with taking part in this research study.
Compensation
If you agree to take part in this research study, we will compensate you $65.00 for the completion of each in person study visit for your time and effort. When you complete the liver biopsy, you will be compensated $100. Subjects will receive their compensation quarterly in the form of a check by mail. If you discontinue early from the study, you will receive a pro-rated (partial) reimbursement amount based on how many study visits you completed.
Resources
Schedule
Study duration and period
We expect that you will be in this research study for about 100 weeks. This does not include the screening period which can last up to 10 weeks. During this time, you will be required to visit the clinic at least 14 times, including the screening visit.
Recruitment period
From Aug. 20, 2019
Location
UC Davis Medical Center
2315 Stockton Boulevard
Sacramento, CA 95817
Contact
Sophia Zaragoza
Research Topic
Conditions:
  • Primary Sclerosing Cholangitis