A Study of the Safety, Tolerability, and Effectiveness of GS-9674 for Adults with Primary Sclerosing Cholangitis without Cirrhosis

Overview

  • Study type

    Interventional
  • Study phase

    II
  • Study IDs

  • Describes the nature of a clinical study. Types include:

    • Observational study — observes people and measures outcomes without affecting results.
    • Interventional study (clinical trial) — studies new tests, treatments, drugs, surgical procedures or devices.
    • Medical records research — uses historical information collected from medical records of large groups of people to study how diseases progress and which treatments and surgeries work best.
  • During the early phases (phases 1 and 2), researchers assess safety, side effects, optimal dosages and risks/benefits. In the later phase (phase 3), researchers study whether the treatment works better than the current standard therapy. They also compare the safety of the new treatment with that of current treatments. Phase 3 trials include large numbers of people to make sure that the result is valid. There are also less common very early (phase 0) and later (phase 4) phases. Phase 0 trials are small trials that help researchers decide if a new agent should be tested in a phase 1 trial. Phase 4 trials look at long-term safety and effectiveness, after a new treatment has been approved and is on the market.

  • Site IRB
    • Scottsdale/Phoenix, Arizona: 16-009492
    NCT ID: NCT02943460
    Sponsor Protocol Number: GS-US-428-4025

About this study

The purpose of this study is to evaluate the safety, tolerability, and effectiveness of GS-9674 in adults who have primary sclerosing cholangitis.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. If you need assistance understanding the eligibility criteria, please contact the study team.

See eligibility criteria

Inclusion Criteria

  • Diagnosis of PSC based on cholangiogram (magnetic resonance cholangiopancreatography (MRCP), endoscopic retrograde cholangiopancreatography (ERCP), or percutaneous transhepatic cholangiogram (PTC)) within the previous 12 months
  • Serum alkaline phosphatase (ALP) > 1.67 x upper limit of the normal range (ULN)
  • If on ursodeoxycholic acid (UDCA), dose must have been stable for at least 12 months prior to screening through the end of treatment
  • If not on UDCA, then no UDCA use for at least 12 months before screening through the end of treatment
  • Screening FibroSURE/FibroTest® < 0.75 unless a historical liver biopsy within 12 months of screening does not reveal cirrhosis
    • With Gilbert's syndrome or hemolysis, FibroSURE/FibroTest® will be calculated using direct bilirubin instead of total bilirubin

Exclusion Criteria

  • Alanine aminotransferase (ALT) > 10 x ULN
  • Total bilirubin > 2 x ULN
  • International normalized ratio (INR) > 1.2 unless on anticoagulant therapy
  • Small-duct PSC (histologic evidence of PSC with normal bile ducts on cholangiography)
  • Other causes of liver disease including secondary sclerosing cholangitis and viral, metabolic, alcoholic, and other autoimmune conditions
  • Ascending cholangitis within 60 days of screening
  • Presence of a percutaneous drain or bile duct stent
  • Use of fibrates or obeticholic acid within 3 months prior to screening through the end of treatment
  • Cirrhosis of the liver as defined by any of the following
    • Historical liver biopsy demonstrating cirrhosis (eg, Ludwig stage 4 or Ishak stage ≥ 5)
    • Prior history of decompensated liver disease, including ascites, hepatic encephalopathy or variceal bleeding
    • Liver stiffness > 14.4 kPa by FibroScan
  • Current, active inflammatory bowel disease (IBD) defined as a partial Mayo score of > 1 and/or a score on the Rectal Bleeding domain > 0.

Note: Other protocol defined Inclusion/Exclusion criteria may apply.

Participating Mayo Clinic locations

Study statuses change often. Please contact us for help.

Mayo Clinic Location Status Contact

Scottsdale/Phoenix, Ariz.

Mayo Clinic principal investigator

Elizabeth Carey, M.D.

Open for enrollment

Contact information:

Vy Nguyen CCRP

(480)342-1328

Nguyen.Vy@mayo.edu

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CLS-20318101

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