An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC)
NCT ID: NCT04185363
Last Updated: 2025-06-29
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
PHASE3
90 participants
INTERVENTIONAL
2020-01-08
2025-04-23
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Maralixibat
All subjects will receive Maralixibat oral solution
Maralixibat
All subjects will receive Maralixibat oral solution (up to 600 microgram per kilogram \[mcg/kg\]) twice daily
Interventions
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Maralixibat
All subjects will receive Maralixibat oral solution (up to 600 microgram per kilogram \[mcg/kg\]) twice daily
Eligibility Criteria
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Inclusion Criteria
2. Completion of study MRX-502
Exclusion Criteria
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
1 Year
18 Years
ALL
No
Sponsors
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Mirum Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
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Locations
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Children's Hospital Los Angeles
Los Angeles, California, United States
Medstar Georgetown University Hospital
Washington D.C., District of Columbia, United States
Advent Health
Orlando, Florida, United States
Children's Hospital at Montefiore
The Bronx, New York, United States
Cincinnati Children's Hospital
Cincinnati, Ohio, United States
Children Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
Medical University of South Carolina
Charleston, South Carolina, United States
University of Texas Southwestern Medical Center
Dallas, Texas, United States
University of Texas, Health Science Center San Antonio
San Antonio, Texas, United States
Seattle Children's Hospital
Seattle, Washington, United States
Hospital Italiano de Buenos Aires
Buenos Aires, , Argentina
Medizinische Universität Wien, Universitätsklinik für Kinder- und Jugendheilkunde
Vienna, , Austria
Cliniques Universitaires Saint-Luc
Brussels, , Belgium
Sociedade Beneficente de Senhoras Hospital Sírio-Libanês
São Paulo, , Brazil
University of Alberta - Women and Children's Health Research Institute
Edmonton, Alberta, Canada
Fundacion Cardioinfantil
Bogotá, , Colombia
Groupement Hospitalier Est Hopital, Femme Mère Enfant de Lyon
Lyon, , France
CHU de Toulouse - Hôpital des Enfants
Toulouse, , France
Medizinische Hochschule
Hanover, , Germany
Azienda Ospedaliera Papa Giovanni XXIII - Unita di Pediatria
Bergamo, , Italy
Ospedale Pediatrico bambino Gesu'
Roma, , Italy
Hotel Dieu de France, Alfred Naccache
Beirut, , Lebanon
Consultario de Joshue David Covarrubias Esquer
Zapopan, , Mexico
Instytut Pomnik Centrum, Zdrowia Dziecka
Warsaw, , Poland
KK Women's and Children's Hospital
Singapore, , Singapore
Koc University Hospital
Istanbul, , Turkey (Türkiye)
Birmingham Children's Hospital
Birmingham, , United Kingdom
King's College Hospital NHS Foundation Trust
London, , United Kingdom
Countries
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Related Links
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Genetics Home Reference - PFIC
US FDA Resources
Mirum Pharmaceuticals homepage
Other Identifiers
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MRX-503
Identifier Type: -
Identifier Source: org_study_id
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