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An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Pediatric Subjects With Alagille Syndrome

NCT02117713 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 34

Last updated 2021-07-01

Study results available
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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.

Conditions

  • Alagille Syndrome

Interventions

DRUG

LUM001 (Maralixibat)

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).

Sponsors & Collaborators

  • Lumena Pharmaceuticals, Inc.

    collaborator INDUSTRY

  • Childhood Liver Disease Research and Education Network

    collaborator OTHER

  • Mirum Pharmaceuticals, Inc.

    lead INDUSTRY

Principal Investigators

  • Study Director · Mirum

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
DOUBLE
Model
SINGLE_GROUP

Eligibility

Min Age
1 Year
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-03-16
Primary Completion
2020-06-01
Completion
2020-06-01
FDA Drug
Yes

Countries

  • United States
  • Canada

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 NCT02117713 on ClinicalTrials.gov