Primary Biliary CirrhosisPhase 4PBCNCT02078882
Study of Abatacept (Orencia) to Treat Primary Biliary Cirrhosis
A Phase 4 study testing abatacept in people with Primary Biliary Cirrhosis, run by Christopher Bowlus, MD at 1 site in United States.
Trial 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 more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Primary Biliary Cirrhosis
- What is being tested?
- abatacept
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 1 siteUnited States
- Study dates
- Sep 2014to Oct 2018
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Confirmed PBC diagnosis based upon at least 2 of 3 criteria
- Anti-mitochondrial antibody (AMA) titer > 1:40
- Alkaline phosphatase > 1.5 times the upper limit of normal for at least 6 months
- 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
Who cannot join
- 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 locations and contacts
1 site in 1 country.
United States1 site
Univeristy of California Davis Medical Center
Sacramento, California, 95817
About this study
Official title: Abatacept For The Treatment Of Primary Biliary Cirrhosis With An Incomplete Biochemical Response To Ursodeoxycholic Acid
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
- Conditions
- Primary Biliary Cirrhosis
- Being tested
- abatacept
- Keywords
- Primary Biliary Cirrhosis, Ursodeoxycholic acid, Abatacept, Cytotoxic T-Lymphocyte Antigen 4 (CTLA-4)
- Medical subject headings
- Liver Cirrhosis, Biliary, Diabetes Mellitus, Insulin-Dependent, 12
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
abataceptBiological
Also called Orencia
125 mg subcutaneously each week for 24 weeks
| Study group | Role | What they receive |
|---|---|---|
| Abatacept 125 mg weekly | Experimental | Open label treatment with Abatacept Receives: Biological: abatacept |
What is being measured
The primary outcome is the main question the study is designed to answer.
Biochemical Response
Number of Participants with a decrease of alkaline phosphatase by > 40%of the Day 0 level at 24 weeks of treatment.
Measured: Week 24
Secondary outcomes (7)
Drug Safety
Number of participants with any adverse events, clinically significant changes in vital signs, laboratory test abnormalities, and clinical tolerability of the drug.
Measured: Weeks 2, 4, 12, 24, and 36
Absolute Change in Alkaline Phosphatase
The absolute change in alkaline phosphatase from Day 0 to Week 24.
Measured: Week 24
Absolute Change in Alanine Transferase (ALT)
The absolute change in alanine transferase (ALT) from Day 0 to Week 24.
Measured: Week 24
Liver Stiffness Measured by Magnetic Resonance Elastography
Change in liver stiffness measured by magnetic resonance elastography from Day 0 to Week 24.
Measured: 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.
Measured: Week 24
Percent Change in Alkaline Phosphatase
The percent change in alkaline phosphatase from Day 0 to Week 24.
Measured: Week 24
Percent Change in Alanine Transferase (ALT)
The percent change in alanine transferase (ALT) from Day 0 to Week 24.
Measured: Week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Mar 2014 (estimated)
- Study startSep 2014
- Primary completionOct 2018
- Study completionOct 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Primary Biliary CirrhosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- Christopher Bowlus, MD
- Sponsor type
- University, hospital or other
- Collaborators
- Bristol-Myers Squibb
- Responsible party
- Sponsor-investigator, Christopher Bowlus, MD, University of California, Davis
- Investigators
- Christopher L Bowlus, MD, Principal investigator, University of California, DavisM. Eric Gershwin, MD, Principal investigator, University of California, Davis
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02078882
- Sponsor's protocol ID
- 599969
- Other IDs
- IM101-457
- Acronym
- PBC
- Status last verified
- Mar 2020
- First submitted
- 3 Mar 2014
- Last updated
- 9 Apr 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 9 Apr 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Abatacept For The Treatment Of Primary Biliary Cirrhosis With An Incomplete Biochemical Response To Ursodeoxycholic Acid
- ClinicalTrials.gov ID
- NCT02078882
- Acronym / sponsor ID
- PBC / 599969
- Conditions
- Primary Biliary Cirrhosis
- Keywords
- Primary Biliary Cirrhosis, Ursodeoxycholic acid, Abatacept, Cytotoxic T-Lymphocyte Antigen 4 (CTLA-4)
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 16 participants (actual)
- Lead sponsor
- Christopher Bowlus, MDUniversity, hospital or other
- Collaborators
- Bristol-Myers Squibb
- Study start
- Sep 2014
- Primary completion
- Oct 2018 (actual)
- Study completion
- Oct 2018 (actual)
- First posted
- 5 Mar 2014 (estimated)
- Results
- Posted 30 Mar 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
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
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Abatacept 125 mg weekly | Experimental | Biological: abataceptOpen label treatment with Abatacept |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Confirmed PBC diagnosis based upon at least 2 of 3 criteria
- 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
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| abataceptAlso: Orencia125 mg subcutaneously each week for 24 weeks | Biological | Abatacept 125 mg weekly |
Primary outcome
Biochemical Response
Time frame: Week 24
Number of Participants with a decrease of alkaline phosphatase by > 40%of the Day 0 level at 24 weeks of treatment.
7 secondary outcomes
Drug Safety
Time frame: Weeks 2, 4, 12, 24, and 36
Absolute Change in Alkaline Phosphatase
Time frame: Week 24
Absolute Change in Alanine Transferase (ALT)
Time frame: Week 24
Liver Stiffness Measured by Magnetic Resonance Elastography
Time frame: Week 24
Primary Billiary Cholangitis Quality of Life
Time frame: Week 24
Percent Change in Alkaline Phosphatase
Time frame: Week 24
Percent Change in Alanine Transferase (ALT)
Time frame: Week 24
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Apr 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- PBC Patient OrganizationLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Christopher L Bowlus, MD principal investigator
University of California, Davis
M. Eric Gershwin, MD principal investigator
University of California, Davis
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing abatacept in people with Primary Biliary Cirrhosis, run by Christopher Bowlus, MD at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 9 Apr 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 30 Mar 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Biochemical Response (time frame: Week 24). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Apr 2020.