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

Официальное название

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

Обзор

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

Подробное описание

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.

Вмешательства

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

Первичные конечные точки

• Proportion of patients who are alive and have not undergone liver transplant [Срок оценки: From baseline to Week 104]

Вторичные конечные точки (8)

• Change in aspartate aminotransferase (AST) to platelet ratio index (APRI) score [Срок оценки: From baseline to Week 104]
• Change in Fibrosis-4 (Fib-4) score [Срок оценки: From baseline to Week 104]
• Change in serum bile acids [Срок оценки: From Baseline to Weeks 26, 52, 78, and 104]
• Change in height [Срок оценки: From Baseline to Weeks 26, 52, 78 and 104]
• Change in weight [Срок оценки: From Baseline to Weeks 26, 52, 78 and 104]
• Change in mid-arm circumference [Срок оценки: From Baseline to Weeks 26, 52, 78 and 104]
• Time to onset of any sentinel events [Срок оценки: From Baseline to Week 104]
• Change in pediatric end-stage liver disease (PELD) score [Срок оценки: From Baseline to Weeks 26, 52, 78 and 104]

Критерии участия

Критерии включения

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

Критерии исключения

• 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

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

США · 10 центров

• 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
• … и ещё 2 центра

Turkey (Türkiye) · 3 центра

• Hacettepe İhsan Doğramacı Children's Hospital — Ankara
• Akdeniz University Medical Faculty — Antalya
• Istanbul University Istanbul Medical Faculty Hospital — Istanbul

Канада · 2 центра

• CHU Sainte-Justine — Montreal
• The Hospital for Sick Children — Toronto

Китай · 2 центра

• Guangzhou Women And Childrens Medical Center — Guangdong
• Children's Hospital of Fudan University — Шанхай

Германия · 2 центра

• Charité - Universitätsmedizin Berlin — Berlin
• University Children´s Hospital Tuebingen — Tübingen

Италия · 2 центра

• ASST Papa Giovanni XXIII — Bergamo
• Azienda Ospedaliera di Padova — Padova

Малайзия · 2 центра

• Hospital Raja Perempuan Zainab II — Kota Bharu
• University Malaya Medical Centre — Kuala Lumpur

Австралия · 1 центр

• Royal Children's Hospital — Parkville

Франция · 1 центр

• Bicêtre Hospital — Le Kremlin-Bicêtre

Нидерланды · 1 центр

• University Medical Center Groningen — Groningen

Новая Зеландия · 1 центр

• Starship Child Health, Te Whatu Ora - Health New Zealand — Auckland

Польша · 1 центр

• Instytut Pomnik-Centrum Zdrowia Dziecka — Warsaw

South Korea · 1 центр

• Seoul National University Childrens Hospital — Seoul

Испания · 1 центр

• Hospital Universitari Vall d'Hebron — Barcelona

Идентификаторы

NCT: NCT05426733 · A4250-016 · 2022-501090-39-00

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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