A Study of Seladelpar in Subjects with Primary Biliary Cholangitis (PBC) and an Inadequate Response to or an Intolerance to Ursodeoxycholic Acid (UDCA)

Overview

  • Study type

    Interventional
  • Study phase

    III
  • 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: 18-008715
    NCT ID: NCT03602560
    Sponsor Protocol Number: CB8025-31735

About this study

The purpose of this study is to evaluate the safety and effectiveness of seladelpar in subjects with primary biliary cholangitis (PBC) and an inadequate response to or intolerance to ursodeoxycholic acid (UDCA)

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: 

  • Must have given written informed consent (signed and dated) and any authorizations required by local law.
  • 18 to 75 years old (inclusive) .
  • Male or female with a diagnosis of PBC, by at least two of the following criteria: 
    • History of AP above ULN for at least six months:
      • Positive anti-mitochondrial antibody (AMA) titers (>1/40 on immunofluorescence or M2 positive by enzyme linked immunosorbent assay [ELISA]) or positive PBC-specific antinuclear antibodies;
      • Documented liver biopsy result consistent with PBC.
  • On a stable and recommended dose of UDCA for the past twelve months OR intolerant to UDCA (last dose of UDCA > 3 months prior to Screening) .
  • AP ≥ 1.67 × ULN.
  • Females of reproductive potential must use at least one barrier contraceptive and a second effective birth control method during the study and for at least 90 days after the last dose. Male subjects who are sexually active with female partners of reproductive potential must use barrier contraception and their female partners must use a second effective birth control method during the study and for at least 90 days after the last dose.

Exclusion Criteria:

  • Previous exposure to seladelpar (MBX-8025).
  • A medical condition, other than PBC, that in the investigator's opinion would preclude full participation in the study or confound its results (e.g., cancer).
  • AST above 3 × ULN.
  • ALT above 3 × ULN.
  • Total bilirubin above 2.0 × ULN.
  • Advanced PBC as defined by the Rotterdam criteria (albumin below LLN AND total bilirubin above 1 × ULN).
  • Creatine kinase (CK) above 1.0 × ULN.
  • eGFR below 60 mL/min/1.73 m2 (calculated by MDRD formula).
  • International normalized ratio (INR) above 1.0 × ULN.
  • Platelet count below 100 × 103/µL
  • Presence of clinically significant hepatic decompensation, including:
    • History of liver transplantation, current placement on liver transplantation list, or current MELD score ≥ 15;
    • Complications of portal hypertension, including known esophageal varices, history of variceal bleeds or related interventions (e.g., transjugular intrahepatic portosystemic shunt placement), relevant ascites, hepatic encephalopathy;
    • Cirrhosis with complications, including history or presence of spontaneous bacterial peritonitis.
  • Other chronic liver diseases:
    • Current features of auto-immune hepatitis as determined by the investigator based on immunoserology, liver biochemistry and histology;
    • Primary sclerosing cholangitis determined by presence of diagnostic cholangiographic findings;
    • History or clinical evidence of alcoholic liver disease;
    • History or clinical evidence of alpha-1-antitrypsin deficiency ;
    • Biopsy confirmed nonalcoholic steatohepatitis;
    • History or evidence of Gilbert' Syndrome with elevated total bilirubin;
    • History or evidence of hemochromatosis;
    • Hepatitis B defined as presence of hepatitis B surface antigen (HBsAg);
    • Hepatitis C defined as presence of HCV RNA 13. Known history of HIV.
  • Evidence of significant alcohol consumption.
  • Evidence of drug abuse.
  • Subjects with inadequate response to obeticholic acid (OCA) or intolerance to OCA: OCA must be discontinued 30 days prior to Screening.
  • Use of colchicine, methotrexate, azathioprine, or long-term systemic corticosteroids (> 2 weeks) within two months prior to Screening.
  • Use of fibrates within 30 days prior to Screening.
  • Use of simvastatin within 7 days prior to Screening.
  • Use of an experimental or unapproved treatment for PBC within 30 days prior to Screening.
  • Use of experimental or unapproved immunosuppressant within 30 days prior to Screening.
  • Treatment with any other investigational therapy or device within 30 days or within five half-lives, whatever is longer, prior to Screening.
  • For females, pregnancy or breast-feeding.
  • Any other condition(s) that would compromise the safety of the subject or compromise the quality of the clinical study, as judged by the investigator.

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:

Angela Eyshou CCRP

(480)342-3906

Eyshou.Angela@mayo.edu

More information

Publications

Publications are currently not available

Study Results Summary

Not yet available

Supplemental Study Information

Not yet available