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)

Mirum Pharmaceuticals, Inc.28 sites in 16 countries90 target enrollmentJanuary 8, 2020

InterventionsMaralixibat

Overview

Phase: Phase 3
Intervention: Maralixibat
Conditions: Progressive Familial Intrahepatic Cholestasis (PFIC)
Sponsor: Mirum Pharmaceuticals, Inc.
Enrollment: 90
Locations: 28
Primary Endpoint: Incidence of Treatment Emergent Adverse Events (TEAEs) during the study
Status: Completed
Last Updated: 10 months ago

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

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

Registry

clinicaltrials.gov

Start Date

January 8, 2020

End Date

April 23, 2025

Last Updated

10 months ago

Study Type

Interventional

Study Design

Single Group

Sex

All

Sponsor

Responsible Party

Sponsor

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

•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

All subjects will receive Maralixibat oral solution

Intervention: Maralixibat

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

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

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)