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A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC)

NCT03905330 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 93

Last updated 2023-12-11

Study results available
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Summary

The purpose of this study is to determine whether the investigational treatment (maralixibat) is safe and effective in pediatric participants with Progressive Familial Intrahepatic Cholestasis (PFIC).

Conditions

  • Progressive Familial Intrahepatic Cholestasis (PFIC)

Interventions

DRUG

Maralixibat

Maralixibat oral solution (up to 600 mcg/kg) orally twice daily for 26 weeks.

OTHER

Placebo

Placebo matching to maralixibat orally twice daily for 26 weeks.

Sponsors & Collaborators

  • Mirum Pharmaceuticals, Inc.

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

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

Timeline & Regulatory

Start
2019-07-09
Primary Completion
2022-09-01
Completion
2022-09-01
FDA Drug
Yes

Countries

  • United States
  • Argentina
  • Austria
  • Belgium
  • Brazil
  • Canada
  • Colombia
  • France
  • Germany
  • Hungary
  • 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 NCT03905330 on ClinicalTrials.gov