Эта страница была переведена автоматически, точность перевода не гарантируется. Пожалуйста, обратитесь к английской версии для исходного текста.

Efanesoctocog alfa prophylaxis у пациентов с гемофилией А с синовиальной гипертрофией (SYNOVIIIUS)

6 ноября 2026 г. обновлено: Sanofi

Проспективное интервенционное исследование эффективности профилактики эфанзоктоког альфа -гипертрофии у пациентов с гемофилией А

The objective of the study is to assess the improvement of synovial hypertrophy during the 12 months of efanesoctocog alfa prophylaxis once per week (QW) in joints with existing evidence of synovial hypertrophy in participants with hemophilia A.

The study duration for each participant is approximately 12 months.

Обзор исследования

Статус

Рекрутинг

Условия

Вмешательство/лечение

Тип исследования

Интервенционный

Регистрация (Оцененный)

35

Фаза

  • Фаза 4

Контакты и местонахождение

В этом разделе приведены контактные данные лиц, проводящих исследование, и информация о том, где проводится это исследование.

Контакты исследования

  • Имя: Trial Transparency email recommended (Toll free for US & Canada)
  • Номер телефона: option 6 800-633-1610
  • Электронная почта: Contact-US@sanofi.com

Места учебы

  • Австралия
    • Western Australia
      • Murdoch, Western Australia, Австралия, 6150
        • Завершенный
        • Investigational Site Number : 0360001
  • Канада
    • Alberta
      • Calgary, Alberta, Канада, T2N 1N4
        • Рекрутинг
        • Investigational Site Number : 1240002
    • Newfoundland and Labrador
      • St. John's, Newfoundland and Labrador, Канада, A1B 3V6
        • Рекрутинг
        • Investigational Site Number : 1240001
    • Ontario
      • Hamilton, Ontario, Канада, L8N 3Z5
        • Рекрутинг
        • Investigational Site Number : 1240003
      • Hamilton, Ontario, Канада, L8N 3Z5
        • Завершенный
        • Investigational Site Number : 1240004
  • Соединенные Штаты
    • California
      • Los Angeles, California, Соединенные Штаты, 90007
        • Завершенный
        • Orthopedic Institute for Children- Site Number : 8400009
      • Sacramento, California, Соединенные Штаты, 95817-1399
        • Рекрутинг
        • University of California Davis Medical Center-Site Number: 8400005
    • Indiana
      • Indianapolis, Indiana, Соединенные Штаты, 46260
        • Рекрутинг
        • Indiana Hemophilia and Thrombosis Center- Site Number : 8400006
    • Iowa
      • Iowa City, Iowa, Соединенные Штаты, 52242
        • Рекрутинг
        • University of Iowa- Site Number : 8400004
    • Ohio
      • Columbus, Ohio, Соединенные Штаты, 43203
        • Завершенный
        • The Ohio State University Wexner Medical Center - CarePoint East- Site Number : 8400001
    • Oregon
      • Portland, Oregon, Соединенные Штаты, 97239-3011
        • Рекрутинг
        • Oregon Health and Science University-Site Number: 8400002
    • Washington
      • Seattle, Washington, Соединенные Штаты, 98101-3932
        • Рекрутинг
        • Washington Center for Bleeding Disorders- Site Number : 8400008
  • Тайвань
      • Kaohsiung City, Тайвань, 80756
        • Рекрутинг
        • Investigational Site Number: 1580003
      • Taichung, Тайвань, 407
        • Рекрутинг
        • Investigational Site Number: 1580001
      • Taipei, Тайвань, 114
        • Рекрутинг
        • Investigational Site Number: 1580002
  • Япония
      • Hiroshima, Япония, 734-8551
        • Завершенный
        • Investigational Site Number : 3920002
      • Saitama, Япония, 330-8777
        • Рекрутинг
        • Investigational Site Number : 3920001
      • Tokyo, Япония, 160-0023
        • Завершенный
        • Investigational Site Number : 3920004
    • Kanagawa
      • Kawasaki, Kanagawa, Япония, 216-8511
        • Рекрутинг
        • Investigational Site Number : 3920003

Критерии участия

Исследователи ищут людей, которые соответствуют определенному описанию, называемому критериям приемлемости. Некоторыми примерами этих критериев являются общее состояние здоровья человека или предшествующее лечение.

Критерии приемлемости

Возраст, подходящий для обучения

12 лет и старше (Ребенок, Взрослый, Пожилой взрослый)

Принимает здоровых добровольцев

Нет

Полы, имеющие право на обучение

Все

Описание

Inclusion Criteria:

  • Participant is diagnosed with moderate to severe hemophilia A (defined as less than or equal to (≤)5 percent (%) of endogenous FVIII clotting activity) at the time of consent/assent.

  • Participant is more than or equal to (≥)12 years old at the time of consent/assent.

  • Participant has existing synovial hypertrophy, defined as at least 1 eligible* joint by the HEAD-US score (Synovitis score: 1 or 2) at the time of consent/assent.

  • Participant has at least 1 eligible* joint without planned future major orthopedic procedures (example, arthroscopic synovectomy, radioisotopic or chemical synoviorthesis), or major orthopedic procedures in the past 3 months prior to the screening visit (Visit 1).

    *An eligible joint is a joint with existing synovial hypertrophy, as defined by a HEAD-US synovitis score of 1 or 2, considering hypertrophic synovium as an indication of the presence of synovitis

  • Participant has received prophylactic treatment with hemophilia prophylaxis prescriptions in the last 12 months prior to the baseline visit (Visit 2).

  • Participant is capable of understanding the written Informed Consent Form (ICF)/assent form, provides a signed and witnessed written ICF/assent form, and agrees to comply with the protocol requirements.

  • If male, no contraceptive measures are required for this study.

  • If female, is not pregnant or breastfeeding, and one of the following conditions applies:

  • Is a women of nonchildbearing potential (WONCBP)

  • Is a WOCBP and agrees to use a highly effective contraceptive method, with a failure rate of less than (<)1 percent during the study treatment period (at least until the Week 52/ End of Treatment [EoT] visit).

  • A WOCBP must have a negative serum pregnancy test at the screening visit (Visit 1)

Exclusion Criteria:

  • Participants are excluded from the study if any of the following criteria apply:
  • Has other associated clotting disorders at the time of consent/assent.
  • Is already under efanesoctocog alfa treatment.
  • Has a current diagnosis of an factor VIII (FVIII) inhibitor, defined as inhibitor titer ≥0.60 BU/mL.
  • Has ITI within the last 2 years prior to the baseline visit (Visit 2).
  • Has been enrolled in a concurrent clinical interventional study or exposed to other investigational drug(s) within 3 months prior to screening for this study.
  • Is currently in an institution because of regulatory or legal order (that is, is a prisoner or a patient who is legally institutionalized).
  • Is not suitable for participation, whatever the reason, as judged by the investigator, including medical or clinical conditions, or patients potentially at risk of noncompliance to study procedures.
  • Is an employee or family member of the investigator or site personnel.
  • Is involved in a specific situation during study implementation or the course of the study that may raise ethics considerations.
  • Has hypersensitivity to efanesoctocog alfa or its components or any of its excipients that, in the opinion of the investigator, contraindicates participation in the study.

The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Учебный план

В этом разделе представлена ​​подробная информация о плане исследования, в том числе о том, как планируется исследование и что оно измеряет.

Как устроено исследование?

Детали дизайна

  • Основная цель: Уход
  • Распределение: Нерандомизированный
  • Интервенционная модель: Одногрупповое задание
  • Маскировка: Нет (открытая этикетка)

Оружие и интервенции

Группа участников / Армия
Вмешательство/лечение
Экспериментальный: Efanesoctocog Alfa

Participants will receive Efanesoctocog Alfa at 50 international unit per kilogram (IU/kg) intravenous (IV) QW
Лекарство: Efanesoctocog alfa

Pharmaceutical form:Lyophilized powder in a sterile vial that requires reconstitution with sterile water for injection (diluent)-Route of administration:Intravenous

Другие имена:
  • БИВВ001
  • Altuviiio®

Что измеряет исследование?

Первичные показатели результатов

Мера результата
Мера Описание
Временное ограничение
Proportion of joints with improvement in the Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) synovitis domain score
Временное ограничение: From Baseline up to Week 52

HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment.
From Baseline up to Week 52

Вторичные показатели результатов

Мера результата
Мера Описание
Временное ограничение
Change from baseline in the distribution of joint HEAD-US synovitis score
Временное ограничение: From Baseline up to Week 52

HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment.
From Baseline up to Week 52
Number of joints with the HEAD-US synovitis domain score that has remained unchanged or worsened
Временное ограничение: From Baseline up to Week 52

HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment.
From Baseline up to Week 52
Percentage of joints with the HEAD-US synovitis domain score that has remained unchanged or worsened
Временное ограничение: From Baseline up to Week 52

HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment.
From Baseline up to Week 52
Number of index joint type with the HEAD-US synovitis domain score that has improved, remained unchanged, or worsened
Временное ограничение: From Baseline up to Week 52

HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment.
From Baseline up to Week 52
Percentage of index joint type with the HEAD-US synovitis domain score that has improved, remained unchanged, or worsened
Временное ограничение: From Baseline up to Week 52

HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment.
From Baseline up to Week 52
Number of participants with at least 1 joint with the HEAD-US synovitis domain score that has improved, remained unchanged, or worsened
Временное ограничение: From Baseline up to Week 52

HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment.
From Baseline up to Week 52
Percentage of participants with at least 1 joint with the HEAD-US synovitis domain score that has improved, remained unchanged, or worsened
Временное ограничение: From Baseline up to Week 52

HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment.
From Baseline up to Week 52
Change from baseline in average HEAD-US synovitis domain score across all index joints
Временное ограничение: From Baseline up to Week 52

HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment.
From Baseline up to Week 52
Change from baseline in total/ domain scores of the HEAD-US
Временное ограничение: From Baseline up to Week 26

HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment.
From Baseline up to Week 26
Change from baseline in total/domain scores of the HJHS
Временное ограничение: From Baseline up to Week 26

HJHS comprises an assessment of specific features, or items, of the 6 index joints and an assessment of global gait. For each of the 6 joints, the following items are scored: swelling (0 to 3), duration of swelling (0 to 1), muscle atrophy (0 to 2), crepitus on motion (0 to 2), flexion loss (0 to 3), extension loss (0 to 3), joint pain (0 to 2), and strength (0 to 4). The maximum score for an individual index joint is 20. Gait is scored from 0 to 4. The maximum HJHS total score is 124, with a higher score indicating worse joint health.
From Baseline up to Week 26
Change from baseline in total/domain scores of the HJHS
Временное ограничение: From Baseline up to Week 52

HJHS comprises an assessment of specific features, or items, of the 6 index joints and an assessment of global gait. For each of the 6 joints, the following items are scored: swelling (0 to 3), duration of swelling (0 to 1), muscle atrophy (0 to 2), crepitus on motion (0 to 2), flexion loss (0 to 3), extension loss (0 to 3), joint pain (0 to 2), and strength (0 to 4). The maximum score for an individual index joint is 20. Gait is scored from 0 to 4. The maximum HJHS total score is 124, with a higher score indicating worse joint health.
From Baseline up to Week 52
Change from baseline in total/ domain scores of magnetic resonance imaging (MRI)
Временное ограничение: From Baseline up to Week 52

MRI will be used to detect changes in synovial hypertrophy in index joints of participants.
From Baseline up to Week 52
Change from baseline in patient-reported outcome (PRO)
Временное ограничение: From Baseline up to Week 52

Change from baseline in PRO will be assessed by the EQ-5D 5-level (EQ-5D-5L), Patient-Reported Outcomes Measurement Information System - Short Form (PROMIS-SF) version 2.0 Pain Intensity 3a, PROMIS-SF version 1.1 Pain Interference 6a, and PROMIS-SF version 2.0 Physical Function 6b.
From Baseline up to Week 52
Patient reported treatment preference and satisfaction
Временное ограничение: At Week 52

Patient reported treatment preference and satisfaction will be assessed through surveys and exit interviews.
At Week 52
Change from baseline in annualized bleeding rate (ABR) (spontaneous, traumatic)
Временное ограничение: At Week 52

The ABR will be defined as (number of bleeding episodes occurring during the treatment period)/(duration of the treatment period [days]) × 365.25.
At Week 52
Change from baseline in annualized joint bleeding rate (AjBR) (spontaneous, traumatic)
Временное ограничение: At Week 52

The AjBR will be defined as (number of joint bleeding episodes occurring during the treatment period)/(duration of the treatment period [days]) × 365.25.
At Week 52
Change from baseline in target joint resolution, recurrence, or development
Временное ограничение: At Week 52

Change from baseline in target joint resolution, recurrence, or development will be assessed.
At Week 52
The occurrence of Adverse Events (AEs), all AEs leading to treatment discontinuation, Serious Adverse Events (SAEs), and Adverse Event of Special Interest (AESIs)
Временное ограничение: Up to Week 52

The safety and tolerability of efanesoctocog alfa evaluated in all participants
Up to Week 52

Соавторы и исследователи

Здесь вы найдете людей и организации, участвующие в этом исследовании.

Спонсор

Sanofi

Публикации и полезные ссылки

Лицо, ответственное за внесение сведений об исследовании, добровольно предоставляет эти публикации. Это может быть что угодно, связанное с исследованием.

Полезные ссылки

Даты записи исследования

Эти даты отслеживают ход отправки отчетов об исследованиях и сводных результатов на сайт ClinicalTrials.gov. Записи исследований и сообщаемые результаты проверяются Национальной медицинской библиотекой (NLM), чтобы убедиться, что они соответствуют определенным стандартам контроля качества, прежде чем публиковать их на общедоступном веб-сайте.

Изучение основных дат

Начало исследования (Действительный)

23 сентября 2025 г.

Первичное завершение (Оцененный)

25 октября 2027 г.

Завершение исследования (Оцененный)

25 октября 2027 г.

Даты регистрации исследования

Первый отправленный

16 апреля 2025 г.

Впервые представлено, что соответствует критериям контроля качества

16 апреля 2025 г.

Первый опубликованный (Действительный)

24 апреля 2025 г.

Обновления учебных записей

Последнее опубликованное обновление (Действительный)

9 ноября 2026 г.

Последнее отправленное обновление, отвечающее критериям контроля качества

6 ноября 2026 г.

Последняя проверка

1 ноября 2026 г.

Дополнительная информация

Термины, связанные с этим исследованием

Дополнительные соответствующие термины MeSH

Другие идентификационные номера исследования

  • LPS18168
  • U1111-1311-1701 (Идентификатор реестра: ICTRP)

Планирование данных отдельных участников (IPD)

Планируете делиться данными об отдельных участниках (IPD)?

ДА

Описание плана IPD

Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

Информация о лекарствах и устройствах, исследовательские документы

Изучает лекарственный продукт, регулируемый FDA США.

Да

Изучает продукт устройства, регулируемый Управлением по санитарному надзору за качеством пищевых продуктов и медикаментов США.

Нет

Эта информация была получена непосредственно с веб-сайта clinicaltrials.gov без каких-либо изменений. Если у вас есть запросы на изменение, удаление или обновление сведений об исследовании, обращайтесь по адресу register@clinicaltrials.gov. Как только изменение будет реализовано на clinicaltrials.gov, оно будет автоматически обновлено и на нашем веб-сайте. .

Подписаться