drugset / Trial / NCT03890120
Study of Cilofexor in Adults With Primary Sclerosing Cholangitis
RandomizedParallel-groupDouble-blindTreatment
Summary
The primary objective of this study is to evaluate whether cilofexor reduces the risk of fibrosis progression among non-cirrhotic adults with primary sclerosing cholangitis (PSC).
Timeline
- Start
- 2019-03-27
- Primary completion
- 2022-11-10
- Completion
- 2022-12-23
Outcome
Missed primary endpoint
Stopped: “Following recommendation of the external Data Monitoring Committee, after it reviewed the results of a planned interim futility analysis.”
paper Cilofexor did not significantly reduce the rate of fibrosis progression (vs placebo) in participants with non-cirrhotic PSC. PMID 41173015 ↗
Publications
- Background Trauner M, Chung C, Sterling K, Liu X, Lu X, Xu J, Tempany-Afdhal C, Goodman ZD, Farkkila M, Tanaka A, Trivedi P, Kowdley KV, Bowlus CL, Levy C, Myers RP. PRIMIS: design of a pivotal, randomized, phase 3 study evaluating the safety and efficacy of the nonsteroidal farnesoid X receptor agonist cilofexor in noncirrhotic patients with primary sclerosing cholangitis. BMC Gastroenterol. 2023 Mar 15;23(1):75. doi: 10.1186/s12876-023-02653-2.
- Background Trauner M, Levy C, Tanaka A, Goodman Z, Thorburn D, et al. A Phase 3 Randomized, Double-blind, Placebo-controlled Study Evaluation the Efficacy and Safety of Cilofexor in Patients With Non-cirrhotic Patients With Primary Sclerosing Cholangitis. J Hepatol. 2023 June;78(S1):S12-S13.
- Trauner M, Levy C, Tanaka A, Goodman Z, Thorburn D, Joshi D, Salminen K, Yimam K, Isayama H, Montano-Loza AJ, Caldwell S, Danta M, Farkkila M, Gallegos-Orozco JF, Gordon SC, Hinrichsen H, Invernizzi P, Vuppalanchi R, Zhu K, Xu J, Liu X, Lu X, Crans G, Bolbolan S, Boyette L, Alani M, Barchuk WT, Watkins TR, Genovese MC, Bowlus CL. Cilofexor in non-cirrhotic primary sclerosing cholangitis (PRIMIS): a randomised, double-blind, multicentre, placebo-controlled, phase 3 trial. Lancet Gastroenterol Hepatol. 2026 Jan;11(1):46-58. doi: 10.1016/S2468-1253(25)00208-0. Epub 2025 Oct 28.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Cilofexor | Small molecule | 100 mg | Oral |