Clinical Trials List
Protocol NumberA4250-011
NCT Number(ClinicalTrials.gov Identfier)NCT04336722
2020-06-01 - 2026-10-30
Phase III
Recruiting2
ICD-10Q44.2
Atresia of bile ducts
ICD-10Q44.3
Congenital stenosis and stricture of bile ducts
ICD-9751.61
Biliary atresia
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
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Trial Applicant
Syneos Health
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Sponsor
Albireo AB
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Trial scale
Multi-Regional Multi-Center
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Update
2025/08/20
Investigators and Locations
Principal Investigator
Co-Principal Investigator
- 劉君恕 Division of Pediatrics
The Actual Total Number of Participants Enrolled
0 Recruiting
The Actual Total Number of Participants Enrolled
0 Recruiting
Condition/Disease
biliary atresia
Objectives
Primary Objective
The primary objective is to evaluate the efficacy of repeated once-daily doses of
odevixibat versus placebo in children with BA post Kasai HPE based on survival with
native liver after 104 weeks of study treatment.
Secondary Objectives
• To evaluate the effect of odevixibat compared to placebo on the time to onset of
sentinel events (defined as clinically significant ascites, bleeding
gastroesophageal varices, thrombocytopenia (platelet count <150,000/µL), liver
transplant, or death) within the 104-weeks treatment period
• To evaluate the effect of odevixibat compared to placebo on total bilirubin after
13, 26, 52, and 104 weeks of study treatment
• To evaluate the effect of odevixibat compared to placebo on serum bile acids after
13, 26, 52, and 104 weeks of study treatment
• To assess the long-term safety and tolerability of repeated daily doses of
odevixibat compared to placebo for 104 weeks in children with BA post Kasai
HPE
Exploratory Objectives
• To evaluate the effect of odevixibat compared to placebo on measures of overall
hepatic health and function throughout the study treatment period
• To evaluate the effect of odevixibat compared to placebo on overall health of the
patients throughout the study treatment period
Test Drug
Odevixibat (A4250)
Active Ingredient
Odevixibat (A4250)
Dosage Form
capsule
Dosage
200, or 600
Endpoints
Primary Outcome Measures :
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.
Secondary Outcome Measures :
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
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.
Secondary Outcome Measures :
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
Inclution Criteria
Inclusion Criteria:
A male or female patient with a clinical diagnosis of BA
Age at Kasai HPE ≤90 days
Eligible to start study treatment within 3 weeks post-Kasai HPE
A male or female patient with a clinical diagnosis of BA
Age at Kasai HPE ≤90 days
Eligible to start study treatment within 3 weeks post-Kasai HPE
Exclusion Criteria
Key Exclusion Criteria:
Patients with intractable ascites
Ileal resection surgery
ALT ≥10× upper limit of normal (ULN) at screening
Patients reliant only on total parenteral nutrition, or not able to take study medication orally, at randomization
Acute ascending cholangitis (patients may be randomized after resolution of acute ascending cholangitis)
Choledochal cystic disease
INR >1.6 (the patient may be treated with Vitamin K intravenously; sample may be redrawn and if INR is ≤1.6 at resampling the patient may be randomized)
Any other conditions or abnormalities, including congenital abnormalities, major cardiac surgery, hepatic, biliary, or GI disease which, in the opinion of the Investigator or Medical Monitor, may compromise the safety of the patient, the integrity of study results, or patient compliance with study requirements
Weight <3.5kg at randomization
Patients with intractable ascites
Ileal resection surgery
ALT ≥10× upper limit of normal (ULN) at screening
Patients reliant only on total parenteral nutrition, or not able to take study medication orally, at randomization
Acute ascending cholangitis (patients may be randomized after resolution of acute ascending cholangitis)
Choledochal cystic disease
INR >1.6 (the patient may be treated with Vitamin K intravenously; sample may be redrawn and if INR is ≤1.6 at resampling the patient may be randomized)
Any other conditions or abnormalities, including congenital abnormalities, major cardiac surgery, hepatic, biliary, or GI disease which, in the opinion of the Investigator or Medical Monitor, may compromise the safety of the patient, the integrity of study results, or patient compliance with study requirements
Weight <3.5kg at randomization
The Estimated Number of Participants
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Taiwan
10 participants
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Global
245 participants
