This study is in progress, not accepting new patients

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

a study on Biliary Atresia

Summary

Location
at San Francisco, California and other locations
Dates
study started
completion around

Description

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.

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

Details

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

Keywords

Biliary Atresia, Kasai, Odevixibat, Odevixibat (A4250)

Eligibility

You can join if…

  • 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

You CAN'T join if...

  • 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

Locations

  • UCSF Benioff Children's Hospital San Francisco
    San Francisco California 94158 United States
  • Stanford Children's Health
    Palo Alto California 94304 United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Albireo, an Ipsen Company
ID
NCT04336722
Phase
Phase 3 Biliary Atresia Research Study
Study Type
Interventional
Participants
About 254 people participating
Last Updated