RESPONSE: Response to Seladelpar in Subjects With Primary Biliary Cholangitis (PBC) and an Inadequate Control to or an Intolerance to Ursodeoxycholic Acid (UDCA)
Summary
The purposes of this study are to evaluate the treatment effect of seladelpar on composite biochemical improvement in cholestasis markers based on ALP and total bilirubin and to evaluate the safety of seladelpar over 12 months of treatment compared to placebo. The study also checked the effect of treatment on the symptoms of PBC, including pruritus.
Timeline
- Start
- 2021-04-21
- Primary completion
- 2023-08-11
- Completion
- 2023-08-11
Outcome
Met primary endpoint
paper A greater percentage of the patients in the seladelpar group than in the placebo group had a biochemical response PMID 38381664 ↗
paper biochemical response (61.7% vs. 20.0%; difference, 41.7 percentage points; 95% confidence interval [CI], 27.7 to 53.4, P<0.001). PMID 38381664 ↗
registry analysis (superiority test); Placebo vs Seladelpar; p < 0.0001; Risk Difference (RD) 41.7 (95% CI 27.7 to 53.4) NCT04620733 ↗
release “In the pivotal Phase 3 RESPONSE trial, seladelpar achieved statistical significance over placebo across primary composite endpoints” gilead.com ↗
release “RESPONSE study. In the study, 62% of participants taking seladelpar achieved the primary endpoint of composite biochemical response at month 12 compared with 20%” gilead.com ↗
Publications
- Levy C, Jones DEJ, Mayo MJ, Bowlus CL, Kowdley KV, Hirschfield GM, Liu J, Burk C, Carroll S, Crittenden DB, Kremer AE. Seladelpar Improved Itch, Itch-Related Sleep Disturbance and Measures of Fatigue in Patients With Primary Biliary Cholangitis and Pruritus in the Phase 3 RESPONSE Trial. Aliment Pharmacol Ther. 2026 Jul;64(1):23-35. doi: 10.1111/apt.70630. Epub 2026 Apr 3.
- Hirschfield GM, Bowlus CL, Mayo MJ, Kremer AE, Vierling JM, Kowdley KV, Levy C, Villamil A, Ladron de Guevara Cetina AL, Janczewska E, Zigmond E, Jeong SH, Yilmaz Y, Kallis Y, Corpechot C, Buggisch P, Invernizzi P, Londono Hurtado MC, Bergheanu S, Yang K, Choi YJ, Crittenden DB, McWherter CA; RESPONSE Study Group. A Phase 3 Trial of Seladelpar in Primary Biliary Cholangitis. N Engl J Med. 2024 Feb 29;390(9):783-794. doi: 10.1056/NEJMoa2312100. Epub 2024 Feb 21.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Seladelpar | Small molecule | 5 mg | Oral |
| Subject | Seladelpar | Small molecule | 10 mg | Oral |