Evaluation of Maralixibat in Biliary Atresia Response Post-Kasai
NCT ID: NCT04524390
Last Updated: 2025-03-19
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE2
75 participants
INTERVENTIONAL
2021-07-08
2024-02-07
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
TRIPLE
Study Groups
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Double Blind - Maralixibat
The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.
Maralixibat
A small molecule inhibitor of the ileal bile acid transporter (IBAT)
Double Blind - Placebo
The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm.
Placebo
Identical to maralixibat except for the active drug substance
Open Label - Maralixibat
The Open-Label period comprised of 4-8 weeks of dose escalation followed by 70 - 74 weeks of stable dosing treatment. During the OLE, all participants, regardless of treatment assignment in the double-blind period, received maralixibat.
Maralixibat
A small molecule inhibitor of the ileal bile acid transporter (IBAT)
Interventions
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Maralixibat
A small molecule inhibitor of the ileal bile acid transporter (IBAT)
Placebo
Identical to maralixibat except for the active drug substance
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
2. HPE or Kasai Procedure within 3 weeks prior to randomization
3. Clinical diagnosis of biliary atresia
Exclusion Criteria
2. Subjects not tolerating enteral feeds at randomization
3. History of ileal resection
4. Diagnosis of biliary atresia splenic malformation syndrome or cystic biliary atresia
5. Evidence of another non-biliary atresia pathology involving the intrahepatic bile duct (e.g., paucity, sclerosing cholangitis)
6. Evidence of liver failure (e.g. significant ascites)
21 Days
111 Days
ALL
No
Sponsors
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Mirum Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
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Locations
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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 District, , 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
Countries
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Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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MRX-701
Identifier Type: -
Identifier Source: org_study_id
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