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

NCT04185363 | Completed Phase 3 DMC FDA Drug

• 1 other identifier: MRX-503
• Study Type: interventional
• Target: 90
• Locations: 16 countries
• Sites: 28

Brief Summary

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

Conditions

Progressive Familial Intrahepatic Cholestasis (PFIC)

Keywords

Cholestasis; Maralixibat; Mutation; PFIC; PFIC2; Bile Duct Diseases; Liver Diseases; Biliary Tract Diseases; Digestive System Diseases; Pediatric

Outcome Measures

Primary Outcomes (1)

• Incidence of Treatment Emergent Adverse Events (TEAEs) during the study

  From baseline through time of interim analysis, up to 120 weeks

Secondary Outcomes (5)

• Change from maralixibat baseline over the course of the study in the weekly average morning ItchRO(Obs)™ (Itch Reported Outcome) severity score

  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

  From baseline through study completion, up to approximately 4 years

• Mean change from baseline over time in serum bile acid (sBA) levels

  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

  From baseline through study completion, up to approximately 4 years

• Mean change from baseline over time in height and weight z-scores

  From baseline through study completion, up to approximately 4 years

Study Arms (1)

Maralixibat

EXPERIMENTAL

All subjects will receive Maralixibat oral solution

Drug: Maralixibat

Interventions

Maralixibat DRUG

All subjects will receive Maralixibat oral solution (up to 600 microgram per kilogram \[mcg/kg\]) twice daily

Maralixibat

Eligibility Criteria

• Age: 1 Year - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

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

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

Sponsors & Collaborators

• Mirum Pharmaceuticals, Inc.: lead

Study Sites (28)

Children's Hospital Los Angeles

Los Angeles, California, 90027, United States

Location

Medstar Georgetown University Hospital

Washington D.C., District of Columbia, 20007, United States

Location

Advent Health

Orlando, Florida, 32803, United States

Location

Children's Hospital at Montefiore

The Bronx, New York, 10461, United States

Location

Cincinnati Children's Hospital

Cincinnati, Ohio, 45229, United States

Location

Children Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15224, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

University of Texas Southwestern Medical Center

Dallas, Texas, 75390, United States

Location

University of Texas, Health Science Center San Antonio

San Antonio, Texas, 78229, United States

Location

Seattle Children's Hospital

Seattle, Washington, 98105, United States

Location

Hospital Italiano de Buenos Aires

Buenos Aires, Argentina

Location

Medizinische Universität Wien, Universitätsklinik für Kinder- und Jugendheilkunde

Vienna, Austria

Location

Cliniques Universitaires Saint-Luc

Brussels, Belgium

Location

Sociedade Beneficente de Senhoras Hospital Sírio-Libanês

São Paulo, Brazil

Location

University of Alberta - Women and Children's Health Research Institute

Edmonton, Alberta, Canada

Location

Fundacion Cardioinfantil

Bogotá, Colombia

Location

Groupement Hospitalier Est Hopital, Femme Mère Enfant de Lyon

Lyon, France

Location

CHU de Toulouse - Hôpital des Enfants

Toulouse, 31059, France

Location

Medizinische Hochschule

Hanover, Germany

Location

Azienda Ospedaliera Papa Giovanni XXIII - Unita di Pediatria

Bergamo, 24127, Italy

Location

Ospedale Pediatrico bambino Gesu'

Roma, Italy

Location

Hotel Dieu de France, Alfred Naccache

Beirut, Lebanon

Location

Consultario de Joshue David Covarrubias Esquer

Zapopan, Mexico

Location

Instytut Pomnik Centrum, Zdrowia Dziecka

Warsaw, Poland

Location

KK Women's and Children's Hospital

Singapore, Singapore

Location

Koc University Hospital

Istanbul, 34010, Turkey (Türkiye)

Location

Birmingham Children's Hospital

Birmingham, United Kingdom

Location

King's College Hospital NHS Foundation Trust

London, United Kingdom

Location

Related Links

• Genetics Home Reference - PFIC
• US FDA Resources
• Mirum Pharmaceuticals homepage

MeSH Terms

Conditions

Cholestasis, progressive familial intrahepatic 1; Cholestasis; Bile Duct Diseases; Liver Diseases; Biliary Tract Diseases; Digestive System Diseases

Interventions

maralixibat

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 2, 2019
• First Posted: December 4, 2019
• Study Start: January 8, 2020
• Primary Completion: April 23, 2025
• Study Completion: April 23, 2025
• Last Updated: June 29, 2025

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will not share

Locations

GB (2); CA (1); FR (2); ME (1); US (10); CO (1); AU (1); SG (1); IT (2); BR (1); PL (1); DE (1); LE (1); BE (1); TU (1); AR (1)