COVID-19 studies are our top priority. For all other trials, there is a 4-week delay in processing a trial submitted to the ANZCTR and additional delays for updates of registered trials. We appreciate your patience.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03155932




Registration number
NCT03155932
Ethics application status
Date submitted
11/05/2017
Date registered
16/05/2017
Date last updated
7/02/2020

Titles & IDs
Public title
Safety, Tolerability, and Efficacy of Etrasimod (APD334) in Patients With Primary Biliary Cholangitis
Scientific 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
Secondary ID [1] 0 0
APD334-010
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Primary Biliary Cholangitis 0 0
Condition category
Condition code
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Infection 0 0 0 0
Other infectious diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - APD334

Experimental: APD334 - APD334 active treatment for 24 weeks.


Treatment: Drugs: APD334
APD334 active treatment for 24 weeks.

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change from baseline to Week 24 in serum ALP concentration. - Assess the change in ALP concentration following administration of APD334.
Timepoint [1] 0 0
Week 24
Primary outcome [2] 0 0
Number of patients with adverse events and abnormal clinical laboratory tests (including hematology, serum chemistry, coagulation and urinalysis). - Safety and tolerability of APD334
Timepoint [2] 0 0
Week 26
Secondary outcome [1] 0 0
Change from baseline to Week 12 in serum ALP concentration. - Assess the change in ALP concentration following administration of APD334.
Timepoint [1] 0 0
Week 12
Secondary outcome [2] 0 0
Change from baseline to Week 12 and 24 in complete blood counts
Timepoint [2] 0 0
Weeks 12 and 24
Secondary outcome [3] 0 0
Change from baseline to Week 12 and 24 in quality of life - measured by the PBC-40 scale
Timepoint [3] 0 0
Weeks 12 and 24
Secondary outcome [4] 0 0
Change from baseline to Week 12 and 24 in fatigue - measured by the PBC-40 scale
Timepoint [4] 0 0
Weeks 12 and 24
Secondary outcome [5] 0 0
Change from baseline to Week 12 and 24 in pruritus - measured by the 5-D scale
Timepoint [5] 0 0
Weeks 12 and 24
Secondary outcome [6] 0 0
Change from baseline to Week 12 and 24 in Schirmer test outcome - Schirmer test in patients with abnormal results at screening
Timepoint [6] 0 0
Weeks 12 and 24
Secondary outcome [7] 0 0
Change from baseline to Week 12 and 24 in tear film break-up time - Tear film break-up time in patients with abnormal results at screening
Timepoint [7] 0 0
Weeks 12 and 24
Secondary outcome [8] 0 0
Change from baseline to Week 12 and 24 in concentration of serum HsCRP
Timepoint [8] 0 0
Weeks 12 and 24
Secondary outcome [9] 0 0
Change from baseline to Week 12 and 24 in concentration of serum ALT
Timepoint [9] 0 0
Weeks 12 and 24
Secondary outcome [10] 0 0
Change from baseline to Week 12 and 24 in concentration of serum AST
Timepoint [10] 0 0
Weeks 12 and 24
Secondary outcome [11] 0 0
Change from baseline to Week 12 and 24 in concentration of serum GGT
Timepoint [11] 0 0
Weeks 12 and 24
Secondary outcome [12] 0 0
Change from baseline to Week 12 and 24 in concentration of serum C4
Timepoint [12] 0 0
Weeks 12 and 24
Secondary outcome [13] 0 0
Change from baseline to Week 12 and 24 in concentration of serum immunoglobulin
Timepoint [13] 0 0
Weeks 12 and 24
Secondary outcome [14] 0 0
Change from baseline to Week 12 and 24 in concentration of serum GP73
Timepoint [14] 0 0
Weeks 12 and 24
Secondary outcome [15] 0 0
Change from baseline to Week 12 and 24 in concentration of serum AMA
Timepoint [15] 0 0
Weeks 12 and 24

Eligibility
Key inclusion criteria
Key

- Males or females aged 18 to 80 years (inclusive) at the time of screening, with
confirmed PBC diagnosis based upon at least 2 of 3 criteria:

- 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)

- ALP >1.5 x ULN for at least 6 months

- Liver biopsy findings consistent with PBC

- Use of UDCA for at least 6 months prior to screening (stable dose for at least 3
months immediately prior to screening)

- Patients must have ALP >1.5 x ULN but <10 x ULN, ALT and 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
Minimum age
18 Years
Maximum age
80 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Chronic liver disease of a non-PBC etiology. However, PBC patients 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 OCA within 30 days prior to Day 1

Note: Other protocol defined Inclusion/Exclusion criteria may apply

Study design
Purpose of the study
Treatment
Allocation to intervention
N/A
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Terminated
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
Recruitment hospital [1] 0 0
Royal Prince Alfred Hospital - Camperdown
Recruitment hospital [2] 0 0
Sunshine Coast University Hospital - Birtinya
Recruitment hospital [3] 0 0
Alfred Health - Melbourne
Recruitment postcode(s) [1] 0 0
- Camperdown
Recruitment postcode(s) [2] 0 0
- Birtinya
Recruitment postcode(s) [3] 0 0
3004 - Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Texas
Country [3] 0 0
United States of America
State/province [3] 0 0
Washington
Country [4] 0 0
New Zealand
State/province [4] 0 0
Auckland
Country [5] 0 0
New Zealand
State/province [5] 0 0
Christchurch

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Arena Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief 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 patients with primary biliary
cholangitis (PBC).
Trial website
https://clinicaltrials.gov/show/NCT03155932
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT03155932