An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat in Children With Biliary Atresia
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Odevixibat.
- Who it may be relevant to
- Registry conditions: Biliary Atresia. Basic parameters: No limits · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Australia, Canada, China, France +9
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat (A4250) in Children With Biliary Atresia (BOLD-EXT)
Overview
An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat (A4250) in Children with Biliary Atresia
Detailed description
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.
Interventions
- Drug Odevixibat
Odevixibat is a small molecule inhibitor of the ileal bile acid transporter (IBAT).
Primary outcome measures
- Proportion of patients who are alive and have not undergone liver transplant [Time frame: From baseline to Week 104]
Secondary outcome measures (8)
- Change in aspartate aminotransferase (AST) to platelet ratio index (APRI) score [Time frame: From baseline to Week 104]
- Change in Fibrosis-4 (Fib-4) score [Time frame: From baseline to Week 104]
- Change in serum bile acids [Time frame: From Baseline to Weeks 26, 52, 78, and 104]
- Change in height [Time frame: From Baseline to Weeks 26, 52, 78 and 104]
- Change in weight [Time frame: From Baseline to Weeks 26, 52, 78 and 104]
- Change in mid-arm circumference [Time frame: From Baseline to Weeks 26, 52, 78 and 104]
- Time to onset of any sentinel events [Time frame: From Baseline to Week 104]
- Change in pediatric end-stage liver disease (PELD) score [Time frame: From Baseline to Weeks 26, 52, 78 and 104]
Eligibility criteria
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United States · 10 centers
- Children's Hospital Colorado — Aurora
- Children's Healhcare of Atlanta- Emory University school of medicine — Atlanta
- Indiana University school of Medicine — Indianapolis
- Boston Children's Hospital — Boston
- Children's Mercy Hospital and Clinics — Kansas City
- Hassenfeld Children's Hospital at NYU Langone — New York
- Columbia University Medical Center New York Presbyterian Morgan Stanley-Komansky Childrens — New York
- The Childrens Hospital at Montefiore Albert Einstein School of Medicine — The Bronx
- … and 2 more centers
Turkey (Türkiye) · 3 centers
- Hacettepe İhsan Doğramacı Children's Hospital — Ankara
- Akdeniz University Medical Faculty — Antalya
- Istanbul University Istanbul Medical Faculty Hospital — Istanbul
Canada · 2 centers
- CHU Sainte-Justine — Montreal
- The Hospital for Sick Children — Toronto
China · 2 centers
- Guangzhou Women And Childrens Medical Center — Guangdong
- Children's Hospital of Fudan University — Shanghai
Germany · 2 centers
- Charité - Universitätsmedizin Berlin — Berlin
- University Children´s Hospital Tuebingen — Tübingen
Italy · 2 centers
- ASST Papa Giovanni XXIII — Bergamo
- Azienda Ospedaliera di Padova — Padova
Malaysia · 2 centers
- Hospital Raja Perempuan Zainab II — Kota Bharu
- University Malaya Medical Centre — Kuala Lumpur
Australia · 1 center
- Royal Children's Hospital — Parkville
France · 1 center
- Bicêtre Hospital — Le Kremlin-Bicêtre
Netherlands · 1 center
- University Medical Center Groningen — Groningen
New Zealand · 1 center
- Starship Child Health, Te Whatu Ora - Health New Zealand — Auckland
Poland · 1 center
- Instytut Pomnik-Centrum Zdrowia Dziecka — Warsaw
South Korea · 1 center
- Seoul National University Childrens Hospital — Seoul
Spain · 1 center
- Hospital Universitari Vall d'Hebron — Barcelona
Identifiers
NCT: NCT05426733 · A4250-016 · 2022-501090-39-00