Study of Abatacept (Orencia) to Treat Primary Biliary Cirrhosis (PBC)

Christopher Bowlus, MD

Status and phase: Completed; Phase 4

Conditions: Primary Biliary Cirrhosis

Treatments: Biological: abatacept

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT02078882

599969

IM101-457 (Other Identifier)

Details and patient eligibility

About

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).

Full description

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

Enrollment: 16 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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16 participants in 1 patient group

Abatacept 125 mg weekly

Experimental group

Description:

Open label treatment with Abatacept

Treatment:

Biological: abatacept