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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

NCT02057718 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 33

Last updated 2023-10-23

Study results available
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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.

Conditions

  • Progressive Familial Intrahepatic Cholestasis (PFIC)

Interventions

DRUG

LUM001 (Maralixibat)

LUM001 also known as Maralixibat (MRX) oral dose up to twice a day (BID).

Sponsors & Collaborators

  • Mirum Pharmaceuticals, Inc.

    lead INDUSTRY

Principal Investigators

  • Study Director · Mirum

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
12 Months
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-03-01
Primary Completion
2020-05-08
Completion
2020-05-08
FDA Drug
Yes

Countries

  • United States
  • France
  • Poland
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02057718 on ClinicalTrials.gov