This Study Will Investigate the Efficacy and Safety of Odevixibat in Children with Biliary Atresia Who Have Undergone a Kasai HPE

Phase 1

• Conditions: Biliary Atresia; MedDRA version: 20.0Level: LLTClassification code 10004653Term: Biliary atresiaSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2019-003807-37-DE
• Lead Sponsor: Albireo AB

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-05-15
• Last Update Posted: 12 March 2024

Recruitment & Eligibility

• Status: A
• Sex: All
• Target Recruitment: 245

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
Are the trial subjects under 18? yes
Number of subjects for this age range: 245
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

- Patients with intractable ascites
- Ileal resection surgery
- ALT =10× upper limit of normal (ULN) at screening
- Patient 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; 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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: The primary objective is to evaluate the efficacy of repeated once-daily doses of odevixibat versus placebo in children with biliary atresia (BA) post Kasai hepatoportoenterostomy(HPE) based native liver survival (NLS) of up to 104 weeks.;Secondary Objective: To evaluate the effect of odevixibat compared to placebo on the time to onset of sentinel events<br> <br>To evaluate the effect of odevixibat compared to placebo on total bilirubin after 13, 26, 52, and 104 weeks <br><br>To evaluate the effect of odevixibat compared to placebo on serum bile acids after 13, 26, 52, and 104 weeks <br><br>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;Primary end point(s): The primary efficacy endpoint is the time from randomization to first occurrence of liver transplant, or death, during the 104 week treatment period.;Timepoint(s) of evaluation of this end point: 104 weeks

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): 1. Proportion of patients who are alive and have not undergone a liver transplant after 104 weeks.<br>2. Time to onset of first sentinel event during the 104-week treatment period. Sentinel events are defined in the protocol<br>3. Total bilirubin level after 13, 26, 52, and 104 weeks.<br>4. Serum bile acid level after 13, 26, 52, and 104 weeks.<br>5. Time to pediatric end-stage liver disease (PELD) score =15.<br>6. Safety parameters including AEs, SAEs, findings on physical examination, laboratory assessments (including fat-soluble vitamins and lipids) and abdominal ultrasound.;Timepoint(s) of evaluation of this end point: 13, 26, 52 and 104 weeks.