An Open-Label, Comparative Study of the Efficacy, Safety and Pharmacodynamics of Single Dose of ANB-002 in Patients With Hemophilia B
Ориентир для пациента и семьи
Простыми словами
Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.
- Что изучают
- В протоколе указаны: ANB-002.
- Кому может быть актуально
- Состояния в реестре: Hemophilia B. Базовые параметры: от 18 лет · Мужчины.
- Что важно проверить
- Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
- Где проводится
- Россия, Таиланд
- Следующий шаг
- Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →
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Обзор
The aim of the study is to demonstrate non-inferiority of ANB-002 compared with preventive use of coagulation factor IX (FIX) in adult subjects with hemophilia B with FIX activity ≤2% and without FIX inhibitor. The study will have an open-label single-arm design.
Подробное описание
After signing the ICF and screening examination the subjects are to be included in the Non-interventional lead-in period in which the subject will receive standard FIX prevention. The lead-in period will last for at least 6 month for every subject.
After completiong the lead-in period subjects will enter the main (interventional) period.
At the first visit of the main period subjects will recieve investigational product ANB-002.
The main period ends 18 months after the administration of ANB-002, after which subjects will switch to the follow-up period and will be evaluated up to 5 years after the ANB-002 infusion.
Вмешательства
- Генная терапия ANB-002
Adeno-associated viral vector carrying the FIX gene single infusion
Первичные конечные точки
- Annualized bleeding rate (ABR) before and after a single dose of ANB-002 [Срок оценки: Lead-in period: at least 6 months prior to ANB-002 infusion (Lead-in arm); Month 12 to Month 18 post infusion (ANB-002 arm)]
Вторичные конечные точки (12)
- FIX activity [Срок оценки: 18 months after drug administration; final assessment - 5 years]
- Annualized FIX consumption [Срок оценки: 18 months after drug administration; final assessment - 5 years]
- Proportion of subjects not receiving FIX prevention [Срок оценки: 18 months after drug administration]
- Annualized bleeding rate (ABR) [Срок оценки: 18 months after drug administration; final assessment - 5 years]
- Proportion of subjects with spontaneous bleedings [Срок оценки: 18 months after drug administration; final assessment - 5 years]
- Annualized rate of spontaneous bleedings [Срок оценки: 18 months after drug administration; final assessment - 5 years]
- Proportion of subjects with hemarthrosis [Срок оценки: 18 months after drug administration; final assessment - 5 years]
- Annualized hemarthrosis rate [Срок оценки: 18 months after drug administration; final assessment - 5 years]
- Proportion of subjects with bleedings requiring FIX concentrate therapy [Срок оценки: 18 months after drug administration; final assessment - 5 years]
- Annualized rate of bleedings requiring therapy with FIX concentrates [Срок оценки: 18 months after drug administration; final assessment - 5 years]
- Proportion of subjects without bleeding within 12 months after stabilization of endogenous FIX activity [Срок оценки: 12 months after stabilization of endogenous FIX activity]
- Proportion of subjects achieving clinical response [Срок оценки: 18 months after drug administration]
Критерии участия
Критерии включения
- Men diagnosed with hemophilia B aged 18 or older
- FIX activity ≤2%
- Absense of FIX inhibitor
- ≥150 previous exposure days of treatment with FIX concentrates
Критерии исключения
- Any diseases of blood and hematopoietic organs other than hemophilia B
- A history of any gene therapy, including ANB-002
- Diagnosed HIV-infection, not controlled with anti-viral therapy
- Active HBV or HCV infection
- Anti-AAV5 antibodies
- Any active systemic infections or recurrent infections requiring systemic therapy
- Any other disorders associated with severe immunodeficiency
- Relevant hepatic disorders or conditions that can be a symptom of existing liver disorder
- Malignancies with less than 5 years of remission
Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.
Здоровые добровольцы: Нет
Дизайн исследования
- Распределение
- Нерандомизированное
- Модель
- Одна группа
- Маскирование
- Открытое
- Основная цель
- Лечение
Центры проведения
Россия · 11 центров
- Chelyabinsk Regional Clinical Hospital — Chelyabinsk
- Federal State Budgetary Organization of Science "Kirov Research Institute of Hematology an — Kirov
- Federal State Budgetary Institution "National Medical Research Centre for Hematology" of t — Moscow
- Federal State Budgetary Institution "National Medical Research Centre for Hematology" of t — Moscow
- Federal Budgetary Institution "Moscow City Clinical Hospital named after S. P. Botkin" — Moscow
- State Novosibirsk Regional Clinical Hospital — Novosibirsk
- State budgetary healthcare institution Leningrad Regional Clinical Hospital — Saint Petersburg
- Russian Research Institute of Hematology and Transfusiology Federal State Institution of F — Saint Petersburg
- … и ещё 3 центра
Таиланд · 3 центра
- King Chulalongkorn Memorial Hospital — Bangkok
- Ramathibodi Hospital — Bangkok
- Siriraj Hospital — Bangkok
Идентификаторы
NCT: NCT06700096 · ANB-002-2