Primary Biliary CholangitisPhase 2NCT03155932
Safety, Tolerability, and Efficacy of Etrasimod (APD334) in Participants With Primary Biliary Cholangitis
A Phase 2 study testing APD334 in people with Primary Biliary Cholangitis, run by Arena Pharmaceuticals at 9 sites in 3 countries.
Trial summary
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). Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Primary Biliary Cholangitis
- What is being tested?
- APD334
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 9 sitesin 3 countries
- Study dates
- 29 Dec 2017to 31 Jan 2019
- Phase
- Phase 2
- 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 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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:
- Anti-mitochondrial antibodies (AMA) titer >1:40 on immunofluorescence or M2 positive by enzyme-linked immunosorbent assay (ELISA) or positive PBC-specific antinuclear antibodies (anti-GP210 and/or anti-SP100)
- Alkaline phosphatase (ALP) >1.5 x upper limit of normal (ULN) for at least 6 months
- Liver biopsy findings consistent with PBC
- 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 <ULN, at all screening visits
- AST, ALT, ALP, and total bilirubin must have 2 values at least 4 weeks apart that are within 20% of each other
Who cannot join
- Chronic liver disease of a non-PBC etiology. However, PBC participants accompanied with primary Sjögren's syndrome (pSS) are eligible to be enrolled.
- History or evidence of clinically significant hepatic decompensation
- Medical conditions that may cause non-hepatic increases in ALP (e.g., Paget's disease)
- Clinically significant infections within 6 weeks prior to treatment start, or infection with hepatitis C virus anytime in the past
- Immunosuppressive, immunomodulating, or investigational agents within 30 days prior to treatment start
- Treatment with obeticholic acid (OCA) within 30 days prior to Day 1
- Note: Other protocol defined Inclusion/Exclusion criteria may apply
Trial locations and contacts
9 sites in 3 countries.
9 sites in 3 countries
United States4 sites
Baylor College of Medicine
Houston, Texas, 77030
Gastroenterology and Hepatology, UC Davis Medical Center
Sacramento, California, 95817
Texas Liver Institute
San Antonio, Texas, 78215
Swedish Medical Center
Seattle, Washington, 98104
Australia3 sites
Sunshine Coast University Hospital
Birtinya, Queensland, 4575
Royal Prince Alfred Hospital
Camperdown, New South Wales, 2050
Alfred Health
Melbourne, Victoria, 3004
New Zealand2 sites
Christchurch Clinical Studies Trust
Christchurch, 8011
Auckland City Hospital
Grafton, Auckland, 1023
About this study
Official title: An Open-label, Pilot, Proof of Concept Study to Evaluate the Safety, Tolerability, and Efficacy of Oral Etrasimod (APD334) in Patients With Primary Biliary Cholangitis
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).
- Conditions
- Primary Biliary Cholangitis
- Being tested
- APD334
- Medical subject headings
- Liver Cirrhosis, Biliary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
APD334Drug
Also called etrasimod
APD334 active treatment for 24 weeks.
| Study group | Role | What they receive |
|---|---|---|
| APD334 | Experimental | APD334 active treatment for 24 weeks. Receives: Drug: APD334 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Serum Alkaline Phosphatase (ALP) Concentration
Reduction in ALP concentration is a surrogate marker of slower disease progression.
Measured: 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.
Measured: Up to Week 26
Secondary outcomes (2)
Change in Serum ALP Concentration
Reduction in ALP concentration is a surrogate marker of slower disease progression.
Measured: Baseline, Week 12
Pharmacokinetic Parameters of Etrasimod, and Its Metabolites
Measured: Up to Week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 May 2017
- Study start29 Dec 2017
- Primary completion31 Jan 2019
- Study completion31 Jan 2019
Why it stopped: Sponsor decision
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 CholangitisStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Open label
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Arena Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Arena CT.gov Administrator, Study director, Arena Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03155932
- Sponsor's protocol ID
- APD334-010
- Status last verified
- Feb 2022
- First submitted
- 11 May 2017
- Last updated
- 24 Mar 2022
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Mar 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Pilot, Proof of Concept Study to Evaluate the Safety, Tolerability, and Efficacy of Oral Etrasimod (APD334) in Patients With Primary Biliary Cholangitis
- ClinicalTrials.gov ID
- NCT03155932
- Acronym / sponsor ID
- APD334-010
- Conditions
- Primary Biliary Cholangitis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Sponsor decision
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Arena PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 29 Dec 2017 (actual)
- Primary completion
- 31 Jan 2019 (actual)
- Study completion
- 31 Jan 2019 (actual)
- First posted
- 16 May 2017 (actual)
- Results
- Posted 24 Mar 2022
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 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).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Open label
| Arm | Type | Receives |
|---|---|---|
| APD334 | Experimental | Drug: APD334APD334 active treatment for 24 weeks. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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 <ULN, at all screening visits
- AST, ALT, ALP, and total bilirubin must have 2 values at least 4 weeks apart that are within 20% of each other
Key exclusion criteria (7 listed)
- Chronic liver disease of a non-PBC etiology. However, PBC participants accompanied with primary Sjögren's syndrome (pSS) are eligible to be enrolled.
- History or evidence of clinically significant hepatic decompensation
- Medical conditions that may cause non-hepatic increases in ALP (e.g., Paget's disease)
- Clinically significant infections within 6 weeks prior to treatment start, or infection with hepatitis C virus anytime in the past
- Immunosuppressive, immunomodulating, or investigational agents within 30 days prior to treatment start
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 |
|---|---|---|
| APD334Also: etrasimodAPD334 active treatment for 24 weeks. | Drug | APD334 |
Primary outcome
Change in Serum Alkaline Phosphatase (ALP) Concentration
Time frame: Baseline, Week 24
Reduction in ALP concentration is a surrogate marker of slower disease progression.
Number of Participants With Adverse Events
Time frame: Up to Week 26
Safety was assessed by monitoring adverse events and clinically relevant changes in vital signs and clinical laboratory results.
2 secondary outcomes
Change in Serum ALP Concentration
Time frame: Baseline, Week 12
Pharmacokinetic Parameters of Etrasimod, and Its Metabolites
Time frame: Up to Week 24
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Mar 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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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.
Arena CT.gov Administrator study director
Arena Pharmaceuticals
9 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing APD334 in people with Primary Biliary Cholangitis, run by Arena Pharmaceuticals at 9 sites in 3 countries.
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 7 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: stopped early, as of the sponsor's last update on 24 Mar 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in United States, Australia, New Zealand. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 24 Mar 2022). 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?
Change in Serum Alkaline Phosphatase (ALP) Concentration (time frame: Baseline, Week 24). The study lists 2 primary outcomes in all. 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 24 Mar 2022.