Double-blind, randomized, placebo-controlled, Phase 3 study to investigate the efficacy and safety of odevixibat compared to placebo in children with biliary atresia who have undergone a Kasai hepatoportoenterostomy.
Up to 70 sites will be initiated for this study in North America, Europe, Middle East, and Asia Pacific.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
254
Odevixibat is a small molecule and selective inhibitor of IBAT.
Placebo identical in appearance to experimental drug (odevixibat).
Time from randomization to first occurrence of liver transplant, or death
Time frame: From baseline to Week 104
Proportion of patients with liver transplant
Proportion of patients who are alive and have not undergone a liver transplant
Time frame: From baseline to Week 104
Time to onset of any sentinel events
Time to onset of any sentinel events
Time frame: From baseline to Week 104
Total bilirubin levels
Total bilirubin level after 13, 26, 52, and 104 weeks of study treatment
Time frame: From baseline to Weeks 13, 26, 52 and 104
Serum bile acid levels
Serum bile acid level after 13, 26, 52, and 104 weeks of study treatment
Time frame: From baseline to Weeks 13, 26, 52 and 104
Time to pediatric end-stage liver disease (PELD) score >15
Time to pediatric end-stage liver disease (PELD) score \>15
Time frame: From baseline to Week 104
Percentage of participants experiencing Adverse Events (AEs) and Serious Adverse Events (SAEs)
An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention.
Time frame: From baseline to Week 104
Percentage of participants with clinically significant changes in Physical Examination
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Children's Hospital Los Angeles
Los Angeles, California, United States
Stanford Children's Health
Palo Alto, California, United States
Rady Children's Hospital
San Diego, California, United States
UCSF Benioff Children's Hospital San Francisco
San Francisco, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Nemours/Alfred I. duPont Hospital for Children
Wilmington, Delaware, United States
Children's National Medical Center
Washington D.C., District of Columbia, United States
University of Miami
Miami, Florida, United States
Children's Healthcare of Atlanta - Emory University School of Medicine
Atlanta, Georgia, United States
Ann & Robert H. Lurie Children's Hospital
Chicago, Illinois, United States
...and 56 more locations
Percentage of participants with clinically significant changes in physical examination findings will be reported. The clinical significance will be graded by the investigator.
Time frame: From baseline to Week 104
Percentage of participants with clinically significant changes in Laboratory Parameters (blood chemistry, hematology and coagulation)
Percentage of participants with clinically significant change in laboratory parameters (blood chemistry, hematology and coagulation) will be reported. The clinical significance will be decided by the investigator.
Time frame: From baseline to Week 104
Percentage of participants with clinically significant changes in Abdominal Ultrasound findings
Percentage of participants with clinically significant change in Abdominal Ultrasound findings will be reported. The clinical significance will be decided by the investigator.
Time frame: From baseline to Week 26 and Week 104