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

NCT04185363 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 90

Last updated 2025-06-29

No results posted yet for this study

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)

Interventions

DRUG

Maralixibat

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

Sponsors & Collaborators

  • Mirum Pharmaceuticals, Inc.

    lead INDUSTRY

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
1 Year
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-01-08
Primary Completion
2025-04-23
Completion
2025-04-23
FDA Drug
Yes

Countries

  • United States
  • Argentina
  • Austria
  • Belgium
  • Brazil
  • Canada
  • Colombia
  • France
  • Germany
  • Italy
  • Lebanon
  • Mexico
  • Poland
  • Singapore
  • Turkey (Türkiye)
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04185363 on ClinicalTrials.gov