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

Trial ID or NCT#

NCT04336722

Status

not recruiting iconNOT RECRUITING

Purpose

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.

Official Title

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

Eligibility Criteria

Ages Eligible for Study: Younger than 111 Days
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers: No
Inclusion Criteria:
  1. * 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
    1. Key
Exclusion Criteria:
  1. * 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
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Investigator(s)

Amrita Narang

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Contact

Amrita Narang, MD

View on ClinicalTrials.gov