Trial Outcomes & Findings for Safety, Tolerability, and Efficacy of Etrasimod (APD334) in Participants With Primary Biliary Cholangitis (NCT NCT03155932)
NCT ID: NCT03155932
Last Updated: 2022-03-24
Results Overview
Reduction in ALP concentration is a surrogate marker of slower disease progression.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
2 participants
Primary outcome timeframe
Baseline, Week 24
Results posted on
2022-03-24
Participant Flow
Enrollment was planned for up to 20 participants. Two participants were enrolled into the study and completed 24 weeks of treatment.
Participant milestones
| Measure |
APD334
Participants received APD334 1 mg tablet once daily (qd) by mouth for 12 weeks. The dose for APD334 was escalated to 2 mg at Week 12 to Week 24, based on the participant's safety and pharmacokinetic (PK) data.
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|---|---|
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Overall Study
STARTED
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2
|
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Overall Study
Participants Escalated to 2 mg Dose of APD334 at Week 12 to Week 24
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2
|
|
Overall Study
COMPLETED
|
2
|
|
Overall Study
NOT COMPLETED
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0
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Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Safety, Tolerability, and Efficacy of Etrasimod (APD334) in Participants With Primary Biliary Cholangitis
Baseline characteristics by cohort
| Measure |
APD334
n=2 Participants
Participants received APD334 1 mg tablet once daily (qd) by mouth for 12 weeks. The dose for APD334 was escalated to 2 mg at Week 12 to Week 24, based on the participant's safety and pharmacokinetic (PK) data.
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|---|---|
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Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
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Age, Categorical
Between 18 and 65 years
|
2 Participants
n=5 Participants
|
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Age, Categorical
>=65 years
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0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
NA Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
NA Participants
n=5 Participants
|
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Race (NIH/OMB)
American Indian or Alaska Native
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NA Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
NA Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
NA Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
NA Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
NA Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
NA Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
NA Participants
n=5 Participants
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PRIMARY outcome
Timeframe: Baseline, Week 24Population: Analyses were not conducted due to low enrollment (N=2). In order to protect participant's privacy, the results from the enrolled participants cannot be reported.
Reduction in ALP concentration is a surrogate marker of slower disease progression.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Up to Week 26Population: Two participants who were enrolled and received treatment in this study.
Safety was assessed by monitoring adverse events and clinically relevant changes in vital signs and clinical laboratory results.
Outcome measures
| Measure |
APD334
n=2 Participants
Participants received APD334 1 mg tablet once daily (qd) by mouth for 12 weeks. The dose for APD334 was escalated to 2 mg at Week 12 to Week 24, based on the participant's safety and pharmacokinetic (PK) data.
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|---|---|
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Number of Participants With Adverse Events
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2 Participants
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SECONDARY outcome
Timeframe: Baseline, Week 12Population: Analyses were not conducted due to low enrollment (N=2). In order to protect participant's privacy, the results from the enrolled participants cannot be reported.
Reduction in ALP concentration is a surrogate marker of slower disease progression.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Up to Week 24Population: Analyses were not conducted due to low enrollment (N=2). In order to protect participant's privacy, the results from the enrolled participants cannot be reported.
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline, Week 12, Week 24Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline, Week 12, Week 24Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline, Week 12, Week 24Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline, Week 12, Week 24Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline, Week 12, Week 24Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline, Week 12, Week 24Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline, Week 12, Week 24Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline, Week 12, Week 24Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline, Week 12, Week 24Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline, Week 12, Week 24Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline, Week 12, Week 24Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline, Week 12, Week 24Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline, Week 12, Week 24Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline, Week 12, Week 24Outcome measures
Outcome data not reported
Adverse Events
APD334
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
APD334
n=2 participants at risk
Participants received APD334 1 mg tablet once daily (qd) by mouth for 12 weeks. The dose for APD334 was escalated to 2 mg at Week 12 to Week 24, based on the participant's safety and pharmacokinetic (PK) data.
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|---|---|
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Skin and subcutaneous tissue disorders
Skin rash
|
50.0%
1/2 • Up to Week 26
A TEAE was defined as any adverse events (AE) that were reported following study treatment administration and up to 2 weeks after the last treatment intake. An SAE was any untoward medical occurrence that at any dose resulted in the following outcomes: death, was life-threatening, required/prolonged hospitalization, disability/incapacity, congenital anomaly/birth defect, and important medical events.
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Infections and infestations
Urinary tract infection
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50.0%
1/2 • Up to Week 26
A TEAE was defined as any adverse events (AE) that were reported following study treatment administration and up to 2 weeks after the last treatment intake. An SAE was any untoward medical occurrence that at any dose resulted in the following outcomes: death, was life-threatening, required/prolonged hospitalization, disability/incapacity, congenital anomaly/birth defect, and important medical events.
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Musculoskeletal and connective tissue disorders
Worsening right hip pain
|
50.0%
1/2 • Up to Week 26
A TEAE was defined as any adverse events (AE) that were reported following study treatment administration and up to 2 weeks after the last treatment intake. An SAE was any untoward medical occurrence that at any dose resulted in the following outcomes: death, was life-threatening, required/prolonged hospitalization, disability/incapacity, congenital anomaly/birth defect, and important medical events.
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Investigations
Elevated gamma-glutamyl transferase-worsening
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50.0%
1/2 • Up to Week 26
A TEAE was defined as any adverse events (AE) that were reported following study treatment administration and up to 2 weeks after the last treatment intake. An SAE was any untoward medical occurrence that at any dose resulted in the following outcomes: death, was life-threatening, required/prolonged hospitalization, disability/incapacity, congenital anomaly/birth defect, and important medical events.
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Gastrointestinal disorders
Loose stools
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50.0%
1/2 • Up to Week 26
A TEAE was defined as any adverse events (AE) that were reported following study treatment administration and up to 2 weeks after the last treatment intake. An SAE was any untoward medical occurrence that at any dose resulted in the following outcomes: death, was life-threatening, required/prolonged hospitalization, disability/incapacity, congenital anomaly/birth defect, and important medical events.
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General disorders
Fatigue
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50.0%
1/2 • Up to Week 26
A TEAE was defined as any adverse events (AE) that were reported following study treatment administration and up to 2 weeks after the last treatment intake. An SAE was any untoward medical occurrence that at any dose resulted in the following outcomes: death, was life-threatening, required/prolonged hospitalization, disability/incapacity, congenital anomaly/birth defect, and important medical events.
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Infections and infestations
Common cold - Upper respiratory tract infection
|
50.0%
1/2 • Up to Week 26
A TEAE was defined as any adverse events (AE) that were reported following study treatment administration and up to 2 weeks after the last treatment intake. An SAE was any untoward medical occurrence that at any dose resulted in the following outcomes: death, was life-threatening, required/prolonged hospitalization, disability/incapacity, congenital anomaly/birth defect, and important medical events.
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Investigations
Elevated alkaline phosphatase-worsening
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50.0%
1/2 • Up to Week 26
A TEAE was defined as any adverse events (AE) that were reported following study treatment administration and up to 2 weeks after the last treatment intake. An SAE was any untoward medical occurrence that at any dose resulted in the following outcomes: death, was life-threatening, required/prolonged hospitalization, disability/incapacity, congenital anomaly/birth defect, and important medical events.
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Gastrointestinal disorders
Lower abdominal cramps
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50.0%
1/2 • Up to Week 26
A TEAE was defined as any adverse events (AE) that were reported following study treatment administration and up to 2 weeks after the last treatment intake. An SAE was any untoward medical occurrence that at any dose resulted in the following outcomes: death, was life-threatening, required/prolonged hospitalization, disability/incapacity, congenital anomaly/birth defect, and important medical events.
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Skin and subcutaneous tissue disorders
Pruritus
|
50.0%
1/2 • Up to Week 26
A TEAE was defined as any adverse events (AE) that were reported following study treatment administration and up to 2 weeks after the last treatment intake. An SAE was any untoward medical occurrence that at any dose resulted in the following outcomes: death, was life-threatening, required/prolonged hospitalization, disability/incapacity, congenital anomaly/birth defect, and important medical events.
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Nervous system disorders
Cognitive defects
|
50.0%
1/2 • Up to Week 26
A TEAE was defined as any adverse events (AE) that were reported following study treatment administration and up to 2 weeks after the last treatment intake. An SAE was any untoward medical occurrence that at any dose resulted in the following outcomes: death, was life-threatening, required/prolonged hospitalization, disability/incapacity, congenital anomaly/birth defect, and important medical events.
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Musculoskeletal and connective tissue disorders
Olecranon bursitis
|
50.0%
1/2 • Up to Week 26
A TEAE was defined as any adverse events (AE) that were reported following study treatment administration and up to 2 weeks after the last treatment intake. An SAE was any untoward medical occurrence that at any dose resulted in the following outcomes: death, was life-threatening, required/prolonged hospitalization, disability/incapacity, congenital anomaly/birth defect, and important medical events.
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Musculoskeletal and connective tissue disorders
Back pain
|
50.0%
1/2 • Up to Week 26
A TEAE was defined as any adverse events (AE) that were reported following study treatment administration and up to 2 weeks after the last treatment intake. An SAE was any untoward medical occurrence that at any dose resulted in the following outcomes: death, was life-threatening, required/prolonged hospitalization, disability/incapacity, congenital anomaly/birth defect, and important medical events.
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Additional Information
Arena CT.gov Administrator
Arena Pharmaceuticals, Inc.
Phone: +1 855-218-9153
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place