NCT03155932

Safety, Tolerability, and Efficacy of Etrasimod (APD334) in Participants With Primary Biliary Cholangitis

Terminated Phase 2 Results posted Last updated 24 March 2022

What this trial tests

Phase 2 trial testing APD334 in Primary Biliary Cholangitis in 2 participants. Terminated before completion.

Timeline

29 December 2017

Primary endpoint
31 January 2019

31 January 2019

Quick facts

Lead sponsor	Arena Pharmaceuticals
Phase	Phase 2
Status	Terminated
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	2
Start date	29 December 2017
Primary completion	31 January 2019
Estimated completion	31 January 2019
Sites	9 locations across New Zealand, United States, Australia

Drugs / interventions tested

• APD334 — full drug profile →

Conditions studied

• Primary Biliary Cholangitis — all drugs for Primary Biliary Cholangitis →

Sponsor

Arena Pharmaceuticals — full company profile →

Who can join

Adults 18 to 80, any sex, with Primary Biliary Cholangitis. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Number of Participants With Adverse Events Primary · Up to Week 26

Safety was assessed by monitoring adverse events and clinically relevant changes in vital signs and clinical laboratory results.

Group	Value	95% CI
APD334	2

Adverse events — posted to ClinicalTrials.gov

Time frame: Up to Week 26. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

APD334

Serious: 0/2 (0%)

Deaths: 0/2

Other adverse events (13 terms — click to expand)

Reaction	System	APD334
Skin rash	Skin and subcutaneous tissue disorders	—
Urinary tract infection	Infections and infestations	—
Worsening right hip pain	Musculoskeletal and connective tissue disorders	—
Elevated gamma-glutamyl transferase-worsening	Investigations	—
Loose stools	Gastrointestinal disorders	—
Fatigue	General disorders	—
Common cold - Upper respiratory tract infection	Infections and infestations	—
Elevated alkaline phosphatase-worsening	Investigations	—
Lower abdominal cramps	Gastrointestinal disorders	—
Pruritus	Skin and subcutaneous tissue disorders	—
Cognitive defects	Nervous system disorders	—
Olecranon bursitis	Musculoskeletal and connective tissue disorders	—
Back pain	Musculoskeletal and connective tissue disorders	—

Data from ClinicalTrials.gov NCT03155932 adverse events section.

Sponsor's own description

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).

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Targeting Sphingosine-1-Phosphate Signaling in Immune-Mediated Diseases: Beyond Multiple Sclerosis.
   Pérez-Jeldres T, Alvarez-Lobos M, Rivera-Nieves J. · · 2021 · cited 150× · PMID 33983615 · DOI 10.1007/s40265-021-01528-8
2. The S1P-S1PR Axis in Neurological Disorders-Insights into Current and Future Therapeutic Perspectives.
   Lucaciu A, Brunkhorst R, Pfeilschifter JM, Pfeilschifter W, et al · · 2020 · cited 53× · PMID 32580348 · DOI 10.3390/cells9061515
3. Fibrotic Events in the Progression of Cholestatic Liver Disease.
   Wu H, Chen C, Ziani S, Nelson LJ, et al · · 2021 · cited 36× · PMID 34062960 · DOI 10.3390/cells10051107
4. Management of Primary Biliary Cholangitis: Current Treatment and Future Perspectives.
   Barba Bernal R, Ferrigno B, Medina Morales E, Castro CM, et al · · 2023 · cited 26× · PMID 36843300 · DOI 10.5152/tjg.2023.22239
5. New treatments/targets for primary biliary cholangitis.
   Corpechot C, Poupon R, Chazouillères O. · · 2019 · cited 20× · PMID 32039371 · DOI 10.1016/j.jhepr.2019.05.005
6. Targeting Chronic Inflammation of the Digestive System in Cancer Prevention: Modulators of the Bioactive Sphingolipid Sphingosine-1-Phosphate Pathway.
   McGowan EM, Lin Y, Chen S. · · 2022 · cited 19× · PMID 35158806 · DOI 10.3390/cancers14030535
7. Sphingolipids in Lung Pathology in the Coronavirus Disease Era: A Review of Sphingolipid Involvement in the Pathogenesis of Lung Damage.
   Khan SA, Goliwas KF, Deshane JS. · · 2021 · cited 17× · PMID 34690821 · DOI 10.3389/fphys.2021.760638
8. The Management of Cholestatic Liver Diseases: Current Therapies and Emerging New Possibilities.
   Mazzetti M, Marconi G, Mancinelli M, Benedetti A, et al · · 2021 · cited 17× · PMID 33919600 · DOI 10.3390/jcm10081763