Status:
COMPLETED
Evaluation of Maralixibat in Biliary Atresia Response Post-Kasai
Lead Sponsor:
Mirum Pharmaceuticals, Inc.
Conditions:
Biliary Atresia
Eligibility:
All Genders
21-111 years
Phase:
PHASE2
Brief 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).
Detailed Description
This is a double-blind randomized, placebo-controlled study in subjects with Biliary Atresia with a primary endpoint at Week 26 followed by long-term open-label period during which all subjects will r...
Eligibility Criteria
Inclusion
- 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
Exclusion
- Subjects with intractable chronic diarrhea at randomization
- Subjects not tolerating enteral feeds at randomization
- History of ileal resection
- Diagnosis of biliary atresia splenic malformation syndrome or cystic biliary atresia
- Evidence of another non-biliary atresia pathology involving the intrahepatic bile duct (e.g., paucity, sclerosing cholangitis)
- Evidence of liver failure (e.g. significant ascites)
Key Trial Info
Start Date :
July 8 2021
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
February 7 2024
Estimated Enrollment :
75 Patients enrolled
Trial Details
Trial ID
NCT04524390
Start Date
July 8 2021
End Date
February 7 2024
Last Update
March 19 2025
Active Locations (22)
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1
Phoenix Children's Division of Gastroenterology & Hepatology
Phoenix, Arizona, United States, 85016
2
Children's Healthcare of Atlanta - Emory University School of Medicine
Atlanta, Georgia, United States, 30329
3
NYU Grossman School of Medicine
New York, New York, United States, 10016
4
New York-Presbyterian - Columbia University Medical Center
New York, New York, United States, 10032