Clinical trial NCT05426733: An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat in Children With Biliary Atresia
Official title: An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat (A4250) in Children With Biliary Atresia (BOLD-EXT)
Study type: INTERVENTIONAL
Overall status: ENROLLING_BY_INVITATION
Phase: PHASE3
Conditions: Biliary Atresia
Lead sponsor: Ipsen
Sponsor class: INDUSTRY
First posted: 2022-06-22
Enrollment: 180
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.

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

Primary outcomes:
measure: Proportion of patients who are alive and have not undergone liver transplant
timeFrame: From baseline to Week 104
Secondary outcomes:
measure: Change in aspartate aminotransferase (AST) to platelet ratio index (APRI) score
timeFrame: From baseline to Week 104
measure: Change in Fibrosis-4 (Fib-4) score
timeFrame: From baseline to Week 104
measure: Change in serum bile acids
timeFrame: From Baseline to Weeks 26, 52, 78, and 104
measure: Change in height
timeFrame: From Baseline to Weeks 26, 52, 78 and 104
measure: Change in weight
timeFrame: From Baseline to Weeks 26, 52, 78 and 104
measure: Change in mid-arm circumference
timeFrame: From Baseline to Weeks 26, 52, 78 and 104
measure: Time to onset of any sentinel events
timeFrame: From Baseline to Week 104
measure: Change in pediatric end-stage liver disease (PELD) score
timeFrame: From Baseline to Weeks 26, 52, 78 and 104
Other outcomes:
Locations:
city: Aurora
country: United States
facility: Children's Hospital Colorado
state: Colorado
city: Atlanta
country: United States
facility: Children's Healhcare of Atlanta- Emory University school of medicine
state: Georgia
city: Indianapolis
country: United States
facility: Indiana University school of Medicine
state: Indiana
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Kansas City
country: United States
facility: Children's Mercy Hospital and Clinics
state: Missouri
city: Bronx
country: United States
facility: The Childrens Hospital at Montefiore Albert Einstein School of Medicine
state: New York
city: New York
country: United States
facility: Hassenfeld Children's Hospital at NYU Langone
state: New York
city: New York
country: United States
facility: Columbia University Medical Center New York Presbyterian Morgan Stanley-Komansky Childrens Hospital
state: New York
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
city: Parkville
country: Australia
facility: Royal Children's Hospital
city: Montréal
country: Canada
facility: CHU Sainte-Justine
city: Toronto
country: Canada
facility: The Hospital for Sick Children
city: Guangdong
country: China
facility: Guangzhou Women And Childrens Medical Center
city: Shanghai
country: China
facility: Children's Hospital of Fudan University
city: Le Kremlin-Bicêtre
country: France
facility: Bicêtre Hospital
city: Berlin
country: Germany
facility: Charité - Universitätsmedizin Berlin
city: Tuebingen
country: Germany
facility: University Children´s Hospital Tuebingen
city: Bergamo
country: Italy
facility: ASST Papa Giovanni XXIII
city: Padova
country: Italy
facility: Azienda Ospedaliera di Padova
city: Seoul
country: Korea, Republic of
facility: Seoul National University Childrens Hospital
city: Kota Bharu
country: Malaysia
facility: Hospital Raja Perempuan Zainab II
city: Kuala Lumpur
country: Malaysia
facility: University Malaya Medical Centre
city: Groningen
country: Netherlands
facility: University Medical Center Groningen
city: Auckland
country: New Zealand
facility: Starship Child Health, Te Whatu Ora - Health New Zealand
city: Warsaw
country: Poland
facility: Instytut Pomnik-Centrum Zdrowia Dziecka
city: Barcelona
country: Spain
facility: Hospital Universitari Vall d'Hebron
city: Ankara
country: Turkey
facility: Hacettepe İhsan Doğramacı Children's Hospital
city: Antalya
country: Turkey
facility: Akdeniz University Medical Faculty
city: Istanbul
country: Turkey
facility: Istanbul University Istanbul Medical Faculty Hospital
Study officials:
References: