CI

At a glance

ClinicalIndex Comparison Record
Phase 2Completed· 31 enrolled
Drug / intervention
LUM001 (Maralixibat) +1 moredrug
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.

Search/NCT02160782
NCT02160782Phase 2Completed

Long-Term, Open-Label Study With a Double-Blind, Placebo-Controlled, Randomized Drug Withdrawal Period of LUM001 (Maralixibat), an Apical Sodium-Dependent Bile Acid Transporter Inhibitor (ASBTi), in Patients With Alagille Syndrome

Mirum Pharmaceuticals, Inc.·interventional·Posted Jun 11, 2014·Updated Jul 14, 2021

In Brief

A Phase 2 clinical trial evaluating LUM001 (Maralixibat) and Placebo for Alagille Syndrome. Completed, enrolled 31 participants across 9 sites in 6 countries.

Detailed Summary

This is a long-term, open-label study with a double-blind, placebo-controlled, randomized drug withdrawal period in children with Alagille Syndrome (ALGS) designed to evaluate the safety and efficacy of LUM001 (Also known as maralixibat or MRX).

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
CountriesAustralia, Belgium, France, Poland, Spain, United Kingdom
Collaborators--

Timeline

Phase 2CompletedFinished
201520162017201820192020202120222023202420252026
First PostedJun 11, 2014
Enrollment StartOct 28, 2014
Primary CompletionMay 28, 2020
TodayJul 2, 2026
Enrollment to primary: 5.6 yearsPosted 12.1 years ago

Interventions

LUM001 (Maralixibat)drug

LUM001, also known as Maralixibat (MRX) will be administered orally Once Daily (OD). To be administered Twice Daily (BID) for patients who are eligible.

Placebodrug

Placebo will be administered orally once daily during randomized withdrawal period