At a glance
ClinicalIndex Comparison RecordPhase 2Completed· 33 enrolled
Drug / intervention
LUM001 (Maralixibat)drug
Likely dose
Not stated in 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.
Open Label Study of the Efficacy and Long Term Safety of LUM001 (Maralixibat), an Apical Sodium-Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Pediatric Patients With Progressive Familial Intrahepatic Cholestasis
In Brief
A Phase 2 clinical trial evaluating LUM001 (Maralixibat) for Progressive Familial Intrahepatic Cholestasis (PFIC). Completed, enrolled 33 participants across 10 sites in 4 countries.
Detailed Summary
This is an open label study in children with Progressive Familial Intrahepatic Cholestasis (PFIC) designed to evaluate the safety and efficacy of LUM001, also known as Maralixibat (MRX). Efficacy will be assessed by evaluating the effect of LUM001 on pruritus and the biochemical markers of pruritus associated with PFIC.
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
CountriesFrance, Poland, United Kingdom, United States
Collaborators--
Timeline
Phase 2CompletedFinished
2014201520162017201820192020202120222023202420252026
First PostedFeb 2014
Enrollment StartMar 2014
Primary CompletionMay 2020
TodayJul 2026
First PostedFeb 7, 2014
Enrollment StartMar 1, 2014
Primary CompletionMay 8, 2020
TodayJul 2, 2026
Enrollment to primary: 6.2 yearsPosted 12.4 years ago
Interventions
LUM001 (Maralixibat)drug
LUM001 also known as Maralixibat (MRX) oral dose up to twice a day (BID).