Open Label Study to Evaluate Safety and Efficacy of LUM001 in Patients With Primary Sclerosing Cholangitis

NCT02061540 ↗

Phase 2 Completed 27 enrolled Mirum Pharmaceuticals, Inc.

NaSingle-groupOpen-labelTreatment

Summary

The study is an open-label study in adults with primary sclerosing cholangitis to evaluate the safety, tolerability, and effect of 14-weeks of daily dosing of LUM001.

Timeline

Start: 2014-03
Primary completion: 2016-02
Completion: 2016-02

Outcome

Met primary endpoint

registry p = 0.0043; Mean Difference (Net) -14.841 (95% CI -27.251 to -2.432); Wilcoxon's signed rank test NCT02061540 ↗

Publications

Bowlus CL, Eksteen B, Cheung AC, Thorburn D, Moylan CA, Pockros PJ, Forman LM, Dorenbaum A, Hirschfield GM, Kennedy C, Jaecklin T, McKibben A, Chien E, Baek M, Vig P, Levy C. Safety, tolerability, and efficacy of maralixibat in adults with primary sclerosing cholangitis: Open-label pilot study. Hepatol Commun. 2023 May 15;7(6):e0153. doi: 10.1097/HC9.0000000000000153. eCollection 2023 Jun 1.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Maralixibat	Small molecule	10 mg	Oral