Evaluation of Maralixibat in Biliary Atresia Response Post-Kasai

Completed · Phase 2 · Has a placebo group

Conditions studied: Biliary Atresia

In brief

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).

Key facts

Study ID

NCT04524390

Run by

Mirum Pharmaceuticals, Inc.

People needed

75

Starts

2021-07-08

Expected to finish

2024-02-07

Last updated by the study team

2025-03-19

Who can join

Age: 0 and older, up to 0. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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)

Where it is running

• Phoenix Children's Division of Gastroenterology & Hepatology — Phoenix, Arizona, United States
• Children's Healthcare of Atlanta - Emory University School of Medicine — Atlanta, Georgia, United States
• NYU Grossman School of Medicine — New York, New York, United States
• New York-Presbyterian - Columbia University Medical Center — New York, New York, United States
• Montefiore Medical Center — The Bronx, New York, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• Texas Children's Hospital — Houston, Texas, United States
• Beijing Pediatric Research Institute — Beijing, Beijing Municipality, China
• Guangzhou Women and Children's Medical Center — Guangzhou, Guangdong, China
• The Children's Hospital, Zhejiang University School of Medicine — Hanzhou, Zhejiang, China
• Children's hospital of Shanghai — Shanghai, China
• Children's Hospital of Fudan University — Shanghai, China
• Hannover Medical School — Hanover, Germany
• Instytut Pomnik-Centrum Zdrowia Dziecka — Warsaw, Poland
• KK women's and Children's hospital — Bukit Timah, Singapore
• Taichung Veterans General Hospital — Taichung, Taiwan
• Linkou Chang Gung Memorial Hospital — Taoyuan, Taiwan
• Birmingham Children's Hospital — Birmingham, United Kingdom
• King's College Hospital NHS — London, United Kingdom
• Hue Central Hospital — Huế, Thừa Thiên Huế Province, Vietnam
• Vietnam National Children's Hospital — Hanoi, Vietnam
• Children's Hospital No. 1 — Ho Chi Minh City, Vietnam

Full record on ClinicalTrials.gov

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