Efficacy and Safety of Odevixibat in Children With Biliary Atresia Who Have Undergone a Kasai HPE (BOLD)

• Status: Recruiting
• Trial end date: 2024-06-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Albireo

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

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