Randomized, Double-Blind, Placebo-Controlled Phase 2 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Subjects With Biliary Atresia After Hepatoportoenterostomy

Who is this study for? Infant patients with Biliary Atresia
What treatments are being studied? Maralixibat
Status: Recruiting
Location: See all (21) locations...
Intervention Type: Other, Drug
Study Type: Interventional
Study Phase: Phase 2
SUMMARY

A study to evaluate the efficacy and safety of maralixibat in infants with Biliary Atresia (BA) after Hepatoportoenterostomy (HPE, also known as the Kasai procedure).

Eligibility
Participation Requirements
Sex: All
Minimum Age: 21 days
Maximum Age: 3 months
Healthy Volunteers: No
View:

• Male or female subjects with body weight ≥2500 g, who are ≥21 days old and <90 days old at the time of HPE (Kasai)

• HPE or Kasai Procedure within 3 weeks prior to randomization

• Clinical diagnosis of biliary atresia

Locations
United States
Arizona
Phoenix Children's Division of Gastroenterology & Hepatology
Recruiting
Phoenix
Washington, D.c.
Medstar Georgetown University Hospital
Recruiting
Washington
Illinois
The University of Chicago Medical Center
Recruiting
Chicago
Louisiana
Ochsner Clinic Foundation
Recruiting
New Orleans
New York
Columbia University Irving Medical Center
Active, not recruiting
New York
Pennsylvania
Children's Hospital of Philadelphia
Recruiting
Philadelphia
UPMC Children's Hospital of Pittsburgh
Recruiting
Pittsburgh
Tennessee
Le Bonheur Children's Hospital
Recruiting
Memphis
Texas
Texas Children's Hospital
Recruiting
Houston
Other Locations
China
Beijing Pediatric Research Institute
Recruiting
Beijing
Guangzhou Women and Children's Medical Center
Recruiting
Guangzhou
The Children's Hospital, Zhejiang University School of Medicine
Recruiting
Hanzhou
Children's Hospital of Fudan University
Recruiting
Shanghai
Children's hospital of Shanghai
Recruiting
Shanghai
Germany
Hannover Medical School
Recruiting
Hanover
Poland
Instytut Pomnik-Centrum Zdrowia Dziecka
Recruiting
Warsaw
Singapore
KK women's and Children's hospital
Recruiting
Bukit Timah
Taiwan
Taichung Veterans General Hospital
Recruiting
Taichung
National Taiwan University Hospital
Recruiting
Taipei
Linkou Chang Gung Memorial Hospital
Recruiting
Taoyuan
United Kingdom
King's College Hospital NHS
Recruiting
London
Contact Information
Primary
Clinical Trials Mirum
clinicaltrials@mirumpharma.com
+16506674085
Backup
Medinfo Mirum
medinfo@mirumpharma.com
Time Frame
Start Date: July 8, 2021
Estimated Completion Date: March 2025
Participants
Target number of participants: 72
Treatments
Experimental: Maralixibat
Maralixibat chloride oral solution administered twice daily, up to 600* microgram per kilogram, for 26 weeks and in the OLE for all patients.~*equivalent to 570 mcg/kg/day maralixibat free base
Placebo Comparator: Placebo
Placebo oral solution for 26 weeks. All placebo participants who complete Week 26 and continue in the open label extension (OLE) will receive maralixibat after Week 26.
Related Therapeutic Areas
Sponsors
Leads: Mirum Pharmaceuticals, Inc.

This content was sourced from clinicaltrials.gov

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