A Multi-part, Double Blind Study to Assess Safety, Tolerability and Efficacy of Tropifexor (LJN452) in PBC Patients

Phase 2 Completed 61 enrolled Novartis Pharmaceuticals

RandomizedParallel-groupTriple-blindTreatment

Summary

A multi-part study to assess safety, tolerability and efficacy of tropifexor (LJN452) in patients with primary biliary cholangitis

Timeline

Start: 2015-09-09
Primary completion: 2018-08-02
Completion: 2018-08-02

Outcome

Met primary endpoint

registry analysis (other test); LJN452 - 0.06 mg qd vs Pooled Placebo; p <0.001; adjusted fold change from baseline 0.47 (90% CI 0.37 to 0.60); ANCOVA NCT02516605 ↗

paper By Day 28, tropifexor caused 26-72% reduction in GGT from baseline at 30- to 150-μg doses (p <0.001 at 60-, 90-, and 150-μg tropifexor vs. placebo). PMID 36267872 ↗

Publications

Schramm C, Wedemeyer H, Mason A, Hirschfield GM, Levy C, Kowdley KV, Milkiewicz P, Janczewska E, Malova ES, Sanni J, Koo P, Chen J, Choudhury S, Klickstein LB, Badman MK, Jones D. Farnesoid X receptor agonist tropifexor attenuates cholestasis in a randomised trial in patients with primary biliary cholangitis. JHEP Rep. 2022 Jul 21;4(11):100544. doi: 10.1016/j.jhepr.2022.100544. eCollection 2022 Nov.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Tropifexor	Small molecule	30 ug	—
Subject	Tropifexor	Small molecule	60 ug	—
Subject	Tropifexor	Small molecule	90 ug	—
Subject	Tropifexor	Small molecule	150 ug	—