MedTrace Logo

A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille Syndrome (ALGS).

NCT04729751 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 27

Last updated 2025-02-06

No results posted yet for this study

Summary

This study is designed to assess whether the investigational drug maralixibat, is safe and well tolerated in children \<12 months of age with Alagille Syndrome \[ALGS\] or Progressive Familial Intrahepatic Cholestasis \[PFIC\].

Conditions

  • Progressive Familial Intrahepatic Cholestasis
  • Alagille Syndrome
  • Cholestatic Liver Disease

Interventions

DRUG

Maralixibat

Maralixibat chloride provided in the form of an oral solution (i.e., 5, 10, 15, and 20 mg/mL) * 400 μg/kg maralixibat chloride is equivalent to 380 µg/kg maralixibat free base * 600 μg/kg maralixibat chloride is equivalent to 570 µg/kg maralixibat free base

Sponsors & Collaborators

  • Mirum Pharmaceuticals, Inc.

    lead INDUSTRY

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
0 Days
Max Age
364 Days
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-09-09
Primary Completion
2024-12-17
Completion
2024-12-17
FDA Drug
Yes

Countries

  • United States
  • Belgium
  • Brazil
  • France
  • Mexico
  • Poland
  • United Kingdom

Study Locations

More Related Trials

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 NCT04729751 on ClinicalTrials.gov