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

Progressive Familial Intrahepatic Cholestasis (PFIC) Clinical Trial

Official title:

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)

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04185363
• Other study ID #: MRX-503
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: January 8, 2020
• Est. completion date: September 2024

Study information

• Verified date: May 2024
• Source: Mirum Pharmaceuticals, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Description:

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

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 90
• Est. completion date: September 2024
• Est. primary completion date: September 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Year to 18 Years

Eligibility

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

Related Conditions & MeSH terms

• Cholestasis
• Cholestasis, Intrahepatic
• Progressive Familial Intrahepatic Cholestasis (PFIC)

Intervention

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

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
Mirum Pharmaceuticals, Inc.

Countries where clinical trial is conducted

United States,  Argentina,  Austria,  Belgium,  Brazil,  Canada,  Colombia,  France,  Germany,  Italy,  Lebanon,  Mexico,  Poland,  Singapore,  Turkey,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of Treatment Emergent Adverse Events (TEAEs) during the study		From baseline through time of interim analysis, up to 120 weeks
Secondary	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
Secondary	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
Secondary	Mean change from baseline over time in serum bile acid (sBA) levels		From baseline through study completion, up to approximately 4 years
Secondary	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
Secondary	Mean change from baseline over time in height and weight z-scores		From baseline through study completion, up to approximately 4 years

External Links

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