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Enrolling by Invitation

NCT Number: NCT05426733

An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat in Children With Biliary Atresia

An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat (A4250) in Children with Biliary Atresia

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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

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Royal Children's Hospital, Parkville, Australia

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About this study

This is a Phase 3, multi-center, open-label extension study to evaluate the long-term efficacy and safety of odevixibat in patients with Biliary Atresia (BA). Patients who completed treatment in the A4250-011 BOLD study and meet eligibility criteria for Study A4250-016 (BOLD-EXT) can participate. The duration of the treatment period is 104 weeks, followed by a 4-week Safety Follow-up Period. Patients who wish to continue receiving odevixibat after 104 weeks can remain on treatment in the optional extension period (OEP).

Up to 180 patients will be enrolled at approximately 70 sites in the North America, Europe, the 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

  • Completion of the 104-week Treatment Period of Study A4250-011
  • Signed informed consent by caregiver

Exclusion criteria

  • Patients who were not compliant with study drug treatment or procedures in Study A4250-011 as per the investigator's discretion
  • Any conditions or abnormalities which, in the opinion of the investigator, may compromise the safety of the patient, or interfere with the patient participating in or completing the study
  • Known hypersensitivity to any components of odevixibat
  • Patients who are scheduled for a liver transplant or are likely to require a liver transplant in the immediate future based on the investigator's judgment

Treatment and study plan

Odevixibat

Drug

Odevixibat is a small molecule inhibitor of the ileal bile acid transporter (IBAT).

Other names: A4250

Primary outcomes

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

    Time frame: From baseline to Week 104

Secondary outcomes

  1. Change in aspartate aminotransferase (AST) to platelet ratio index (APRI) score

    Time frame: From baseline to Week 104

    The APRI (aspartate aminotransferase to platelet ratio index) score estimates the severity of liver fibrosis. A higher score indicates a greater amount of scarring (fibrosis) in the liver.

    • A score ≤ 0.5 indicates a low likelihood of fibrosis
    • A score ≥ 1.5 indicates a higher probability of fibrosis

  2. Change in Fibrosis-4 (Fib-4) score

    Time frame: From baseline to Week 104

    The Fib-4 score provides an estimate of the amount of scarring (fibrosis) in the liver. A higher score indicates a greater amount of scarring in the liver.

    • A score < 1.45 indicates a low likelihood of fibrosis
    • A score > 3.25 indicates a higher probability of fibrosis

  3. Change in serum bile acids

    Time frame: From Baseline to Weeks 26, 52, 78, and 104

  4. Change in height

    Time frame: From Baseline to Weeks 26, 52, 78 and 104

  5. Change in weight

    Time frame: From Baseline to Weeks 26, 52, 78 and 104

  6. Change in mid-arm circumference

    Time frame: From Baseline to Weeks 26, 52, 78 and 104

    Measure of growth

  7. Time to onset of any sentinel events

    Time frame: From Baseline to Week 104

  8. Change in pediatric end-stage liver disease (PELD) score

    Time frame: From Baseline to Weeks 26, 52, 78 and 104

    The PELD (pediatric end-stage liver disease) score is a measure of disease severity. PELD scores can range from a negative value (such as -10) to high numbers (such as 45). A higher score indicates more severe disease.

Sponsors and collaborators

Lead sponsor

Albireo, an Ipsen Company

Industry

Registry information

Official study title

An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat (A4250) in Children With Biliary Atresia (BOLD-EXT)

Acronym: BOLD-EXT

Important dates

Study start
2022
Primary completion
2028
Study completion
2028
First posted
Jun 22, 2022
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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