Trial Outcomes & Findings for Study of Abatacept (Orencia) to Treat Primary Biliary Cirrhosis (NCT NCT02078882)

Results Overview

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

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

16 participants

Primary outcome timeframe

Week 24

Results posted on

2020-04-09

Participant Flow

Participant milestones

Participant milestones
Measure	Abatacept 125 mg Weekly Open label treatment with Abatacept abatacept: 125 mg subcutaneously each week for 24 weeks
Overall Study STARTED	16
Overall Study COMPLETED	16
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Study of Abatacept (Orencia) to Treat Primary Biliary Cirrhosis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Abatacept 125 mg Weekly n=16 Participants Open label treatment with Abatacept abatacept: 125 mg subcutaneously each week for 24 weeks
Age, Categorical <=18 years	0 Participants n=39 Participants
Age, Categorical Between 18 and 65 years	14 Participants n=39 Participants
Age, Categorical >=65 years	2 Participants n=39 Participants
Age, Continuous	52 years n=39 Participants
Sex: Female, Male Female	15 Participants n=39 Participants
Sex: Female, Male Male	1 Participants n=39 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	3 Participants n=39 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	13 Participants n=39 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=39 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=39 Participants
Race (NIH/OMB) Asian	0 Participants n=39 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=39 Participants
Race (NIH/OMB) Black or African American	0 Participants n=39 Participants
Race (NIH/OMB) White	16 Participants n=39 Participants
Race (NIH/OMB) More than one race	0 Participants n=39 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=39 Participants
Region of Enrollment United States	16 participants n=39 Participants

PRIMARY outcome

Timeframe: Week 24

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

Outcome measures

Outcome measures
Measure	Abatacept 125 mg Weekly n=16 Participants Open label treatment with Abatacept abatacept: 125 mg subcutaneously each week for 24 weeks
Biochemical Response	1 Participants

SECONDARY outcome

Timeframe: Weeks 2, 4, 12, 24, and 36

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

Outcome measures

Outcome measures
Measure	Abatacept 125 mg Weekly n=16 Participants Open label treatment with Abatacept abatacept: 125 mg subcutaneously each week for 24 weeks
Drug Safety	4 Participants

SECONDARY outcome

Timeframe: Week 24

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

Outcome measures

Outcome measures
Measure	Abatacept 125 mg Weekly n=16 Participants Open label treatment with Abatacept abatacept: 125 mg subcutaneously each week for 24 weeks
Absolute Change in Alkaline Phosphatase	-2.8 IU/L Interval -97.5 to 42.0

SECONDARY outcome

Timeframe: Week 24

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

Outcome measures

Outcome measures
Measure	Abatacept 125 mg Weekly n=16 Participants Open label treatment with Abatacept abatacept: 125 mg subcutaneously each week for 24 weeks
Absolute Change in Alanine Transferase (ALT)	0.5 IU/L Interval -13.5 to 4.5

SECONDARY outcome

Timeframe: Week 24

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

Outcome measures

Outcome measures
Measure	Abatacept 125 mg Weekly n=16 Participants Open label treatment with Abatacept abatacept: 125 mg subcutaneously each week for 24 weeks
Liver Stiffness Measured by Magnetic Resonance Elastography	-0.1 kPa Interval -0.43 to 0.2

SECONDARY outcome

Timeframe: Week 24

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.

Outcome measures

Outcome measures
Measure	Abatacept 125 mg Weekly n=16 Participants Open label treatment with Abatacept abatacept: 125 mg subcutaneously each week for 24 weeks
Primary Billiary Cholangitis Quality of Life	0 units on a scale Interval -4.0 to 13.0

SECONDARY outcome

Timeframe: Week 24

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

Outcome measures

Outcome measures
Measure	Abatacept 125 mg Weekly n=16 Participants Open label treatment with Abatacept abatacept: 125 mg subcutaneously each week for 24 weeks
Percent Change in Alkaline Phosphatase	0.01 percent change Interval -0.28 to 0.13

SECONDARY outcome

Timeframe: Week 24

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

Outcome measures

Outcome measures
Measure	Abatacept 125 mg Weekly n=16 Participants Open label treatment with Abatacept abatacept: 125 mg subcutaneously each week for 24 weeks
Percent Change in Alanine Transferase (ALT)	0.01 percent change Interval -0.29 to 0.11

OTHER_PRE_SPECIFIED outcome

Timeframe: Week 24

Change in IgM level from Day 0 to Week 24

Outcome measures

Outcome measures
Measure	Abatacept 125 mg Weekly n=16 Participants Open label treatment with Abatacept abatacept: 125 mg subcutaneously each week for 24 weeks
Immunoglobulin M (IgM) Levels	0.0 mg/dL Interval -44.0 to 45.0

OTHER_PRE_SPECIFIED outcome

Timeframe: Week 24

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

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Day 0 and Weeks 4, 12, 24, and 36

Trough serum levels of abatacept

Outcome measures

Outcome data not reported

Adverse Events

Abatacept 125 mg Weekly

Serious events: 0 serious events

Other events: 4 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Abatacept 125 mg Weekly n=16 participants at risk Open label treatment with Abatacept abatacept: 125 mg subcutaneously each week for 24 weeks
Gastrointestinal disorders Nausea	12.5% 2/16 • Number of events 2 • 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.
Gastrointestinal disorders Vomiting	6.2% 1/16 • Number of events 1 • 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.
Gastrointestinal disorders Elevated liver enzymes	6.2% 1/16 • Number of events 1 • 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.
Gastrointestinal disorders Right upper quadrant pain	6.2% 1/16 • Number of events 1 • 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.
Infections and infestations Upper respiratory infection	6.2% 1/16 • Number of events 1 • 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.
Infections and infestations Urinary Tract Infections	6.2% 1/16 • Number of events 1 • 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.
Skin and subcutaneous tissue disorders Urticarial Rash	6.2% 1/16 • Number of events 1 • 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.
Cardiac disorders Chest Pain	6.2% 1/16 • Number of events 1 • 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.
Blood and lymphatic system disorders Hilar Adenopathy	6.2% 1/16 • Number of events 1 • 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.

Additional Information

Christopher L Bowlus

University of California Davis

Phone: 9167348696

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place