Study of Abatacept (Orencia) to Treat Primary Biliary Cirrhosis

Phase 4CompletedNCT02078882

Christopher Bowlus, MD16 enrolled

Overview

The purpose of this study is to determine if abatacept (Orencia) is effective in patients with primary biliary cirrhosis who do not respond adequately to standard treatment with ursodeoxycholic acid (UDCA, Urso, Ursodiol, Actigall).

This is an open label, active treatment trial to assess the efficacy and safety of abatacept in subject with PBC who have had an incomplete biochemical response to UDCA. In this trial, 20 subjects with PBC who have had an incomplete biochemical response to UDCA will be assigned to treatment with weekly subcutaneous injections of 125 mg of abatacept. The treatment phase of the study will last 24 weeks with an off-treatment follow up at Week 36. Inclusion criteria include: * Confirmed diagnosis of PBC * Alkaline phosphatase \> 1.67 times the upper limit of normal after 6 months of treatment with UDCA

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Primary Biliary Cirrhosis

Interventions

abataceptBIOLOGICAL

125 mg subcutaneously each week for 24 weeks

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Confirmed PBC diagnosis based upon at least 2 of 3 criteria 1. Anti-mitochondrial antibody (AMA) titer \> 1:40 2. Alkaline phosphatase \> 1.5 times the upper limit of normal for at least 6 months 3. Liver biopsy findings consistent with PBC * Incomplete response to UDCA defined by an alkaline phosphatase \> 1.67 X the upper limit of normal after 6 months of UDCA at a minimum dose of 13 mg/kg/d * Taking a stable dose of UDCA for at least 3 months prior to Day 0 * aspartate aminotransferase (AST) and alanine aminotransferase ALT \< 5 times the upper limit of normal Exclusion Criteria: * Presence of concomitant liver diseases including viral hepatitis, primary sclerosing cholangitis, alcoholic liver disease, Wilson's disease, hemochromatosis, or Gilbert's syndrome. * Prior liver transplantation * Decompensated liver disease * Use of immunosuppressants within 6 months of Day 0 * Use of biologic agents within 12 months of Day 0

Locations (1)

Univeristy of California Davis Medical Center

Sacramento, California, United States

Outcomes

Primary Outcomes

Biochemical Response

Number of Participants with a decrease of alkaline phosphatase by \> 40%of the Day 0 level at 24 weeks of treatment.

Time frame: Week 24

Secondary Outcomes

Drug Safety

Number of participants with any adverse events, clinically significant changes in vital signs, laboratory test abnormalities, and clinical tolerability of the drug.

Time frame: Weeks 2, 4, 12, 24, and 36

Absolute Change in Alkaline Phosphatase

The absolute change in alkaline phosphatase from Day 0 to Week 24.

Time frame: Week 24

Absolute Change in Alanine Transferase (ALT)

The absolute change in alanine transferase (ALT) from Day 0 to Week 24.

Time frame: Week 24

Liver Stiffness Measured by Magnetic Resonance Elastography

Change in liver stiffness measured by magnetic resonance elastography from Day 0 to Week 24.

Time frame: Week 24

Primary Billiary Cholangitis Quality of Life

Change in quality of life measured by change in primary biliary cholangitis (PBC)-40 from Day 0 to Week 24. is a patient-derived, disease specific quality of life measure developed and validated for use in PBC with subscores for domains of symptoms, itch, fatigue, cognition, social, and emotional. Subdomains are summed with a total score range of 36 to 200. Higher scores indicate worse quality of life.

Time frame: Week 24

Percent Change in Alkaline Phosphatase

The percent change in alkaline phosphatase from Day 0 to Week 24.

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.