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Safety and Efficacy Study of LUM001 (Maralixibat) With a Drug Withdrawal Period in Participants With Alagille Syndrome (ALGS)

NCT02160782 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 31

Last updated 2021-07-14

Study results available
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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).

Conditions

  • Alagille Syndrome

Interventions

DRUG

LUM001 (Maralixibat)

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

DRUG

Placebo

Placebo will be administered orally once daily during randomized withdrawal period

Sponsors & Collaborators

  • Mirum Pharmaceuticals, Inc.

    lead INDUSTRY

Principal Investigators

  • Study Director · Mirum

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Model
PARALLEL

Eligibility

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

Timeline & Regulatory

Start
2014-10-28
Primary Completion
2020-05-28
Completion
2020-05-28
FDA Drug
Yes

Countries

  • Australia
  • Belgium
  • France
  • Poland
  • Spain
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs

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 NCT02160782 on ClinicalTrials.gov