Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| IM101-457 | Other Identifier | Other |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Class |
|---|---|
| Bristol-Myers Squibb | INDUSTRY |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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:
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Abatacept 125 mg weekly | Experimental | Open label treatment with Abatacept |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| abatacept | Biological | 125 mg subcutaneously each week for 24 weeks |
|
| Measure | Description | Time Frame |
|---|---|---|
| Biochemical Response | Number of Participants with a decrease of alkaline phosphatase by > 40%of the Day 0 level at 24 weeks of treatment. | Week 24 |
| Measure | Description | Time Frame |
|---|---|---|
| Drug Safety | Number of participants with any adverse events, clinically significant changes in vital signs, laboratory test abnormalities, and clinical tolerability of the drug. | Weeks 2, 4, 12, 24, and 36 |
| Absolute Change in Alkaline Phosphatase |
| Measure | Description | Time Frame |
|---|---|---|
| Immunoglobulin M (IgM) Levels | Change in IgM level from Day 0 to Week 24 | Week 24 |
| 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 |
Inclusion Criteria:
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
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Christopher L Bowlus, MD | University of California, Davis | Principal Investigator |
| M. Eric Gershwin, MD | University of California, Davis | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Univeristy of California Davis Medical Center | Sacramento | California | 95817 | United States |
Not provided
| Label | URL |
|---|---|
| PBC Patient Organization | View source |
Not provided
Not provided
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Abatacept 125 mg Weekly | Open label treatment with Abatacept abatacept: 125 mg subcutaneously each week for 24 weeks |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Abatacept 125 mg Weekly | Open label treatment with Abatacept abatacept: 125 mg subcutaneously each week for 24 weeks |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Biochemical Response | Number of Participants with a decrease of alkaline phosphatase by > 40%of the Day 0 level at 24 weeks of treatment. | Posted | Count of Participants | Participants | Week 24 |
|
|
36 weeks
Adverse events were summarized according to MedDRA System Organ class, MedDRA preferred term, severity and causal relationship assessed by investigators and were reviewed by Data Monitoring Board.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Abatacept 125 mg Weekly | Open label treatment with Abatacept abatacept: 125 mg subcutaneously each week for 24 weeks |
Not provided
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Nausea | Gastrointestinal disorders | Nausea | Systematic Assessment |
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Christopher L Bowlus | University of California Davis | 9167348696 | clbowlus@ucdavis.edu |
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Sep 22, 2015 | Feb 3, 2020 | Prot_SAP_000.pdf |
| ICF | No | No | Yes | Informed Consent Form | Sep 22, 2015 | Feb 3, 2020 | ICF_001.pdf |
Not provided
| ID | Term |
|---|---|
| D008105 | Liver Cirrhosis, Biliary |
| C563326 | Diabetes Mellitus, Insulin-Dependent, 12 |
| ID | Term |
|---|---|
| D002780 | Cholestasis, Intrahepatic |
| D002779 | Cholestasis |
| D001649 | Bile Duct Diseases |
| D001660 | Biliary Tract Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| D000069594 | Abatacept |
| ID | Term |
|---|---|
| D018796 | Immunoconjugates |
| D000906 | Antibodies |
| D007136 | Immunoglobulins |
| D012712 | Serum Globulins |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
The absolute change in alkaline phosphatase from Day 0 to Week 24.
| Week 24 |
| Absolute Change in Alanine Transferase (ALT) | The absolute change in alanine transferase (ALT) from Day 0 to Week 24. | Week 24 |
| Liver Stiffness Measured by Magnetic Resonance Elastography | Change in liver stiffness measured by magnetic resonance elastography from Day 0 to Week 24. | 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. | Week 24 |
| Percent Change in Alkaline Phosphatase | The percent change in alkaline phosphatase from Day 0 to Week 24. | Week 24 |
| Percent Change in Alanine Transferase (ALT) | The percent change in alanine transferase (ALT) from Day 0 to Week 24. | Week 24 |
| Week 24 |
| Abatacept Levels | Trough serum levels of abatacept | Day 0 and Weeks 4, 12, 24, and 36 |
| Participants |
|
| Age, Continuous | Mean | Full Range | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
|
| Secondary | Drug Safety | Number of participants with any adverse events, clinically significant changes in vital signs, laboratory test abnormalities, and clinical tolerability of the drug. | Posted | Count of Participants | Participants | Weeks 2, 4, 12, 24, and 36 |
|
|
|
| Secondary | Absolute Change in Alkaline Phosphatase | The absolute change in alkaline phosphatase from Day 0 to Week 24. | Posted | Median | Inter-Quartile Range | IU/L | Week 24 |
|
|
|
| Secondary | Absolute Change in Alanine Transferase (ALT) | The absolute change in alanine transferase (ALT) from Day 0 to Week 24. | Posted | Median | Inter-Quartile Range | IU/L | Week 24 |
|
|
|
| Secondary | Liver Stiffness Measured by Magnetic Resonance Elastography | Change in liver stiffness measured by magnetic resonance elastography from Day 0 to Week 24. | Posted | Median | Inter-Quartile Range | kPa | Week 24 |
|
|
|
| Secondary | 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. | Posted | Median | Inter-Quartile Range | units on a scale | Week 24 |
|
|
|
| Secondary | Percent Change in Alkaline Phosphatase | The percent change in alkaline phosphatase from Day 0 to Week 24. | Posted | Median | 95% Confidence Interval | percent change | Week 24 |
|
|
|
| Secondary | Percent Change in Alanine Transferase (ALT) | The percent change in alanine transferase (ALT) from Day 0 to Week 24. | Posted | Median | Inter-Quartile Range | percent change | Week 24 |
|
|
|
| Other Pre-specified | Immunoglobulin M (IgM) Levels | Change in IgM level from Day 0 to Week 24 | Posted | Median | Inter-Quartile Range | mg/dL | Week 24 |
|
|
|
| Other Pre-specified | 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 | Not Posted | Week 24 | Participants |
| Other Pre-specified | Abatacept Levels | Trough serum levels of abatacept | Not Posted | Day 0 and Weeks 4, 12, 24, and 36 | Participants |
| 0 |
| 16 |
| 0 |
| 16 |
| 4 |
| 16 |
| Vomiting | Gastrointestinal disorders | MedDRA | Systematic Assessment |
|
| Elevated liver enzymes | Gastrointestinal disorders | MedDRA | Systematic Assessment |
|
| Right upper quadrant pain | Gastrointestinal disorders | MedDRA | Systematic Assessment |
|
| Upper respiratory infection | Infections and infestations | MedDRA | Systematic Assessment |
|
| Urinary Tract Infections | Infections and infestations | MedDRA | Systematic Assessment |
|
| Urticarial Rash | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
|
| Chest Pain | Cardiac disorders | MedDRA | Systematic Assessment |
|
| Hilar Adenopathy | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
|
Not provided
Not provided
| D004066 |
| Digestive System Diseases |
| D008107 | Liver Diseases |
| D008103 | Liver Cirrhosis |
| D005355 | Fibrosis |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D001798 |
| Blood Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D005916 | Globulins |