An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC)

June 25, 2025 updated by: Mirum Pharmaceuticals, Inc.

An Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in the Treatment of Subjects With Progressive Familial Intrahepatic Cholestasis (PFIC)

The primary objective of this open label extension study is to evaluate the long-term safety and tolerability of maralixibat.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

The study will be conducted at multiple sites in North America, Europe, Asia, and South America.

Study Type

Interventional

Enrollment (Actual)

90

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Argentina
      • Buenos Aires, Argentina
        • Hospital Italiano de Buenos Aires
  • Austria
      • Wien, Austria
        • Medizinische Universität Wien, Universitätsklinik für Kinder- und Jugendheilkunde
  • Belgium
      • Brussels, Belgium
        • Cliniques Universitaires Saint-luc
  • Brazil
      • São Paulo, Brazil
        • Sociedade Beneficente de Senhoras Hospital Sírio-Libanês
  • Canada
    • Alberta
      • Edmonton, Alberta, Canada
        • University of Alberta - Women and Children's Health Research Institute
  • Colombia
      • Bogotá, Colombia
        • Fundacion Cardioinfantil
  • France
      • Lyon, France
        • Groupement Hospitalier Est Hopital, Femme Mère Enfant de Lyon
      • Toulouse, France, 31059
        • CHU de Toulouse - Hôpital des Enfants
  • Germany
      • Hanover, Germany
        • Medizinische Hochschule
  • Italy
      • Bergamo, Italy, 24127
        • Azienda Ospedaliera Papa Giovanni XXIII - Unita di Pediatria
      • Roma, Italy
        • Ospedale Pediatrico Bambino Gesu'
  • Lebanon
      • Beirut, Lebanon
        • Hotel Dieu de France, Alfred Naccache
  • Mexico
      • Zapopan, Mexico
        • Consultario de Joshue David Covarrubias Esquer
  • Poland
      • Warsaw, Poland
        • Instytut Pomnik Centrum, Zdrowia Dziecka
  • Singapore
      • Singapore, Singapore
        • KK Women's and Children's Hospital
  • Turkey
      • Istanbul, Turkey, 34010
        • Koc University Hospital
  • United Kingdom
      • Birmingham, United Kingdom
        • Birmingham Children's Hospital
      • London, United Kingdom
        • King's College Hospital NHS Foundation Trust
  • United States
    • California
      • Los Angeles, California, United States, 90027
        • Children's Hospital Los Angeles
    • District of Columbia
      • Washington, District of Columbia, United States, 20007
        • MedStar Georgetown University Hospital
    • Florida
      • Orlando, Florida, United States, 32803
        • Advent Health
    • New York
      • Bronx, New York, United States, 10461
        • Children's Hospital at Montefiore
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Cincinnati Children's Hospital
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15224
        • Children Hospital of Pittsburgh
    • South Carolina
      • Charleston, South Carolina, United States, 29425
        • Medical University of South Carolina
    • Texas
      • Dallas, Texas, United States, 75390
        • University of Texas Southwestern Medical Center
      • San Antonio, Texas, United States, 78229
        • University of Texas, Health Science Center San Antonio
    • Washington
      • Seattle, Washington, United States, 98105
        • Seattle Children's Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 year to 18 years (Child, Adult)

Accepts Healthy Volunteers

No

Description

Key Inclusion Criteria:

  1. Provide informed consent and assent (as applicable) per Institutional Review Board/Ethics Committee (IRB/EC)
  2. Completion of study MRX-502

Exclusion Criteria:

  1. Any female who is pregnant or lactating or who is planning to become pregnant
  2. Administration of prohibited medication between the MRX-502 EOT visit and the MRX 503 Baseline Visit (Day 0)
  3. History of non-compliance in study MRX-502, non-adherence to medical regimens, unreliability, mental instability or incompetence that could compromise the validity of informed consent or lead to non-adherence with the study protocol based on Investigator judgment
  4. Experienced an adverse event (AE) or serious adverse event (SAE) related to maralixibat during the MRX-502 study that led to permanent discontinuation of the subject from maralixibat
  5. Any other conditions or laboratory abnormalities that, in the opinion of the Investigator or Sponsor Medical Monitor, may compromise the safety of the subject, or interfere with the subject participating in or completing the study

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Maralixibat
All subjects will receive Maralixibat oral solution
Drug: Maralixibat
All subjects will receive Maralixibat oral solution (up to 600 microgram per kilogram [mcg/kg]) twice daily

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Incidence of Treatment Emergent Adverse Events (TEAEs) during the study
Time Frame: From baseline through time of interim analysis, up to 120 weeks
From baseline through time of interim analysis, up to 120 weeks

Secondary Outcome Measures

Outcome Measure
Time Frame
Change from maralixibat baseline over the course of the study in the weekly average morning ItchRO(Obs)™ (Itch Reported Outcome) severity score
Time Frame: From baseline through study completion, up to approximately 4 years
From baseline through study completion, up to approximately 4 years
Maintenance of treatment effect based on the average morning ItchRO(Obs)™ severity scores over the time
Time Frame: From baseline through study completion, up to approximately 4 years
From baseline through study completion, up to approximately 4 years
Mean change from baseline over time in serum bile acid (sBA) levels
Time Frame: From baseline through study completion, up to approximately 4 years
From baseline through study completion, up to approximately 4 years
Change from maralixibat baseline over the course of the study in the weekly average morning ItchRO(Obs)™ (Itch Reported Outcome) frequency score
Time Frame: From baseline through study completion, up to approximately 4 years
From baseline through study completion, up to approximately 4 years
Mean change from baseline over time in height and weight z-scores
Time Frame: From baseline through study completion, up to approximately 4 years
From baseline through study completion, up to approximately 4 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Mirum Pharmaceuticals, Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 8, 2020

Primary Completion (Actual)

April 23, 2025

Study Completion (Actual)

April 23, 2025

Study Registration Dates

First Submitted

December 2, 2019

First Submitted That Met QC Criteria

December 2, 2019

First Posted (Actual)

December 4, 2019

Study Record Updates

Last Update Posted (Actual)

June 29, 2025

Last Update Submitted That Met QC Criteria

June 25, 2025

Last Verified

June 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • MRX-503

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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