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: Completed
Location: See all (22) 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: f
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
Phoenix
Georgia
Children's Healthcare of Atlanta - Emory University School of Medicine
Atlanta
New York
New York-Presbyterian - Columbia University Medical Center
New York
NYU Grossman School of Medicine
New York
Montefiore Medical Center
The Bronx
Pennsylvania
Children's Hospital of Philadelphia
Philadelphia
Texas
Texas Children's Hospital
Houston
Other Locations
China
Beijing Pediatric Research Institute
Beijing
Guangzhou Women and Children's Medical Center
Guangzhou
The Children's Hospital, Zhejiang University School of Medicine
Hanzhou
Children's Hospital of Fudan University
Shanghai
Children's hospital of Shanghai
Shanghai
Germany
Hannover Medical School
Hanover
Poland
Instytut Pomnik-Centrum Zdrowia Dziecka
Warsaw
Singapore
KK women's and Children's hospital
Bukit Timah
Taiwan
Taichung Veterans General Hospital
Taichung
Linkou Chang Gung Memorial Hospital
Taoyuan District
United Kingdom
Birmingham Children's Hospital
Birmingham
King's College Hospital NHS
London
Viet Nam
Vietnam National Children's Hospital
Hanoi
Children's Hospital No. 1
Ho Chi Minh City
Hue Central Hospital
Huế
Time Frame
Start Date: 2021-07-08
Completion Date: 2024-02-07
Participants
Target number of participants: 75
Treatments
Experimental: 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.
Placebo_comparator: 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.
Experimental: 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.
Authors
Related Therapeutic Areas
Sponsors
Leads: Mirum Pharmaceuticals, Inc.