Study of the Safety, Pharmacodynamics and Efficacy of ANB-002 in Patients With Hemophilia B (SAFRAN)
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, dose 1, ANB-002, dose 2, ANB-002, dose 3.
- 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
- Беларусь, Россия
- Следующий шаг
- Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →
Не всё понятно в терминах? Прочитайте наш гид для пациентов →
Официальное название
An Open-label, Non-comparative, Single Dose-Escalation Study of the Safety, Pharmacodynamics and Efficacy of ANB-002 in Patients With Hemophilia B
Обзор
The goal of this multicenter, two-stage, open-label study is to investigate the safety, immunogenicity, and efficacy of ANB-002 in subjects with hemophilia В. The study will have a dose-escalation design with elements of phase I/II seamless adaptive design.
Подробное описание
The study design includes ANB-002 dose escalation in at three cohorts. In Cohort 1, the subject is treated with a single dose of ANB-002 (dose 1) administered as an intravenous infusion. Follow-up for the assessment of dose-limiting toxicity (DLT) will be carried out for 28 days. If no DLT events are observed in the subject of Cohort 1, the following subjects will be included in Cohort 1.
In Cohort 2, the subjects are treated with a single dose of ANB-002 (dose 2). In Cohort 3, the subject are treated with a single dose of ANB-002 (dose 3).
The decision to continue enrolling in cohorts will be made at the Independent Data Monitoring Committee (IDMC) meeting. After the IDMC makes a decision regarding the dosing subjects, further enrolment to the cohort will be carried out.
Based on the data from the follow-up period of subjects included in Сohorts 1-3, a potential therapeutic dose for further study will be determined and additional patients will be included to recieve this dose.
In exploratory Cohort 4 patients with anti-AAV5 antibodies and/or hepatitis B in anamnesis will be included. These subjects will recieve the dose 3 of ANB-002.
The total duration of participation of one subjects in the study will be 5 years.
Вмешательства
- Генная терапия ANB-002, dose 1
Adeno-associated viral vector carrying the FIX gene single infusion at dose 1. - Генная терапия ANB-002, dose 2
Adeno-associated viral vector carrying the FIX gene single infusion at dose 2. - Генная терапия ANB-002, dose 3
Adeno-associated viral vector carrying the FIX gene single infusion at dose 3.
Первичные конечные точки
- Change in FIX activity from baseline [Срок оценки: 12 months; final assessment - 5 years]
- Proportion of subjects with adverse reactions [Срок оценки: 12 months; final assessment - 5 years]
Вторичные конечные точки (9)
- Changes in FIX activity [Срок оценки: 5 years]
- Annualized use of FIX concentrates [Срок оценки: 5 years]
- Annualized bleeding rate [Срок оценки: 5 years]
- Annualized rate of bleedings requiring therapy with FIX concentrates [Срок оценки: 5 years]
- Changes in Haemo-A-QoL (Hemophilia-Specific Quality of Life) scores from baseline [Срок оценки: 5 years]
- Changes in EuroQol-5D-3L (European Quality of Life Questionnaire) scores from baseline [Срок оценки: 5 years]
- Changes in SF-36 (Short Form-36) scores from baseline [Срок оценки: 5 years]
- Response duration based on FIX activity [Срок оценки: 5 years]
- Evaluation of the condition of joints based on the Hemophilia Joint Health Score (HJHS) [Срок оценки: 5 years]
Критерии участия
Критерии включения
- Male with hemophilia B.
- Age ≥18 years.
- FIX activity at screening ≤2% without FIX inhibitor.
- ≥150 previous exposure days of treatment with FIX concentrates.
Критерии исключения
- Previous gene therapy.
- Other blood or hematopoietic disorders.
- Positive Anti-AAV5 antibodies (for Cohorts 1-3).
- Diagnosed HIV-infection, not controlled with anti-viral therapy.
- Hepatitis B (for Cohorts 1-3), acute or chronic hepatitis C.
- Any active systemic infections or recurrent infections requiring systemic therapy at screening.
- Any other disorders associated with severe immunodeficiency.
- Significant hepatic disorders (liver cirrhosis, liver fibrosis, etc).
- Malignancies with remission <5 years.
Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.
Здоровые добровольцы: Нет
Дизайн исследования
- Распределение
- Нерандомизированное
- Модель
- Последовательный дизайн
- Маскирование
- Открытое
- Основная цель
- Лечение
Центры проведения
Россия · 15 центров
- State Autonomous Institution for Healthcare "Chelyabinsk Regional Clinical Hospital" — Chelyabinsk
- State budgetary healthcare institution Leningrad Regional Clinical Hospital — Gatchina
- Kuzbass Clinical Hospital named after S.V. Belyaev — Kemerovo
- 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
- LLC "Medis" — Nizhny Novgorod
- … и ещё 7 центров
Беларусь · 2 центра
- Republican Scientific and Practical Center for Radiation Medicine and Human Ecology — Homyel
- Minsk Scientific and Practical Center for Surgery, Transplantology and Hematology — Minsk
Идентификаторы
NCT: NCT06120582 · ANB-002-1