Open Label Study to Evaluate Efficacy and Long Term Safety of LUM001 (Maralixibat) in the Treatment of Cholestatic Liver Disease in Patients With Progressive Familial Intrahepatic Cholestasis

ClinicalTrials.gov ID: NCT02057718

Public ClinicalTrials.gov record NCT02057718. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

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ClinicalTrials.gov public records Last synced Jul 22, 2026, 1:23 PM EDT

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Official title

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

Study identification

NCT ID: NCT02057718
Recruitment status: Completed
Study type: Interventional
Phase: Phase 2
Lead sponsor: Mirum Pharmaceuticals, Inc.; Industry
Enrollment: 33 participants

Conditions and interventions

Conditions

Progressive Familial Intrahepatic Cholestasis (PFIC)

Interventions

LUM001 (Maralixibat) Drug

Drug

Eligibility (public fields only)

Age range: 12 Months to 18 Years
Sex: All
Healthy volunteers: Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date: Feb 28, 2014
Primary completion: May 7, 2020
Completion: May 7, 2020
Last update posted: Oct 22, 2023

2014 – 2020

United States locations

U.S. sites: 5
U.S. states: 4
U.S. cities: 5

Facility	City	State	ZIP	Site status
Children's Hospital Los Angeles	Los Angeles	California	90027	—
Children's Hospital Colorado	Aurora	Colorado	80045	—
Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio	45229	—
The Children's Hospital of Philadelphia	Philadelphia	Pennsylvania	19104	—
Children's Hospital of Pittsburgh of UPMC	Pittsburgh	Pennsylvania	15224	—

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

Non-U.S. locations

This page focuses on the U.S. directory. The official record also lists 5 non-U.S. sites.

About this trial record page

What this page shows: Public field values for ClinicalTrials.gov record NCT02057718, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do: No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from: Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh: Last update posted Oct 22, 2023 · Synced Jul 22, 2026

Open the official record

The complete protocol, eligibility criteria, and contact information for NCT02057718 live on ClinicalTrials.gov.

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