Efficacy and Safety of Odevixibat in Children With Biliary Atresia Who Have Undergone a Kasai HPE (BOLD)
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|ClinicalTrials.gov Identifier: NCT04336722|
Recruitment Status : Recruiting
First Posted : April 7, 2020
Last Update Posted : June 24, 2021
|Condition or disease||Intervention/treatment||Phase|
|Biliary Atresia||Drug: Odevixibat Drug: Placebo||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||200 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Triple (Participant, Care Provider, Investigator)|
|Official Title:||A Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Odevixibat (A4250) in Children With Biliary Atresia Who Have Undergone a Kasai Hepatoportoenterostomy|
|Actual Study Start Date :||July 10, 2020|
|Estimated Primary Completion Date :||May 2024|
|Estimated Study Completion Date :||June 2024|
Experimental: Odevixibat (A4250)
Capsules for oral administration once daily for 104 weeks.
Odevixibat is a small molecule and selective inhibitor of IBAT.
Placebo Comparator: Placebo
Capsules for oral administration (to match active) once daily for 104 weeks.
Placebo identical in appearance to experimental drug (odevixibat).
- Proportion of patients with liver transplant [ Time Frame: From baseline to Week 104 ]Proportion of patients who are alive and have not undergone a liver transplant after 104 weeks of study treatment.
- Time to onset of any sentinel events [ Time Frame: From baseline to Week 104 ]Time to onset of any sentinel events
- Time to pediatric end-stage liver disease (PELD) score >15 [ Time Frame: From baseline to Week 104 ]Time to pediatric end-stage liver disease (PELD) score >15
- Total bilirubin levels [ Time Frame: From baseline to Weeks 13, 26, 52 and 104 ]Total bilirubin level after 13, 26, 52, and 104 weeks of study treatment
- Serum bile acid levels [ Time Frame: From baseline to Weeks 13, 26, 52 and 104 ]Serum bile acid level after 13, 26, 52, and 104 weeks of study treatment
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04336722
|Contact: Albireo||+1 (857) email@example.com|