CI

At a glance

ClinicalIndex Comparison Record
Phase 2Completed· 34 enrolled
Drug / intervention
LUM001 (Maralixibat)drug
Likely dose
LUM001 (Maralixibat) 280 microgramsfrom 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/NCT02117713
NCT02117713Phase 2Completed
CompareCT.gov

A Multicenter Extension Study to Evaluate the Long-Term Safety and Durability of the Therapeutic Effect of LUM001, an Apical Sodium-Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Pediatric Subjects With Alagille Syndrome

Mirum Pharmaceuticals, Inc.·interventional·Posted Apr 21, 2014·Updated Jul 1, 2021

In Brief

A Phase 2 clinical trial evaluating LUM001 (Maralixibat) for Alagille Syndrome. Completed, enrolled 34 participants across 11 sites in 2 countries.

Detailed Summary

This is a multicentre, extension study of LUM001 in children diagnosed with Alagille Syndrome who have completed participation in a core LUM001 treatment protocol. The primary objective is to evaluate long-term safety and tolerability of LUM001. Efficacy will be assessed by evaluating the effect of LUM001 on the biochemical markers and pruritus associated with Alagille Syndrome.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsAlagille Syndrome
CountriesCanada, United States
CollaboratorsLumena Pharmaceuticals, Inc., Childhood Liver Disease Research and Education Network

Timeline

Phase 2CompletedFinished
2014201520162017201820192020202120222023202420252026
First PostedApr 21, 2014
Enrollment StartMar 16, 2015
Primary CompletionJun 1, 2020
TodayJul 2, 2026
Enrollment to primary: 5.2 yearsPosted 12.2 years ago

Interventions

LUM001 (Maralixibat)drug

Dosing of LUM001 also known as Maralixibat (MRX) with the objective of achieving optimal control of pruritus at a dose level that is tolerated by the participant and up to a maximum daily dose of 280 micrograms per kilogram (mcg/kg).