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

Completed · Phase 3

Conditions studied: Progressive Familial Intrahepatic Cholestasis (PFIC)

In brief

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

Key facts

Study ID

NCT04185363

Run by

Mirum Pharmaceuticals, Inc.

People needed

84

Starts

2020-01-08

Expected to finish

2025-04-23

Last updated by the study team

2026-06-23

Who can join

Age: 1 and older, up to 18. Sex: any. Healthy volunteers: not accepted.

You may not qualify if…

• Any female who is pregnant or lactating or who is planning to become pregnant
• Administration of prohibited medication between the MRX-502 EOT visit and the MRX 503 Baseline Visit (Day 0)
• 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
• 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
• 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
• Cognitive impairment of the subject or caregiver that would, in the opinion of the investigator, preclude appropriate understanding of study information and compliance with study procedures

Where it is running

• 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

Full record on ClinicalTrials.gov

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