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Sponsor decision
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The purpose of this open-label, pilot, proof of concept study is to evaluate the safety, tolerability, and efficacy of oral etrasimod (APD334) in participants with primary biliary cholangitis (PBC).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| APD334 | Experimental | APD334 active treatment for 24 weeks. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| APD334 | Drug | APD334 active treatment for 24 weeks. |
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| Measure | Description | Time Frame |
|---|---|---|
| Change in Serum Alkaline Phosphatase (ALP) Concentration | Reduction in ALP concentration is a surrogate marker of slower disease progression. | Baseline, Week 24 |
| Number of Participants With Adverse Events | Safety was assessed by monitoring adverse events and clinically relevant changes in vital signs and clinical laboratory results. | Up to Week 26 |
| Measure | Description | Time Frame |
|---|---|---|
| Change in Serum ALP Concentration | Reduction in ALP concentration is a surrogate marker of slower disease progression. | Baseline, Week 12 |
| Pharmacokinetic Parameters of Etrasimod, and Its Metabolites |
| Measure | Description | Time Frame |
|---|---|---|
| Exploratory - Change in Complete Blood Count | Baseline, Week 12, Week 24 | |
| Exploratory - Change in Incidence of Fatigue as Assessed by Peripheral Biliary Cholangitis (PBC-40) Scale | Baseline, Week 12, Week 24 |
Key Inclusion Criteria:
Males or females aged 18 to 80 years (inclusive) at the time of screening, with confirmed Primary Biliary Cholangitis (PBC) diagnosis based upon at least 2 of 3 criteria:
Use of ursodeoxycholic acid (UDCA) for at least 6 months prior to screening (stable dose for at least 3 months immediately prior to screening)
Participants must have ALP >1.5 x ULN but <10 x ULN, alanine aminotransferase (ALT) and aspartate aminotransferase (AST) <5 x ULN, and total bilirubin \
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| Name | Affiliation | Role |
|---|---|---|
| Arena CT.gov Administrator | Arena Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Gastroenterology and Hepatology, UC Davis Medical Center | Sacramento | California | 95817 | United States | ||
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Enrollment was planned for up to 20 participants. Two participants were enrolled into the study and completed 24 weeks of treatment.
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| ID | Title | Description |
|---|---|---|
| FG000 | 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. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| 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 | Jun 27, 2018 |
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Open label
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| Up to Week 24 |
| Exploratory - Change in Incidence of Pruritus as Assessed by 5-Dimensions (5-D) Itch Scale | Baseline, Week 12, Week 24 |
| Exploratory - Change in Schirmer Test Outcome | Baseline, Week 12, Week 24 |
| Exploratory - Change in Tear Film Break-Up Time | Baseline, Week 12, Week 24 |
| Exploratory - Change in Concentration of Serum High Sensitivity C-Reactive Protein (hsCRP) | Baseline, Week 12, Week 24 |
| Exploratory - Change in Concentration of Serum Alanine Transaminase (ALT) | Baseline, Week 12, Week 24 |
| Exploratory - Change in Concentration of Serum Aspartate Transaminase (AST) | Baseline, Week 12, Week 24 |
| Exploratory - Change in Concentration of Serum Gamma-Glutamyl Transferase (GGT) | Baseline, Week 12, Week 24 |
| Exploratory - Change in Concentration of Serum C4 | Baseline, Week 12, Week 24 |
| Exploratory - Change in Concentration of Serum Immunoglobulin | Baseline, Week 12, Week 24 |
| Exploratory - Change in Concentration of Serum GP73 | Baseline, Week 12, Week 24 |
| Exploratory - Change in Concentration of Serum Anti-Mitochondrial Antibodies (AMA) | Baseline, Week 12, Week 24 |
| Exploratory - Change in Quality of Life | Baseline, Week 12, Week 24 |
| Baylor College of Medicine |
| Houston |
| Texas |
| 77030 |
| United States |
| Texas Liver Institute | San Antonio | Texas | 78215 | United States |
| Swedish Medical Center | Seattle | Washington | 98104 | United States |
| Royal Prince Alfred Hospital | Camperdown | New South Wales | 2050 | Australia |
| Sunshine Coast University Hospital | Birtinya | Queensland | 4575 | Australia |
| Alfred Health | Melbourne | Victoria | 3004 | Australia |
| Auckland City Hospital | Grafton | Auckland | 1023 | New Zealand |
| Christchurch Clinical Studies Trust | Christchurch | 8011 | New Zealand |
| Participants Escalated to 2 mg Dose of APD334 at Week 12 to Week 24 |
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| COMPLETED |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | 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. |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
| ||||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| 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 | Change in Serum Alkaline Phosphatase (ALP) Concentration | Reduction in ALP concentration is a surrogate marker of slower disease progression. | 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. | Posted | Baseline, Week 24 |
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| |||||||||||||||||||
| Primary | Number of Participants With Adverse Events | Safety was assessed by monitoring adverse events and clinically relevant changes in vital signs and clinical laboratory results. | Two participants who were enrolled and received treatment in this study. | Posted | Count of Participants | Participants | Up to Week 26 |
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| Secondary | Change in Serum ALP Concentration | Reduction in ALP concentration is a surrogate marker of slower disease progression. | 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. | Posted | Baseline, Week 12 |
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| Secondary | Pharmacokinetic Parameters of Etrasimod, and Its Metabolites | 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. | Posted | Up to Week 24 |
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| Other Pre-specified | Exploratory - Change in Complete Blood Count | Not Posted | Baseline, Week 12, Week 24 | Participants | ||||||||||||||||||||||
| Other Pre-specified | Exploratory - Change in Incidence of Fatigue as Assessed by Peripheral Biliary Cholangitis (PBC-40) Scale | Not Posted | Baseline, Week 12, Week 24 | Participants | ||||||||||||||||||||||
| Other Pre-specified | Exploratory - Change in Incidence of Pruritus as Assessed by 5-Dimensions (5-D) Itch Scale | Not Posted | Baseline, Week 12, Week 24 | Participants | ||||||||||||||||||||||
| Other Pre-specified | Exploratory - Change in Schirmer Test Outcome | Not Posted | Baseline, Week 12, Week 24 | Participants | ||||||||||||||||||||||
| Other Pre-specified | Exploratory - Change in Tear Film Break-Up Time | Not Posted | Baseline, Week 12, Week 24 | Participants | ||||||||||||||||||||||
| Other Pre-specified | Exploratory - Change in Concentration of Serum High Sensitivity C-Reactive Protein (hsCRP) | Not Posted | Baseline, Week 12, Week 24 | Participants | ||||||||||||||||||||||
| Other Pre-specified | Exploratory - Change in Concentration of Serum Alanine Transaminase (ALT) | Not Posted | Baseline, Week 12, Week 24 | Participants | ||||||||||||||||||||||
| Other Pre-specified | Exploratory - Change in Concentration of Serum Aspartate Transaminase (AST) | Not Posted | Baseline, Week 12, Week 24 | Participants | ||||||||||||||||||||||
| Other Pre-specified | Exploratory - Change in Concentration of Serum Gamma-Glutamyl Transferase (GGT) | Not Posted | Baseline, Week 12, Week 24 | Participants | ||||||||||||||||||||||
| Other Pre-specified | Exploratory - Change in Concentration of Serum C4 | Not Posted | Baseline, Week 12, Week 24 | Participants | ||||||||||||||||||||||
| Other Pre-specified | Exploratory - Change in Concentration of Serum Immunoglobulin | Not Posted | Baseline, Week 12, Week 24 | Participants | ||||||||||||||||||||||
| Other Pre-specified | Exploratory - Change in Concentration of Serum GP73 | Not Posted | Baseline, Week 12, Week 24 | Participants | ||||||||||||||||||||||
| Other Pre-specified | Exploratory - Change in Concentration of Serum Anti-Mitochondrial Antibodies (AMA) | Not Posted | Baseline, Week 12, Week 24 | Participants | ||||||||||||||||||||||
| Other Pre-specified | Exploratory - Change in Quality of Life | Not Posted | Baseline, Week 12, Week 24 | Participants |
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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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 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. | 0 | 2 | 0 | 2 | 2 | 2 |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Skin rash | Skin and subcutaneous tissue disorders | MedDra 20.0 | Systematic Assessment |
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| Urinary tract infection | Infections and infestations | MedDra 20.0 | Systematic Assessment |
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| Worsening right hip pain | Musculoskeletal and connective tissue disorders | MedDra 20.0 | Systematic Assessment |
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| Elevated gamma-glutamyl transferase-worsening | Investigations | MedDra 20.0 | Systematic Assessment |
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| Loose stools | Gastrointestinal disorders | MedDra 20.0 | Systematic Assessment |
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| Fatigue | General disorders | MedDra 20.0 | Systematic Assessment |
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| Common cold - Upper respiratory tract infection | Infections and infestations | MedDra 20.0 | Systematic Assessment |
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| Elevated alkaline phosphatase-worsening | Investigations | MedDra 20.0 | Systematic Assessment |
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| Lower abdominal cramps | Gastrointestinal disorders | MedDra 20.0 | Systematic Assessment |
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| Pruritus | Skin and subcutaneous tissue disorders | MedDra 20.0 | Systematic Assessment |
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| Cognitive defects | Nervous system disorders | MedDra 20.0 | Systematic Assessment |
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| Olecranon bursitis | Musculoskeletal and connective tissue disorders | MedDra 20.0 | Systematic Assessment |
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| Back pain | Musculoskeletal and connective tissue disorders | MedDra 20.0 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Arena CT.gov Administrator | Arena Pharmaceuticals, Inc. | +1 855-218-9153 | ct.gov@arenapharm.com |
| Feb 25, 2022 |
| Prot_SAP_001.pdf |
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| ID | Term |
|---|---|
| D008105 | Liver Cirrhosis, Biliary |
| ID | Term |
|---|---|
| D002780 | Cholestasis, Intrahepatic |
| D002779 | Cholestasis |
| D001649 | Bile Duct Diseases |
| D001660 | Biliary Tract Diseases |
| D004066 | Digestive System Diseases |
| D008107 | Liver Diseases |
| D008103 | Liver Cirrhosis |
| D005355 | Fibrosis |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
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| ID | Term |
|---|---|
| C000656249 | etrasimod |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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