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CompletedNCT02078882PBCUpdated Apr 9, 2020Results posted

Study of Abatacept (Orencia) to Treat Primary Biliary Cirrhosis

A Phase 4 interventional study of abatacept in Primary Biliary Cirrhosis, sponsored by Christopher Bowlus, MD. Completed at 1 site in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2020-04-09.

Sponsored by Christopher Bowlus, MD · Phase 4, Interventional, and Treatment

Phase
Phase 4
Study type
Interventional
Enrollment
16
Allocation
Not applicable
Ages
18 Years and older
Sex
All
01

Study summary

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).

Read the detailed 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
02

Conditions studied

  • Primary Biliary Cirrhosis

Keywords

  • Primary Biliary Cirrhosis
  • Ursodeoxycholic acid
  • Abatacept
  • Cytotoxic T-Lymphocyte Antigen 4 (CTLA-4)
03

In context

Liver Cirrhosis

1,642 studies on the registry are indexed under Liver Cirrhosis; 358 are open to participants now.

This study's enrollment of 16 is below the median of 72 across 995 interventional studies indexed under Liver Cirrhosis.

Browse Liver Cirrhosis studies →

Lead sponsor

This is the only study on the registry with Christopher Bowlus, MD as lead sponsor.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

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

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
05

Study design

Phase
Phase 4
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
16 participants (actual)

Study arms

  • Experimental
    Abatacept 125 mg weekly

    Open label treatment with Abatacept

    Biological: abatacept

Interventions

  • Biologicalabatacept

    125 mg subcutaneously each week for 24 weeks

    Also known as: Orencia

06

What researchers measure

Primary outcomes

  1. 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

  1. 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

  2. Absolute Change in Alkaline Phosphatase

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

    Time frame: Week 24

  3. Absolute Change in Alanine Transferase (ALT)

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

    Time frame: Week 24

  4. 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

  5. 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

  6. Percent Change in Alkaline Phosphatase

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

    Time frame: Week 24

  7. Percent Change in Alanine Transferase (ALT)

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

    Time frame: Week 24

Other outcomes

  1. Immunoglobulin M (IgM) Levels

    Change in IgM level from Day 0 to Week 24

    Time frame: Week 24

  2. Memory T Cell Frequencies

    Change in cluster of differentiation 4 (CD4)+ cluster of differentiation 44 (CD44)+ cluster of differentiation 62 ligand (CD62L)- and cluster of differentiation 8+ CD44+ CD62L- frequencies in peripheral blood mononuclear cells from Day 0 to Week 24

    Time frame: Week 24

  3. Abatacept Levels

    Trough serum levels of abatacept

    Time frame: Day 0 and Weeks 4, 12, 24, and 36

07

Results

Posted Mar 30, 2020

Participant flow

Participant flow — Overall Study
MilestoneAbatacept 125 mg Weekly
Started16
Completed16
Not completed0

Outcome measures

PrimaryBiochemical 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
Reported as:
Count of participants · Participants
Biochemical Response
ParticipantsAbatacept 125 mg Weekly
Biochemical Response1
SecondaryDrug 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
Reported as:
Count of participants · Participants
Drug Safety
ParticipantsAbatacept 125 mg Weekly
Drug Safety4
SecondaryAbsolute Change in Alkaline Phosphatase

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

Time frame:
Week 24
Reported as:
Median · IU/L
Absolute Change in Alkaline Phosphatase
IU/LAbatacept 125 mg Weekly
Absolute Change in Alkaline Phosphatase-2.8 (-97.5 to 42.0)
SecondaryAbsolute Change in Alanine Transferase (ALT)

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

Time frame:
Week 24
Reported as:
Median · IU/L
Absolute Change in Alanine Transferase (ALT)
IU/LAbatacept 125 mg Weekly
Absolute Change in Alanine Transferase (ALT)0.5 (-13.5 to 4.5)
SecondaryLiver 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
Reported as:
Median · kPa
Liver Stiffness Measured by Magnetic Resonance Elastography
kPaAbatacept 125 mg Weekly
Liver Stiffness Measured by Magnetic Resonance Elastography-0.1 (-0.43 to 0.20)
SecondaryPrimary 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
Reported as:
Median · units on a scale
Primary Billiary Cholangitis Quality of Life
units on a scaleAbatacept 125 mg Weekly
Primary Billiary Cholangitis Quality of Life0 (-4 to 13)
SecondaryPercent Change in Alkaline Phosphatase

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

Time frame:
Week 24
Reported as:
Median · percent change
Percent Change in Alkaline Phosphatase
percent changeAbatacept 125 mg Weekly
Percent Change in Alkaline Phosphatase0.01 (-0.28 to 0.13)
SecondaryPercent Change in Alanine Transferase (ALT)

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

Time frame:
Week 24
Reported as:
Median · percent change
Percent Change in Alanine Transferase (ALT)
percent changeAbatacept 125 mg Weekly
Percent Change in Alanine Transferase (ALT)0.01 (-0.29 to 0.11)
Other pre-specifiedImmunoglobulin M (IgM) Levels

Change in IgM level from Day 0 to Week 24

Time frame:
Week 24
Reported as:
Median · mg/dL
Immunoglobulin M (IgM) Levels
mg/dLAbatacept 125 mg Weekly
Immunoglobulin M (IgM) Levels0.0 (-44.0 to 45.0)
Other pre-specifiedMemory T Cell Frequencies

Change in cluster of differentiation 4 (CD4)+ cluster of differentiation 44 (CD44)+ cluster of differentiation 62 ligand (CD62L)- and cluster of differentiation 8+ CD44+ CD62L- frequencies in peripheral blood mononuclear cells from Day 0 to Week 24

Time frame:
Week 24

Results for this outcome have not been posted.

Other pre-specifiedAbatacept Levels

Trough serum levels of abatacept

Time frame:
Day 0 and Weeks 4, 12, 24, and 36

Results for this outcome have not been posted.

Adverse events

Collected over 36 weeks. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Abatacept 125 mg Weekly0/16 (0%)0/16 (0%)4/16 (25%)
Most frequent other events
Most frequent other events
EventAbatacept 125 mg Weekly
NauseaGastrointestinal disorders2/16
VomitingGastrointestinal disorders1/16
Elevated liver enzymesGastrointestinal disorders1/16
Right upper quadrant painGastrointestinal disorders1/16
Upper respiratory infectionInfections and infestations1/16
Urinary Tract InfectionsInfections and infestations1/16
Urticarial RashSkin and subcutaneous tissue disorders1/16
Chest PainCardiac disorders1/16
Hilar AdenopathyBlood and lymphatic system disorders1/16

Baseline characteristics

Age, Categorical
Age, Categorical(Participants)Abatacept 125 mg Weekly
<=18 years0
Between 18 and 65 years14
>=65 years2
Age, Continuous
Age, Continuous(years)Abatacept 125 mg Weekly
Mean52 (39 to 70)
Sex: Female, Male
Sex: Female, Male(Participants)Abatacept 125 mg Weekly
Female15
Male1
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Abatacept 125 mg Weekly
Hispanic or Latino3
Not Hispanic or Latino13
Unknown or Not Reported0
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Abatacept 125 mg Weekly
American Indian or Alaska Native0
Asian0
Native Hawaiian or Other Pacific Islander0
Black or African American0
White16
More than one race0
Unknown or Not Reported0
Region of Enrollment
Region of Enrollment(participants)Abatacept 125 mg Weekly
United States16
08

Study locations

1 site
  • Univeristy of California Davis Medical Center
    Sacramento, California 95817, United States
09

References and documents

Study documents

  • Protocol and statistical analysis plan · Sep 22, 2015
  • Informed consent form · Sep 22, 2015

Documents are hosted by the registry — open the source record to download them.

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Apr 9, 2020, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT02078882
Lead sponsor
Christopher Bowlus, MD
Collaborators
Bristol-Myers Squibb
Responsible party
Christopher Bowlus, MD (Professor, Gastroenterology and Hepatology, Internal Medicine, University of California, Davis) — Sponsor-investigator
First posted
Mar 5, 2014
Start date
Sep 2014
Primary completion
Oct 2018
Completion
Oct 2018
Results posted
Mar 30, 2020
Last update
Apr 9, 2020

Study contacts

Christopher L Bowlus, MD
principal investigator · University of California, Davis
M. Eric Gershwin, MD
principal investigator · University of California, Davis

Oversight

Data monitoring committee
Yes
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Mar 2020. You cannot join it, but the record below documents what was studied.

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