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 (BOLD)

Phase 3

Recruiting

• Conditions: 10019806; 10004606; Biliary Atresia; Childhood disease of the liver

• Registration Number: NL-OMON54084
• Lead Sponsor: Albireo AB

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 9 September 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: Not specified
• Target Recruitment: 10

Inclusion Criteria

1. A male or female patient with a clinical diagnosis of BA
2. Age at Kasai HPE <=90 days
3. Eligible to start treatment within 3 weeks post-Kasai HPE

Exclusion Criteria

• Patients with intractable ascites
• Ileal resection surgery
• ALT >=10× upper limit of normal (ULN) at Screening
• Patient reliant on total parenteral nutrition, or not able to take study drug
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, and if INR
is <=1.6 at resampling the patient may be randomized)
• Patient has had exposure to an investigational drug or biologic agent
within 30 days prior to randomization, or 10 half-lives of the study drug,
whichever is longer
• 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
• Known hypersensitivity to odevixibat or any component of the drug formulation

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>The primary efficacy endpoint is the time from randomization to first<br /><br>occurrence of liver transplant, or death, during the 104-week treatment period.</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>• Proportion of patients who are alive and have not undergone a liver<br /><br>transplant after 104 weeks.pharmacotherapeutic or procedural intervention such<br /><br>as chronic diuretics or paracentesis.<br /><br>• Time to onset of first sentinel event during the 104-week treatment period.<br /><br>Sentinel events are defined in the protocol.<br /><br>• Total bilirubin level after 13, 26, 52, and 104 weeks.<br /><br>• Serum bile acid level after 13, 26, 52, and 104 weeks.<br /><br>• Time to pediatric end-stage liver disease (PELD) score >=15.<br /><br>• Safety parameters including AEs, SAEs, findings on physical examination,<br /><br>laboratory assessments (including fat-soluble vitamins and lipids) and<br /><br>abdominal ultrasound.</p><br>