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

Completed · Phase 3 · Has a placebo group

Conditions studied: Biliary Atresia

In brief

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.

Key facts

Study ID

NCT04336722

Run by

Albireo, an Ipsen Company

People needed

254

Starts

2020-07-08

Expected to finish

2026-06-22

Last updated by the study team

2026-07-02

Who can join

Age: any, up to 0. Sex: any. Healthy volunteers: not accepted.

You may qualify 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
• 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

Where it is running

• Stanford Children's Health — Palo Alto, California, United States
• Rady Children's Hospital — San Diego, California, United States
• UCSF Benioff Children's Hospital San Francisco — San Francisco, California, United States
• Children's Hospital Colorado — Aurora, Colorado, United States
• Nemours/Alfred I. duPont Hospital for Children — Wilmington, Delaware, United States
• Children's National Medical Center — Washington D.C., District of Columbia, United States
• University of Miami — Miami, Florida, United States
• Children's Healthcare of Atlanta - Emory University School of Medicine — Atlanta, Georgia, United States
• Ann & Robert H. Lurie Children's Hospital — Chicago, Illinois, United States
• Indiana University school of Medicine — Indianapolis, Indiana, United States
• Johns Hopkins Children's Center — Baltimore, Maryland, United States
• Boston Children's Hospital — Boston, Massachusetts, United States
• University of Michigan Children's Hospital — Ann Arbor, Michigan, United States
• Children's Mercy Hospital — Kansas City, Missouri, United States
• Washington University in St. Louis — St Louis, Missouri, United States
• NYU Grossman school of Medicine — New York, New York, United States
• Icahn School of Medicine at Mount Sinai — New York, New York, United States
• NewYork-Presbyterian Morgan Stanley Children's Hospital — New York, New York, United States
• The Children's Hospital at Montefiore — The Bronx, New York, United States
• Duke Central Tower — Durham, North Carolina, United States
• Cincinnati Children's Hospital — Cincinnati, Ohio, United States
• Cleveland Clinic — Cleveland, Ohio, United States
• Oregon Health & Science University — Portland, Oregon, United States
• The Children´s Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• Children's Hospital Los Angeles — Los Angeles, California, United States

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.