CI

At a glance

ClinicalIndex Comparison Record
Phase 3Completed· 84 enrolled / 84 target
Drug / intervention
Maralixibatdrug
Likely dose
Maralixibat 600 microgramfrom record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT04185363
NCT04185363Phase 3CompletedMonitor (1.1/mo)Completion was 14mo ago
CompareCT.gov

An Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in the Treatment of Subjects With Progressive Familial Intrahepatic Cholestasis (PFIC)

Mirum Pharmaceuticals, Inc.·interventional·Posted Dec 4, 2019·Updated Jun 23, 2026

In Brief

A Phase 3 clinical trial evaluating Maralixibat for Progressive Familial Intrahepatic Cholestasis (PFIC). Completed, enrolled 84 participants across 28 sites in 16 countries.

Signals

Enrolling slower than its timeline implies

Detailed Summary

The primary objective of this open label extension study is to evaluate the long-term safety and tolerability of maralixibat.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsProgressive Familial Intrahepatic Cholestasis (PFIC)
CountriesArgentina, Austria, Belgium, Brazil, Canada, Colombia, France, Germany, Italy, Lebanon, Mexico, Poland, Singapore, Turkey (Türkiye), United Kingdom, United States
Collaborators--

Timeline

Phase 3CompletedFinished
2020202120222023202420252026
First PostedDec 4, 2019
Enrollment StartJan 8, 2020
Primary CompletionApr 23, 2025
TodayJul 2, 2026
Enrollment to primary: 5.3 yearsPosted 6.6 years ago

Arms & Interventions

Maralixibatexperimental

All subjects will receive Maralixibat oral solution

Drug: Maralixibat

Interventions

Maralixibatdrug

All subjects will receive Maralixibat oral solution (up to 600 microgram per kilogram \[mcg/kg\]) twice daily