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

Albireo, an Ipsen Company102 sites in 10 countries254 target enrollmentJuly 8, 2020

ConditionsBiliary Atresia

InterventionsOdevixibat Placebo

DrugsOdevixibat Placebo

Overview

Phase: Phase 3
Intervention: Odevixibat
Conditions: Biliary Atresia
Sponsor: Albireo, an Ipsen Company
Enrollment: 254
Locations: 102
Primary Endpoint: Time from randomization to first occurrence of liver transplant, or death
Status: Active, not recruiting
Last Updated: 2 months ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

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.

Detailed Description

Up to 70 sites will be initiated for this study in North America, Europe, Middle East, and Asia Pacific.

Registry

clinicaltrials.gov

Start Date

July 8, 2020

End Date

June 26, 2026

Last Updated

2 months ago

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

Albireo, an Ipsen Company

Responsible Party

Sponsor

Eligibility 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

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

Arms & Interventions

Odevixibat (A4250)

Capsules for oral administration once daily for 104 weeks.

Intervention: Odevixibat

Placebo

Capsules for oral administration (to match active) once daily for 104 weeks.

Intervention: Placebo

Outcomes

Primary Outcomes

Time from randomization to first occurrence of liver transplant, or death

Time Frame: From baseline to Week 104

Secondary Outcomes

Time to onset of any sentinel events(From baseline to Week 104)
Percentage of participants with clinically significant changes in Physical Examination(From baseline to Week 104)
Serum bile acid levels(From baseline to Weeks 13, 26, 52 and 104)
Time to pediatric end-stage liver disease (PELD) score >15(From baseline to Week 104)
Percentage of participants experiencing Adverse Events (AEs) and Serious Adverse Events (SAEs)(From baseline to Week 104)
Percentage of participants with clinically significant changes in Laboratory Parameters (blood chemistry, hematology and coagulation)(From baseline to Week 104)
Percentage of participants with clinically significant changes in Abdominal Ultrasound findings(From baseline to Week 26 and Week 104)
Proportion of patients with liver transplant(From baseline to Week 104)
Total bilirubin levels(From baseline to Weeks 13, 26, 52 and 104)