Ignite Creation Date:
2025-12-25 @ 3:00 AM
Ignite Modification Date:
2025-12-31 @ 5:58 AM
Study NCT ID:
NCT05426733
Status:
ENROLLING_BY_INVITATION
Last Update Posted:
2025-12-03
First Post:
2022-06-13
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat in Children With Biliary Atresia
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D001656', 'term': 'Biliary Atresia'}], 'ancestors': [{'id': 'D001649', 'term': 'Bile Duct Diseases'}, {'id': 'D001660', 'term': 'Biliary Tract Diseases'}, {'id': 'D004066', 'term': 'Digestive System Diseases'}, {'id': 'D004065', 'term': 'Digestive System Abnormalities'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000713258', 'term': 'odevixibat'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 180}}, 'statusModule': {'overallStatus': 'ENROLLING_BY_INVITATION', 'startDateStruct': {'date': '2022-07-05', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-11', 'completionDateStruct': {'date': '2028-08-31', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-11-25', 'studyFirstSubmitDate': '2022-06-13', 'studyFirstSubmitQcDate': '2022-06-16', 'lastUpdatePostDateStruct': {'date': '2025-12-03', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2022-06-22', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2028-08-31', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Proportion of patients who are alive and have not undergone liver transplant', 'timeFrame': 'From baseline to Week 104'}], 'secondaryOutcomes': [{'measure': 'Change in aspartate aminotransferase (AST) to platelet ratio index (APRI) score', 'timeFrame': 'From baseline to Week 104', 'description': '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.\n\n* A score ≤ 0.5 indicates a low likelihood of fibrosis\n* A score ≥ 1.5 indicates a higher probability of fibrosis'}, {'measure': 'Change in Fibrosis-4 (Fib-4) score', 'timeFrame': 'From baseline to Week 104', 'description': '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.\n\n* A score \\< 1.45 indicates a low likelihood of fibrosis\n* A score \\> 3.25 indicates a higher probability of fibrosis'}, {'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', 'description': 'Measure of growth'}, {'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', 'description': '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.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Biliary Atresia']}, 'descriptionModule': {'briefSummary': 'An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat (A4250) in Children with Biliary Atresia', 'detailedDescription': '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).\n\nUp to 180 patients will be enrolled at approximately 70 sites in the North America, Europe, the Middle East and Asia-Pacific.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Completion of the 104-week Treatment Period of Study A4250-011\n* Signed informed consent by caregiver\n\nExclusion Criteria:\n\n* Patients who were not compliant with study drug treatment or procedures in Study A4250-011 as per the investigator's discretion\n* 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\n* Known hypersensitivity to any components of odevixibat\n* 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"}, 'identificationModule': {'nctId': 'NCT05426733', 'acronym': 'BOLD-EXT', 'briefTitle': 'An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat in Children With Biliary Atresia', 'organization': {'class': 'INDUSTRY', 'fullName': 'Ipsen'}, 'officialTitle': 'An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat (A4250) in Children With Biliary Atresia (BOLD-EXT)', 'orgStudyIdInfo': {'id': 'A4250-016'}, 'secondaryIdInfos': [{'id': '2022-501090-39-00', 'type': 'CTIS'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Odevixibat (A4250)', 'description': 'Capsules for oral administration once daily for 104 weeks.', 'interventionNames': ['Drug: Odevixibat']}], 'interventions': [{'name': 'Odevixibat', 'type': 'DRUG', 'otherNames': ['A4250'], 'description': 'Odevixibat is a small molecule inhibitor of the ileal bile acid transporter (IBAT).', 'armGroupLabels': ['Odevixibat (A4250)']}]}, 'contactsLocationsModule': {'locations': [{'zip': '80045', 'city': 'Aurora', 'state': 'Colorado', 'country': 'United States', 'facility': "Children's Hospital Colorado", 'geoPoint': {'lat': 39.72943, 'lon': -104.83192}}, {'zip': '30322', 'city': 'Atlanta', 'state': 'Georgia', 'country': 'United States', 'facility': "Children's Healhcare of Atlanta- Emory University school of medicine", 'geoPoint': {'lat': 33.749, 'lon': -84.38798}}, {'zip': '46202', 'city': 'Indianapolis', 'state': 'Indiana', 'country': 'United States', 'facility': 'Indiana University school of Medicine', 'geoPoint': {'lat': 39.76838, 'lon': -86.15804}}, {'zip': '02115', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': "Boston Children's Hospital", 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '64108', 'city': 'Kansas City', 'state': 'Missouri', 'country': 'United States', 'facility': "Children's Mercy Hospital and Clinics", 'geoPoint': {'lat': 39.09973, 'lon': -94.57857}}, {'zip': '10016', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': "Hassenfeld Children's Hospital at NYU Langone", 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '10032', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'Columbia University Medical Center New York Presbyterian Morgan Stanley-Komansky Childrens Hospital', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '10467', 'city': 'The Bronx', 'state': 'New York', 'country': 'United States', 'facility': 'The Childrens Hospital at Montefiore Albert Einstein School of Medicine', 'geoPoint': {'lat': 40.84985, 'lon': -73.86641}}, {'zip': '45229', 'city': 'Cincinnati', 'state': 'Ohio', 'country': 'United States', 'facility': "Cincinnati Children's Hospital Medical Center", 'geoPoint': {'lat': 39.12711, 'lon': -84.51439}}, {'zip': '98105', 'city': 'Seattle', 'state': 'Washington', 'country': 'United States', 'facility': "Seattle Children's Hospital", 'geoPoint': {'lat': 47.60621, 'lon': -122.33207}}, {'zip': '3052', 'city': 'Parkville', 'country': 'Australia', 'facility': "Royal Children's Hospital", 'geoPoint': {'lat': -37.78333, 'lon': 144.95}}, {'city': 'Montreal', 'country': 'Canada', 'facility': 'CHU Sainte-Justine', 'geoPoint': {'lat': 45.50884, 'lon': -73.58781}}, {'city': 'Toronto', 'country': 'Canada', 'facility': 'The Hospital for Sick Children', 'geoPoint': {'lat': 43.70643, 'lon': -79.39864}}, {'city': 'Guangdong', 'country': 'China', 'facility': 'Guangzhou Women And Childrens Medical Center', 'geoPoint': {'lat': 42.76832, 'lon': 129.33635}}, {'city': 'Shanghai', 'country': 'China', 'facility': "Children's Hospital of Fudan University", 'geoPoint': {'lat': 31.22222, 'lon': 121.45806}}, {'city': 'Le Kremlin-Bicêtre', 'country': 'France', 'facility': 'Bicêtre Hospital', 'geoPoint': {'lat': 48.81471, 'lon': 2.36073}}, {'city': 'Berlin', 'country': 'Germany', 'facility': 'Charité - Universitätsmedizin Berlin', 'geoPoint': {'lat': 52.52437, 'lon': 13.41053}}, {'city': 'Tübingen', 'country': 'Germany', 'facility': 'University Children´s Hospital Tuebingen', 'geoPoint': {'lat': 48.52266, 'lon': 9.05222}}, {'city': 'Bergamo', 'country': 'Italy', 'facility': 'ASST Papa Giovanni XXIII', 'geoPoint': {'lat': 45.69601, 'lon': 9.66721}}, {'city': 'Padua', 'country': 'Italy', 'facility': 'Azienda Ospedaliera di Padova', 'geoPoint': {'lat': 45.40797, 'lon': 11.88586}}, {'city': 'Kota Bharu', 'country': 'Malaysia', 'facility': 'Hospital Raja Perempuan Zainab II', 'geoPoint': {'lat': 6.12361, 'lon': 102.24333}}, {'zip': '59100', 'city': 'Kuala Lumpur', 'country': 'Malaysia', 'facility': 'University Malaya Medical Centre', 'geoPoint': {'lat': 3.1412, 'lon': 101.68653}}, {'city': 'Groningen', 'country': 'Netherlands', 'facility': 'University Medical Center Groningen', 'geoPoint': {'lat': 53.21917, 'lon': 6.56667}}, {'zip': '1023', 'city': 'Auckland', 'country': 'New Zealand', 'facility': 'Starship Child Health, Te Whatu Ora - Health New Zealand', 'geoPoint': {'lat': -36.84853, 'lon': 174.76349}}, {'city': 'Warsaw', 'country': 'Poland', 'facility': 'Instytut Pomnik-Centrum Zdrowia Dziecka', 'geoPoint': {'lat': 52.22977, 'lon': 21.01178}}, {'city': 'Seoul', 'country': 'South Korea', 'facility': 'Seoul National University Childrens Hospital', 'geoPoint': {'lat': 37.566, 'lon': 126.9784}}, {'city': 'Barcelona', 'country': 'Spain', 'facility': "Hospital Universitari Vall d'Hebron", 'geoPoint': {'lat': 41.38879, 'lon': 2.15899}}, {'city': 'Ankara', 'country': 'Turkey (Türkiye)', 'facility': "Hacettepe İhsan Doğramacı Children's Hospital", 'geoPoint': {'lat': 39.91987, 'lon': 32.85427}}, {'city': 'Antalya', 'country': 'Turkey (Türkiye)', 'facility': 'Akdeniz University Medical Faculty', 'geoPoint': {'lat': 36.90812, 'lon': 30.69556}}, {'city': 'Istanbul', 'country': 'Turkey (Türkiye)', 'facility': 'Istanbul University Istanbul Medical Faculty Hospital', 'geoPoint': {'lat': 41.01384, 'lon': 28.94966}}]}, 'ipdSharingStatementModule': {'url': 'https://www.ipsen.com/science/clinical-trials/clinical-data-transparency/', 'timeFrame': 'Where applicable, data from eligible studies are available 6 months after the studied medicine and indication have been approved in the US and/or EU.', 'ipdSharing': 'YES', 'description': 'Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, annotated case report form, statistical analysis plan, and dataset specifications.\n\nPatient level data will be anonymized and study documents will be redacted to protect the privacy of study participants.', 'accessCriteria': "Further details on Ipsen's sharing criteria and process for sharing are available here (https://www.ipsen.com/science/clinical-trials/clinical-data-transparency/)."}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Albireo, an Ipsen Company', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}