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

Phase 3

Completed

• Conditions: Progressive Familial Intrahepatic Cholestasis (PFIC)
• Interventions: Drug: Maralixibat

• Registration Number: NCT04185363
• Lead Sponsor: Mirum Pharmaceuticals, Inc.

Brief Summary

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

Detailed Description

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

Study Timeline

• Study First Submitted: 2019-12-02
• Study First Submitted QC: 2019-12-02
• Study First Posted: 2019-12-04
• Study Start: 2020-01-08
• Primary Completion: 2025-04-23
• Study Completion: 2025-04-23
• Status Verified: 2025-06
• Last Update Submitted: 2025-06-25
• Last Update Posted: 2025-06-29

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 90

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Maralixibat	Maralixibat	All subjects will receive Maralixibat oral solution

Primary Outcome Measures

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

Secondary Outcome Measures

Name	Time	Method
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) severity score	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

Trial Locations

Locations (28)

KK Women's and Children's Hospital; 🇸🇬 Singapore, Singapore

Children's Hospital Los Angeles; 🇺🇸 Los Angeles, California, United States

Medstar Georgetown University Hospital; 🇺🇸 Washington, District of Columbia, United States

Advent Health; 🇺🇸 Orlando, Florida, United States

Children's Hospital at Montefiore; 🇺🇸 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

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