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NCT Number: NCT04336722

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

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.

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Key information

Conditions

Biliary Atresia Bile Duct Diseases Biliary Tract Diseases Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Abnormalities Digestive System Diseases

Age range

Up to 111 day

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Women's and Children's Hospital (Women's and Children's Health Network, Incorporated.), North Adelaide, South Australia, Australia

Loading trial locations.

About this study

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

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Odevixibat

Drug

Odevixibat is a small molecule and selective inhibitor of IBAT.

Placebo

Drug

Placebo identical in appearance to experimental drug (odevixibat).

Primary outcomes

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

    Time frame: From baseline to Week 104

Secondary outcomes

  1. Proportion of patients with liver transplant

    Time frame: From baseline to Week 104

    Proportion of patients who are alive and have not undergone a liver transplant

  2. Time to onset of any sentinel events

    Time frame: From baseline to Week 104

    Time to onset of any sentinel events

  3. Total bilirubin levels

    Time frame: From baseline to Weeks 13, 26, 52 and 104

    Total bilirubin level after 13, 26, 52, and 104 weeks of study treatment

  4. Serum bile acid levels

    Time frame: From baseline to Weeks 13, 26, 52 and 104

    Serum bile acid level after 13, 26, 52, and 104 weeks of study treatment

  5. Time to pediatric end-stage liver disease (PELD) score >15

    Time frame: From baseline to Week 104

    Time to pediatric end-stage liver disease (PELD) score >15

  6. Percentage of participants experiencing Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Time frame: From baseline to Week 104

    An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention.

  7. Percentage of participants with clinically significant changes in Physical Examination

    Time frame: From baseline to Week 104

    Percentage of participants with clinically significant changes in physical examination findings will be reported. The clinical significance will be graded by the investigator.

  8. Percentage of participants with clinically significant changes in Laboratory Parameters (blood chemistry, hematology and coagulation)

    Time frame: From baseline to Week 104

    Percentage of participants with clinically significant change in laboratory parameters (blood chemistry, hematology and coagulation) will be reported. The clinical significance will be decided by the investigator.

  9. Percentage of participants with clinically significant changes in Abdominal Ultrasound findings

    Time frame: From baseline to Week 26 and Week 104

    Percentage of participants with clinically significant change in Abdominal Ultrasound findings will be reported. The clinical significance will be decided by the investigator.

Sponsors and collaborators

Lead sponsor

Albireo, an Ipsen Company

Industry

Registry information

Official study 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

Important dates

Study start
2020
Primary completion
2026
Study completion
2026
First posted
Apr 7, 2020
Registry last updated
Jul 2, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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