Study Investigating the Activity of Vidofludimus Calcium as a Treatment for Primary Sclerosing Cholangitis (PSC)

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: 18-005367
    • Rochester, Minnesota: 18-005367
    NCT ID: NCT03722576
    Sponsor Protocol Number: 18-005367

About this study

The purpose of this study is to examine the safety, tolerability, and efficacy of daily dosing with vidofludimus calcium over a 6-month period on the clinical course and progression of 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.

Inclusion Criteria:

  • Male or female subject age 18-75 years old.
  • Diagnosis of PSC consistent with the guidelines published by the AASLD. All subjects must have an elevated serum ALP of at least 1.5 times upper limit of normal (ULN) at baseline plus cholangiographic evidence of PSC (MRI, endoscopic retrograde cholangiography, or direct cholangiography).
  • Indirect bilirubin <1.2 times the ULN.
  • An ultrasound (or equivalent imaging modality) that excludes biliary obstruction and malignancy within 6 months of study enrollment.
  • PSC with or without inflammatory bowel disease, such as ulcerative colitis or Crohn’s disease.
  • Must agree to comply with the study protocol and provide informed consent.

Exclusion Criteria:

  • Pregnancy, attempting to become pregnant, or breastfeeding.
  • Active hepatitis A or B infection.
  • Active hepatitis C infection (antibody positive); patients with a history of hepatitis C infection will be eligible for this study if they have undetectable levels of HCV RNA.
  • HIV/AIDS (per medical record or HIVAb/HIV antigen) , tuberculosis, or positive interferon-gamma assay (IGRAs) for Mycobacterium tuberculosis.
  • Other cholestatic liver disease such as primary biliary cholangitis and cholestatic diseases of pregnancy.
  • Metabolic liver diseasessuchasWilson’sdisease,Gilbert’ssyndromeorhemochromatosis.
  • Serum uric acid levels at Screening >1.2 ULN.
  • Inherited diseases of the liver such as α-1antitrypsin deficiency.
  • Immunoglobulin G4-related cholangitis.
  • PSC with concomitant autoimmune hepatitis (AIH) and/or primary biliary cholangitis.
  • Secondary sclerosing cholangitis (SSC).
  • Active acute ascending cholangitis requiring antibiotics.
  • CCA (malignant biliary stricture, neoplasm, and cytology/histopathology or positive fluorescence in situ hybridization (FISH) consistent with adenocarcinoma of the bile duct).
  • A liver biopsy, if one has been previously obtained, which showed non-alcoholic steatohepatitis (NASH). Patients with suspected fatty liver by imaging will not be excluded.
  • Presence of complications of advanced PSC such as hepatic encephalopathy, portal hypertension, hepato-renal syndrome, and hepato-pulmonary syndrome.
  • History of liver transplantation, anticipated need for liver transplantation within 12 months fromrandomization,aModelofEnd-stageLiverDisease(MELD)scoreof ≥15, or a Child Pugh score >6.
  • Ongoing alcohol abuse (>4 drinks per day for men, and >2 drinks per day for women).
  • Moderate-to-severe renal impairment with a calculated creatinine clearance of <60mL/min.
  • Any other conditions or abnormalities that, in the opinion of the investigator, may compromise the safety of the subject or interfere with the subject participating in or completing the study.
  • Evidence of, or treatment for, C. difficile infection within 30 days before the initiation of the study drug.
  • Evidence of active C. difficile infection during the screening phase confirmed by a positive C. difficile toxin B.
  • Subjects who have been treated for intestinal pathogens other than C. difficile infection within 30 days prior to study drug initiation.
  • Received or plan to receive live vaccine within 30 days prior to, and through the end of the study.
  • Use of methotrexate at dose ≥17.5mg/week.
  • Rosuvastatin exceeding 10 mg daily.

 

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.

Contact us for the latest status

Contact information:

Travis Johnson R.T.(R)(VI)

(480)342-2942

Johnson.Travis@mayo.edu

Rochester, Minn.

Mayo Clinic principal investigator

John Eaton, M.D.

Contact us for the latest status

Contact information:

Gastroenterology Study Coordinator

(507) 284-2698

More information

Publications

Publications are currently not available

Study Results Summary

Not yet available

Supplemental Study Information

Not yet available