Исследование по оценке безопасности, эффективности, фармакокинетики и фармакодинамики эмицизумаба у участников с легкой или умеренной формой гемофилии А без ингибиторов FVIII (HAVEN 6)
5 августа 2026 г. обновлено: Hoffmann-La Roche
Многоцентровое открытое исследование по оценке безопасности, эффективности, фармакокинетики и фармакодинамики эмицизумаба у пациентов с легкой или умеренной формой гемофилии А без ингибиторов FVIII
Это многоцентровое открытое одногрупповое исследование, предназначенное для оценки безопасности, эффективности, фармакокинетики и фармакодинамики эмицизумаба у участников с легкой или умеренной формой гемофилии А без ингибиторов фактора VIII (FVIII).
Обзор исследования
Статус
Завершенный
Условия
Вмешательство/лечение
Тип исследования
Интервенционный
Регистрация (Действительный)
73
Фаза
- Фаза 3
Контакты и местонахождение
В этом разделе приведены контактные данные лиц, проводящих исследование, и информация о том, где проводится это исследование.
Места учебы
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Brussels, Бельгия, 1200
- Cliniques Universitaires St-Luc
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Leuven, Бельгия, 3000
- UZ Leuven Gasthuisberg
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Bonn, Германия, 53127
- Universitatsklinikum Bonn
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München, Германия, 80336
- Klinikum der Universität München, Campus Innenstadt
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Madrid, Испания, 28046
- Hospital Universitario La Paz
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Seville, Испания, 41013
- Hospital Universitario Virgen del Rocío
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Alberta
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Edmonton, Alberta, Канада, T6G 1Z1
- Kaye Edmonton Clinic
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Newfoundland and Labrador
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St. John's, Newfoundland and Labrador, Канада, A1B 3V6
- Eastern Health - General Hospital
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Amsterdam, Нидерланды, 1105 AZ
- Amsterdam UMC Location AMC
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Warsaw, Польша, 02-776
- Instytut Hematologii i Transfuzjologii
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Cardiff, Соединенное Королевство, CF14 4XW
- Cardiff and Vale NHS Trust
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London, Соединенное Королевство, NW3 2QG
- Royal Free Hospital
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London, Соединенное Королевство, WC1N 3HR
- Great Ormond Street Hospital for Children NHS Foundation Trust
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California
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Los Angeles, California, Соединенные Штаты, 90027
- Childrens Hospital LA
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Georgia
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Atlanta, Georgia, Соединенные Штаты, 30308
- Hemophilia of Georgia Center for Bleeding & Clotting Disorders
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Indiana
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Indianapolis, Indiana, Соединенные Штаты, 46260
- Indiana Hemophilia & Thrombosis center
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Michigan
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Ann Arbor, Michigan, Соединенные Штаты, 48109
- University of Michigan, C.S. Mott Children's Hospital
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Washington
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Seattle, Washington, Соединенные Штаты, 98101
- Washington Institute for Coagulation
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Bron, Франция, 69677
- Hopital Cardio-vasculaire Louis Pradel
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Le Kremlin-Bicêtre, Франция, 94275
- CH de Bicêtre
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Paris, Франция, 75015
- Groupe Hospitalier Necker Enfants Malades
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Johannesburg, Южная Африка, 2193
- Charlotte Maxeke Johannesburg Hospital
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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
- Ребенок
- Взрослый
- Пожилой взрослый
Принимает здоровых добровольцев
Нет
Описание
Критерии включения:
- Диагноз легкой степени (уровень FVIII от >5% до
- Вес ≥3 килограммов (кг)
- Необходимость профилактики на основе оценки следователя
- Отрицательный тест на ингибитор (т.е.
- Отсутствие задокументированного ингибитора (т.е.
- Документирование сведений о профилактическом или эпизодическом лечении FVIII и количестве эпизодов кровотечения по крайней мере за последние 24 недели до включения в исследование.
- Адекватная гематологическая функция печени и почек
- Для женщин детородного возраста: согласие воздерживаться от употребления алкоголя или использовать противозачаточные средства (как определено в протоколе) в течение периода лечения и в течение как минимум 24 недель после последней дозы исследуемого препарата.
Критерий исключения:
- Наследственное или приобретенное нарушение свертываемости крови, отличное от врожденной гемофилии легкой или средней степени тяжести А
- История злоупотребления запрещенными наркотиками или алкоголем в течение 48 недель до скрининга, по мнению исследователя.
- Предшествующее (в течение последних 12 месяцев) или текущее лечение тромбоэмболической болезни или признаков тромбоэмболической болезни
- Другие состояния, которые в настоящее время могут увеличить риск кровотечения или тромбоза
- История клинически значимой гиперчувствительности, связанной с терапией моноклональными антителами или компонентами инъекции эмицизумаба.
- Запланированная операция во время фазы введения нагрузочной дозы эмицизумаба (разрешены операции у участников, получающих эмицизумаб, начиная с 5-й недели)
- Известная ВИЧ-инфекция с подсчетом CD4
- Сопутствующее заболевание, состояние, значительное отклонение от нормы при скрининговом обследовании или лабораторных тестах или лечение, которое может помешать проведению исследования или, по мнению исследователя, представляет дополнительный неприемлемый риск при введении исследуемого препарата участнику.
- Получение любого из следующего: исследуемый препарат для лечения или снижения риска гемофильных кровотечений в течение 5 периодов полувыведения с момента последнего приема препарата, за исключением предшествующей профилактики эмицизумабом; Исследуемый препарат, не связанный с гемофилией, в течение последних 30 дней или 5 периодов полувыведения, в зависимости от того, что короче; или любой другой исследуемый препарат, который в настоящее время вводится или планируется вводить
- Невозможность соблюдения протокола исследования по мнению исследователя
- Беременность или кормление грудью, или намерение забеременеть во время исследования (женщины детородного возраста должны иметь отрицательный результат сывороточного теста на беременность в течение 7 дней до начала приема исследуемого препарата)
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: Н/Д
- Интервенционная модель: Одногрупповое задание
- Маскировка: Нет (открытая этикетка)
Оружие и интервенции
Группа участников / Армия |
Вмешательство/лечение |
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Экспериментальный: Эмицизумаб
Участники с легкой и умеренной формой гемофилии А без ингибиторов фактора VIII (FVIII) будут зачислены для получения режима нагрузочной дозы эмицизумаба, после чего участник выберет один из 3 режимов поддерживающей дозы.
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Четыре нагрузочные дозы эмицизумаба по 3 миллиграмма на килограмм массы тела (мг/кг) будут вводиться подкожно (п/к) один раз в неделю (ч.н.) в течение 4 недель, после чего участник предпочитает один из трех следующих режимов поддерживающей п/к дозы: 1,5 мг. /кг QW, 3 мг/кг один раз каждые 2 недели (Q2W) или 6 мг/кг один раз каждые 4 недели (Q4W).
Другие имена:
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Что измеряет исследование?
Первичные показатели результатов
Мера результата |
Мера Описание |
Временное ограничение |
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Основанная на модели годовая частота кровотечений для пролеченных кровотечений
Временное ограничение: Со дня введения первой дозы эмицизумаба до не менее 52 недель лечения эмицизумабом (медиана [диапазон, мин-макс] периода эффективности: 55,64 [8,6–89,9] недель)
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Количество пролеченных кровотечений за период эффективности оценивали как годовую частоту кровотечений (ABR) с использованием модели отрицательной биномиальной регрессии, которая учитывает разное время наблюдения.
Леченное кровотечение определялось как кровотечение, за которым непосредственно последовало лекарство от гемофилии, которое, как сообщалось, было «лечением кровотечения».
Было реализовано правило 72 часов: два кровотечения одного типа и из одной анатомической локализации считались одним кровотечением, если второе кровотечение произошло в течение 72 часов после последней обработки первого кровотечения.
Кровотечения из-за операции/процедуры были исключены.
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Со дня введения первой дозы эмицизумаба до не менее 52 недель лечения эмицизумабом (медиана [диапазон, мин-макс] периода эффективности: 55,64 [8,6–89,9] недель)
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Mean Calculated ABR for Treated Bleeds
Временное ограничение: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
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The number of treated bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
A treated bleed was defined as a bleed that was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
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From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
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Median Calculated ABR for Treated Bleeds
Временное ограничение: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
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The number of treated bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
A treated bleed was defined as a bleed that was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
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From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
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Вторичные показатели результатов
Мера результата |
Мера Описание |
Временное ограничение |
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Средняя расчетная годовая частота кровотечений для пролеченных спонтанных кровотечений
Временное ограничение: Со дня введения первой дозы эмицизумаба до не менее 52 недель лечения эмицизумабом (медиана [диапазон, мин-макс] периода эффективности: 55,64 [8,6–89,9] недель)
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Количество пролеченных спонтанных кровотечений в течение периода эффективности представлено здесь как рассчитанная годовая частота кровотечений (ККК), которая была пересчитана в годовом исчислении для каждого участника по следующей формуле: ККК = (количество кровотечений/количество дней в течение периода эффективности) х 365,25. .
Пролеченное спонтанное кровотечение определялось как пролеченное кровотечение (кровотечение, непосредственно сопровождаемое приемом лекарства от гемофилии, которое, как сообщается, является «лечением кровотечения») без какого-либо другого известного способствующего фактора, такого как травма или процедура/операция.
Было реализовано правило 72 часов: два кровотечения одного типа и из одной анатомической локализации считались одним кровотечением, если второе кровотечение произошло в течение 72 часов после последней обработки первого кровотечения.
Кровотечения из-за операции/процедуры были исключены.
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Со дня введения первой дозы эмицизумаба до не менее 52 недель лечения эмицизумабом (медиана [диапазон, мин-макс] периода эффективности: 55,64 [8,6–89,9] недель)
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Медианная расчетная годовая частота кровотечений для пролеченных спонтанных кровотечений
Временное ограничение: Со дня введения первой дозы эмицизумаба до не менее 52 недель лечения эмицизумабом (медиана [диапазон, мин-макс] периода эффективности: 55,64 [8,6–89,9] недель)
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Количество пролеченных спонтанных кровотечений в течение периода эффективности представлено здесь как рассчитанная годовая частота кровотечений (ККК), которая была пересчитана в годовом исчислении для каждого участника по следующей формуле: ККК = (количество кровотечений/количество дней в течение периода эффективности) х 365,25. .
Пролеченное спонтанное кровотечение определялось как пролеченное кровотечение (кровотечение, непосредственно сопровождаемое приемом лекарства от гемофилии, которое, как сообщается, является «лечением кровотечения») без какого-либо другого известного способствующего фактора, такого как травма или процедура/операция.
Было реализовано правило 72 часов: два кровотечения одного типа и из одной анатомической локализации считались одним кровотечением, если второе кровотечение произошло в течение 72 часов после последней обработки первого кровотечения.
Кровотечения из-за операции/процедуры были исключены.
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Со дня введения первой дозы эмицизумаба до не менее 52 недель лечения эмицизумабом (медиана [диапазон, мин-макс] периода эффективности: 55,64 [8,6–89,9] недель)
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Процент участников, которые предпочитают подкожное лечение эмицизумабом, их предыдущее лечение гемофилии IV или не имеют предпочтений, по оценке с помощью опроса о предпочтениях эмицизумаба на неделе 17
Временное ограничение: Неделя 17
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Опрос о предпочтениях в отношении эмицизумаба представляет собой целевую анкету, разработанную спонсором для регистрации предпочтений участников в отношении лечения подкожным (п/к) эмицизумабом, внутривенным (в/в) введением фактора VIII (FVIII) или отсутствием предпочтений.
95% доверительные интервалы были рассчитаны с использованием метода Пирсона-Клоппера.
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Неделя 17
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Процент лиц, осуществляющих уход, которые предпочитают подкожное лечение эмицизумабом, предыдущее лечение их ребенка от внутривенной гемофилии или не имеют предпочтений, по оценке с помощью опроса о предпочтениях эмицизумаба на 17-й неделе
Временное ограничение: Неделя 17
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Опрос о предпочтениях в отношении эмицизумаба представляет собой целевую анкету, разработанную спонсором для регистрации предпочтений лиц, осуществляющих уход, в отношении лечения их ребенка подкожным (п/к) эмицизумабом, внутривенным (в/в) введением фактора VIII (FVIII) или отсутствием предпочтений.
95% доверительные интервалы были рассчитаны с использованием метода Пирсона-Клоппера.
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Неделя 17
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Изменение среднесуточной продолжительности пиковой активности по сравнению с исходным уровнем с течением времени
Временное ограничение: Исходный уровень (недели 1–2) и недели 13 (период 12–13 недель), 25 (период недель 24–25), 37 (период недель 36–37) и 49 (период недель 48–49)
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Для оценки физической активности участников в возрасте ≥5 лет проинструктировали постоянно (24 часа в день) каждый день носить на запястье исследуемое акселерометрическое устройство в течение установленных 2-недельных периодов во время исследования.
Участник считался соответствующим требованиям оценки физической активности, если он носил исследуемое устройство постоянно (≥8 часов в день) каждый день в течение как минимум 8 дней каждого из назначенных двухнедельных периодов в ходе исследования.
Если этот критерий соответствия не был достигнут в определенный момент времени, участник не включался в анализ назначенных 2-недельных периодов, когда соответствие не было достигнуто.
Ежедневные измерения были усреднены за 14-дневный период времени.
Подсчет активности был мерой ускорения, измеренного устройством.
Продолжительность дневной пиковой активности определялась как сумма активности от умеренной до высокой в день (в минутах).
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Исходный уровень (недели 1–2) и недели 13 (период 12–13 недель), 25 (период недель 24–25), 37 (период недель 36–37) и 49 (период недель 48–49)
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Изменение среднесуточного количества шагов по сравнению с исходным уровнем с течением времени
Временное ограничение: Исходный уровень (недели 1–2) и недели 13 (недели 12–13), 25 (недели 24–25), 37 (недели 36–37) и 49 (недели 48–49)
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Для оценки физической активности участников в возрасте ≥5 лет проинструктировали постоянно (24 часа в день) каждый день носить на запястье исследуемое акселерометрическое устройство в течение установленных 2-недельных периодов во время исследования.
Участник считался соответствующим требованиям оценки физической активности, если он носил исследуемое устройство постоянно (≥8 часов в день) каждый день в течение как минимум 8 дней каждого из назначенных двухнедельных периодов в ходе исследования.
Если этот критерий соответствия не был достигнут в определенный момент времени, участник не включался в анализ назначенных 2-недельных периодов, когда соответствие не было достигнуто.
Ежедневные измерения были усреднены за 14-дневный период времени.
Подсчет активности был мерой ускорения, измеренного устройством.
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Исходный уровень (недели 1–2) и недели 13 (недели 12–13), 25 (недели 24–25), 37 (недели 36–37) и 49 (недели 48–49)
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Model-Based ABR for All Bleeds
Временное ограничение: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
|
The number of all bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times.
In this outcome measure, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded.
As "all bleeds" comprises both treated and non-treated bleeds, the 72-hour rule was implemented separately for treated and non-treated bleeds.
For treated bleeds, the 72-hour rule meant that two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
For non-treated bleeds, the 72-hour rule was implemented by calculating a treatment-free period of 72 hours from the bleed itself.
|
From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
|
|
Mean Calculated ABR for All Bleeds
Временное ограничение: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
|
The number of all bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
In this outcome measure, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded.
As "all bleeds" comprises both treated and non-treated bleeds, the 72-hour rule was implemented separately for treated and non-treated bleeds.
For treated bleeds, the 72-hour rule meant that two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
For non-treated bleeds, the 72-hour rule was implemented by calculating a treatment-free period of 72 hours from the bleed itself.
|
From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
|
|
Median Calculated ABR for All Bleeds
Временное ограничение: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
|
The number of all bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
In this outcome measure, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded.
As "all bleeds" comprises both treated and non-treated bleeds, the 72-hour rule was implemented separately for treated and non-treated bleeds.
For treated bleeds, the 72-hour rule meant that two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
For non-treated bleeds, the 72-hour rule was implemented by calculating a treatment-free period of 72 hours from the bleed itself.
|
From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
|
|
Model-Based ABR for Treated Joint Bleeds
Временное ограничение: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
|
The number of treated joint bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times.
A treated joint bleed was defined as a bleed with type reported as "joint" based on at least one of the following symptoms: increasing swelling or warmth of the skin over the joint and/or increasing pain, decreased range of motion, or difficulty using the joint compared with baseline, and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
|
|
Mean Calculated ABR for Treated Joint Bleeds
Временное ограничение: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
|
The number of treated joint bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
A treated joint bleed was defined as a bleed with type reported as "joint" based on at least one of the following symptoms: increasing swelling or warmth of the skin over the joint and/or increasing pain, decreased range of motion, or difficulty using the joint compared with baseline, and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
|
|
Median Calculated ABR for Treated Joint Bleeds
Временное ограничение: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
|
The number of treated joint bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
A treated joint bleed was defined as a bleed with type reported as "joint" based on at least one of the following symptoms: increasing swelling or warmth of the skin over the joint and/or increasing pain, decreased range of motion, or difficulty using the joint compared with baseline, and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
|
|
Model-Based ABR for Treated Target Joint Bleeds
Временное ограничение: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
|
The number of treated target joint bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times.
A treated target joint bleed was defined as a joint bleed in a target joint, which is a joint location where at least 3 bleeds have occurred over the last 24 weeks prior to study entry or an unresolved target joint (target joint that does not fulfil ≤2 bleeds into this joint within a consecutive 12-month period), and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
|
|
Mean Calculated ABR for Treated Target Joint Bleeds
Временное ограничение: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
|
The number of treated target joint bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
A treated target joint bleed was defined as a joint bleed in a target joint, which is a joint location where at least 3 bleeds have occurred over the last 24 weeks prior to study entry or an unresolved target joint (target joint that does not fulfil ≤2 bleeds into this joint within a consecutive 12-month period), and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
|
|
Median Calculated ABR for Treated Target Joint Bleeds
Временное ограничение: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
|
The number of treated target joint bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
A treated target joint bleed was defined as a joint bleed in a target joint, which is a joint location where at least 3 bleeds have occurred over the last 24 weeks prior to study entry or an unresolved target joint (target joint that does not fulfil ≤2 bleeds into this joint within a consecutive 12-month period), and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
|
|
Model-Based ABR for Treated Spontaneous Bleeds
Временное ограничение: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
|
The number of treated spontaneous bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times.
A treated spontaneous bleed was defined as a treated bleed (bleed directly followed by a hemophilia medication reported to be a "treatment for bleed") with no other known contributing factor such as trauma or procedure/surgery.
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
|
|
Long-term Efficacy of Emicizumab: Model-Based ABR for Treated Bleeds
Временное ограничение: Median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of treated bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times.
A treated bleed was defined as a bleed that was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
Median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
Long-term Efficacy of Emicizumab: Mean Calculated ABR for Treated Bleeds
Временное ограничение: Median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of treated bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
A treated bleed was defined as a bleed that was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
Median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
Long-term Efficacy of Emicizumab: Median Calculated ABR for Treated Bleeds
Временное ограничение: Median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of treated bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
A treated bleed was defined as a bleed that was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
Median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
Long-term Efficacy of Emicizumab: Model-Based ABR for All Bleeds
Временное ограничение: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of all bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times.
In this outcome measure, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded.
As "all bleeds" comprises both treated and non-treated bleeds, the 72-hour rule was implemented separately for treated and non-treated bleeds.
For treated bleeds, the 72-hour rule meant that two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
For non-treated bleeds, the 72-hour rule was implemented by calculating a treatment-free period of 72 hours from the bleed itself.
|
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
Long-term Efficacy of Emicizumab: Mean Calculated ABR for All Bleeds
Временное ограничение: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of all bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
In this outcome measure, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded.
As "all bleeds" comprises both treated and non-treated bleeds, the 72-hour rule was implemented separately for treated and non-treated bleeds.
For treated bleeds, the 72-hour rule meant that two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
For non-treated bleeds, the 72-hour rule was implemented by calculating a treatment-free period of 72 hours from the bleed itself.
|
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
Long-term Efficacy: Median Calculated ABR for All Bleeds
Временное ограничение: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of all bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
In this outcome measure, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded.
As "all bleeds" comprises both treated and non-treated bleeds, the 72-hour rule was implemented separately for treated and non-treated bleeds.
For treated bleeds, the 72-hour rule meant that two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
For non-treated bleeds, the 72-hour rule was implemented by calculating a treatment-free period of 72 hours from the bleed itself.
|
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
Long-term Efficacy of Emicizumab: Model-Based ABR for Treated Joint Bleeds
Временное ограничение: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of treated joint bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times.
A treated joint bleed was defined as a bleed with type reported as "joint" based on at least one of the following symptoms: increasing swelling or warmth of the skin over the joint and/or increasing pain, decreased range of motion, or difficulty using the joint compared with baseline, and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
Long-term Efficacy of Emicizumab: Mean Calculated ABR for Treated Joint Bleeds
Временное ограничение: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of treated joint bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
A treated joint bleed was defined as a bleed with type reported as "joint" based on at least one of the following symptoms: increasing swelling or warmth of the skin over the joint and/or increasing pain, decreased range of motion, or difficulty using the joint compared with baseline, and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
Long-term Efficacy of Emicizumab: Median Calculated ABR for Treated Joint Bleeds
Временное ограничение: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of treated joint bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
A treated joint bleed was defined as a bleed with type reported as "joint" based on at least one of the following symptoms: increasing swelling or warmth of the skin over the joint and/or increasing pain, decreased range of motion, or difficulty using the joint compared with baseline, and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
Long-term Efficacy of Emicizumab: Model-Based ABR for Treated Target Joint Bleeds
Временное ограничение: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of treated target joint bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times.
A treated target joint bleed was defined as a joint bleed in a target joint, which is a joint location where at least 3 bleeds have occurred over the last 24 weeks prior to study entry or an unresolved target joint (target joint that does not fulfil ≤2 bleeds into this joint within a consecutive 12-month period), and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
Long-term Efficacy of Emicizumab: Mean Calculated ABR for Treated Target Joint Bleeds
Временное ограничение: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of treated target joint bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
A treated target joint bleed was defined as a joint bleed in a target joint, which is a joint location where at least 3 bleeds have occurred over the last 24 weeks prior to study entry or an unresolved target joint (target joint that does not fulfil ≤2 bleeds into this joint within a consecutive 12-month period), and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
Long-term Efficacy of Emicizumab: Median Calculated ABR for Treated Target Joint Bleeds
Временное ограничение: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of treated target joint bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
A treated target joint bleed was defined as a joint bleed in a target joint, which is a joint location where at least 3 bleeds have occurred over the last 24 weeks prior to study entry or an unresolved target joint (target joint that does not fulfil ≤2 bleeds into this joint within a consecutive 12-month period), and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
Long-term Efficacy of Emicizumab: Model-Based ABR for Treated Spontaneous Bleeds
Временное ограничение: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of treated spontaneous bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times.
A treated spontaneous bleed was defined as a treated bleed (bleed directly followed by a hemophilia medication reported to be a "treatment for bleed") with no other known contributing factor such as trauma or procedure/surgery.
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
Long-term Efficacy of Emicizumab: Mean Calculated ABR for Treated Spontaneous Bleeds
Временное ограничение: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of treated spontaneous bleeds over the efficacy period is presented here as a calculated annualized bleed rate (ABR) that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
A treated spontaneous bleed was defined as a treated bleed (bleed directly followed by a hemophilia medication reported to be a "treatment for bleed") with no other known contributing factor such as trauma or procedure/surgery.
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
Long-term Efficacy of Emicizumab: Median Calculated ABR for Treated Spontaneous Bleeds
Временное ограничение: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of treated spontaneous bleeds over the efficacy period is presented here as a calculated annualized bleed rate (ABR) that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
A treated spontaneous bleed was defined as a treated bleed (bleed directly followed by a hemophilia medication reported to be a "treatment for bleed") with no other known contributing factor such as trauma or procedure/surgery.
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
CATCH Questionnaire for Adult Participants: Change From Baseline in the Daily Activity Risk Perception (RP) and Impact Domain Scores Over Time
Временное ограничение: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and Study completion (SC)/Early discontinuation (ED) (up to approximately 274 weeks)
|
Comprehensive Assessment Tool of Challenges in Hemophilia (CATCH) was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥ 18 years).
The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain.
Of this, the daily activity RP and impact domain has 48 items with each item scored on 4 or 5 points ordinal scales.
Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept and applying a linear transformation.
The transformed scores range from 0-100 scale for both the RP and Impact.
Higher scores indicating higher perceived risk of having a bleed while doing daily activities and higher impact of hemophilia on daily activities.
|
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and Study completion (SC)/Early discontinuation (ED) (up to approximately 274 weeks)
|
|
CATCH Questionnaire for Adult Participants: Change From Baseline in the Social Activity RP and Impact Domain Scores Over Time
Временное ограничение: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
|
CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥ 18 years).
The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain.
Of this, the social activity RP and impact domain has 22 items with each item scored on 4 or 5 points ordinal scales.
Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept and applying a linear transformation.
The transformed scores range from 0-100 scale for both the RP and Impact.
Higher scores indicating higher perceived risk of having a bleed while doing social activities and higher impact of hemophilia on social activities.
|
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
|
|
CATCH Questionnaire for Adult Participants: Change From Baseline in the Recreational Activity RP and Impact Domain Scores Over Time
Временное ограничение: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
|
CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥ 18 years).
The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain.
Of this, the recreational activity RP and impact domain has 32 items and a larger bank of additional items with each item scored on 4 or 5 points ordinal scales.
Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept and applying a linear transformation.
The transformed scores range from 0-100 scale for both the RP and Impact.
Higher scores indicating higher perceived risk of having a bleed while doing recreational activities and higher impact of hemophilia on recreational activities.
|
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
|
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CATCH Questionnaire for Adult Participants: Change From Baseline in the Work Impact (WI) and College Impact (CI) Domains Score Over Time
Временное ограничение: WI: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks); CI: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, and SC/ED (up to approximately 274 weeks)
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CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥ 18 years).
The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain.
Of this, work and college impact has 10 items and the items were scored on 4 or 5 points ordinal scales.
Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept & applying a linear transformation.
The transformed scores range from 0-100 scale.
Higher scores indicated impact of hemophilia on work and college activities.
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WI: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks); CI: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, and SC/ED (up to approximately 274 weeks)
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CATCH Questionnaire for Adult Participants: Change From Baseline in the Preoccupation Domain Score Over Time
Временное ограничение: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
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CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥18 years).
The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain.
Of this, preoccupation has 10 items which were scored on 4 or 5 points ordinal scales.
Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept & applying a linear transformation.
The transformed scores range from 0-100 scale.
Higher scores indicated greater preoccupation related to hemophilia.
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Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
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CATCH Questionnaire for Adult Participants: Change From Baseline in the Treatment Burden Domain Score Over Time
Временное ограничение: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
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CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥18 years).
The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain.
Of this, treatment burden has 8 items which were scored on 4 or 5 points ordinal scales.
Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept & applying a linear transformation.
The transformed scores range from 0-100 scale.
Higher scores indicated greater perceived burden of the hemophilia treatment.
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Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
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CATCH Questionnaire for Adult Participants: Number of Participants by Responses to Their Level of Pain Associated With a Bleed Over Time
Временное ограничение: Baseline, Weeks 25, 49, 61, 85, 121, 133, 157 and 169 and SC/ED (up to approximately 274 weeks)
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CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥18 years).
The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain.
Of this, the pain domain consisting of five items, assessed using an 11-point numeric rating scale where 0 = no pain and 10 = pain as bad as you can imagine.
The data reported here are from participants who answered the following question: "Did you get a bleed in the past 7 days?"
If the response was yes, participants were asked to rate their pain by selecting the number that best described the pain associated with the bleed.
Data is reported only for categories/timepoints with non-zero values.
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Baseline, Weeks 25, 49, 61, 85, 121, 133, 157 and 169 and SC/ED (up to approximately 274 weeks)
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CATCH Questionnaire for Adult Participants: Number of Participants by Responses to Their Level of Pain in Target Joints Over Time
Временное ограничение: Baseline, Weeks 1, 13, 25, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
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CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥18 years).
The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain.
Of this, the pain domain consists of five items, assessed using an 11-point numeric rating scale where 0 = no pain and 10 = pain as bad as you can imagine.
The data reported here are from participants who answered the following question "Do you have target joints?"
(If yes, please rate your pain by picking the number that best describes your pain in your target joint(s) over the past 7 days).
Data is reported only for categories/timepoints with non-zero values.
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Baseline, Weeks 1, 13, 25, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
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CATCH Questionnaire for Adult Participants: Number of Participants by Responses to Their Level of Pain at Its Worst Over Time
Временное ограничение: Baseline, Weeks 1, 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
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CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥18 years).
The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain.
Of this, pain domain consists of five items, assessed using an 11-point numeric rating scale where 0 = no pain and 10 = pain as bad as you can imagine.
The data reported here are from participants who answered the following question "Please rate your pain by picking the number that best describes your pain at its WORST over the past 7 days".
Data is reported only for categories/timepoints with non-zero values.
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Baseline, Weeks 1, 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
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CATCH Questionnaire for Adult Participants: Number of Participants by Responses to Their Level of Pain at Its Least Over Time
Временное ограничение: Baseline, Weeks 1, 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
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CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥18 years).
The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain.
Of this, pain domain consists of five items, assessed using an 11-point numeric rating scale where 0 = no pain and 10 = pain as bad as you can imagine.
The data reported here are from participants who answered the following question "Please rate your pain by picking the number that best describes your pain at its LEAST over the past 7 days."
Data is reported only for categories/timepoints with non-zero values.
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Baseline, Weeks 1, 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
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CATCH Questionnaire for Adult Participants: Number of Participants by Responses to Their Level of Pain at Its Least on the Average Over Time
Временное ограничение: Baseline, Weeks 1, 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
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CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥18 years).
The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain.
Of this, the pain domain consists of five items, assessed using an 11-point numeric rating scale where 0 = no pain and 10 = pain as bad as you can imagine.
The data reported here are from participants who answered the following question "Please rate your pain by picking the number that best describes your pain at its average over the past 7 days".
Data is reported only for categories/timepoints with non-zero values.
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Baseline, Weeks 1, 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
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CATCH Questionnaire for Pediatric Participants: Change From Baseline in the Daily Activity RP and Impact Domain Score Over Time
Временное ограничение: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
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CATCH was used to measure the effect of hemophilia and its treatment on pediatric participants (aged 8-17 years).
The pediatric version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, school impact, preoccupation, treatment burden, and pain).
Of this, daily activity RP and impact has 38 items with each item scored on 3 or 4 points ordinal scale.
Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept and applying a linear transformation.
The transformed scores range from 0-100 scale.
Higher scores indicating higher perceived risk of having a bleed while doing daily activities and higher impact of hemophilia on daily activities.
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Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
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CATCH Questionnaire for Pediatric Participants: Change From Baseline in the Social Activity Risk Perception and Impact Domain Score Over Time
Временное ограничение: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
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CATCH was used to measure the effect of hemophilia and its treatment on pediatric participants (aged 8-17 years).
The pediatric version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, school impact, preoccupation, treatment burden, and pain).
Of this, social activity RP and impact has 16 items which were scored on 3 or 4 points ordinal scale.
Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept & applying a linear transformation.
The transformed scores range from 0-100 scale.
Higher scores indicated higher perceived risk of having a bleed while doing social activities and higher impact of hemophilia on social activities.
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Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
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CATCH Questionnaire for Pediatric Participants: Change From Baseline in the Recreational Activity RP and Impact Domain Score Over Time
Временное ограничение: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
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CATCH was used to measure the effect of hemophilia and its treatment on pediatric participants (aged 8-17 years).
The pediatric version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, school impact, preoccupation, treatment burden, and pain.
Of this, recreational activity RP & impact has 34 items + larger bank of additional items.
The items were scored on 3 or 4 points ordinal scales.
Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept & applying a linear transformation.
The transformed scores range from 0-100 scale.
Higher scores indicated higher perceived risk of having a bleed while doing recreational activities and higher impact of hemophilia on recreational activities.
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Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
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CATCH Questionnaire for Pediatric Participants: Change From Baseline in the School Impact Domain Score Over Time
Временное ограничение: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
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CATCH was used to measure the effect of hemophilia and its treatment on pediatric participants (aged 8-17 years).
The pediatric version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, school impact, preoccupation, treatment burden, and pain.
Of this, school impact has 11 items and the items were scored on 3 or 4 points ordinal scales.
Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept & applying a linear transformation.
The transformed scores range from 0-100 scale.
Higher scores indicated higher impact of hemophilia on school activities.
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Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
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CATCH Questionnaire for Pediatric Participants: Change From Baseline in the Preoccupation Domain Score Over Time
Временное ограничение: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
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CATCH was used to measure the effect of hemophilia and its treatment on pediatric participants (aged 8-17 years).
The pediatric version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, school impact, preoccupation, treatment burden, and pain.
Of this, preoccupation has 3 items which were scored on 3 or 4 points ordinal scales.
Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept & applying a linear transformation.
The transformed scores range from 0-100 scale.
Higher scores indicated greater preoccupation related to hemophilia.
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Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
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CATCH Questionnaire for Pediatric Participants: Change From Baseline in the Treatment Burden Domain Score Over Time
Временное ограничение: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
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CATCH was used to measure the effect of hemophilia and its treatment on pediatric participants (aged 8-17 years).
The pediatric version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, school impact, preoccupation, treatment burden, and pain.
Of this, treatment burden has 7 items which were scored on 3 or 4 points ordinal scales.
Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept & applying a linear transformation.
The transformed scores range from 0-100 scale.
Higher scores indicated lower perceived burden of the hemophilia treatment.
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Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
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CATCH Questionnaire for Pediatric Participants: Number of Participants by Responses to Their Level of Hurt Associated With a Bleed Over Time
Временное ограничение: Baseline, Weeks 13, 25, 37, 49, 61, 85, 97, 109, 121, 133, 145 and 157
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CATCH was used to measure the effect of hemophilia and its treatment on pediatric participants (aged 8-17 years).
The pediatric version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, school impact, preoccupation, treatment burden, and pain).
Of this, pain domain (Hurt) consists of two items, assessed using an 11-point numeric rating scale where 0 = no hurt and 10 = a lot of hurt.
The data reported here are from participants who answered the following question "Did you get a bleed in the past 7 days?
( If yes, how much did the bleed hurt?)."
Data is reported only for categories/timepoints with non-zero values.
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Baseline, Weeks 13, 25, 37, 49, 61, 85, 97, 109, 121, 133, 145 and 157
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CATCH Questionnaire for Pediatric Participants: Number of Participants by Responses to Their Level of Hurt at Its Worst Over Time
Временное ограничение: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145,157, 169 and SC/ED (up to approximately 274 weeks)
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CATCH was used to measure the effect of hemophilia and its treatment on pediatric participants (aged 8-17 years).
The pediatric version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, school impact, preoccupation, treatment burden, and pain.
Of this, pain domain (Hurt) consists of two items, assessed using an 11-point numeric rating scale where 0 = no hurt and 10 = a lot of hurt.
The data reported here are from participants who answered the following question "What is the WORST that your body has hurt over the past 7 days".
Data is reported only for categories/timepoints with non-zero values.
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Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145,157, 169 and SC/ED (up to approximately 274 weeks)
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CATCH Questionnaire for Caregivers: Change From Baseline in the Preoccupation Domain Score Over Time
Временное ограничение: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
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CATCH was used to measure the effect of hemophilia and its treatment on caregivers of pediatric participants (aged <8 years).
The caregiver version has 2 domains (preoccupation and treatment burden).
Of this, preoccupation has 13 items with each of the items were scored on 4 or 5 ordinal scales.
Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept and applying a linear transformation.
The transformed scores range from 0-100 scale.
Higher scores indicated greater preoccupation related to hemophilia.
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Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
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CATCH Questionnaire for Caregivers: Change From Baseline in the Treatment Burden Domain Score Over Time
Временное ограничение: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
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CATCH was used to measure the effect of hemophilia and its treatment on caregivers of pediatric participants (aged <8 years).
The caregiver version has 2 domains (preoccupation and treatment burden).
Of this, treatment burden has 8 items which were scored on 4 or 5 ordinal scales.
Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept and applying a linear transformation.
The transformed scores range from 0-100 scale.
Higher scores indicated greater perceived burden of the hemophilia treatment.
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Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
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Change From Baseline in Hemophilia Joint Health Scores Over Time
Временное ограничение: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
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The HJHS measures joint health of the joints (knees, ankles, and elbows) most commonly affected by bleeding in hemophilia.
The HJHS 2.1 provides joint specific total scores which are added to obtain the sum of joints totals and also a global gait score.
These two scores are then added to obtain HJHS total score.
The minimum score per joint is 0, the maximum score is 20.
In addition, Gait is scored on a scale from 0 to 4 based on the number of skills that are not within the normal limits.
The total score is the sum of scores across all six joints plus the gait score (range from 0 to 124, with 0 being normal and 124 being the most severe disease).
A higher score indicates worse joint health.
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Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
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Menstrual Bleed Questionnaire (MBQ) for Female Participants of Childbearing Potential: Change From Baseline in the MBQ Total Score Over Time
Временное ограничение: Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 85, 89, 93, 97, 101, 104, 109, 113, 117, 121, 125, 129, 133, 137, 141 and145
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The MBQ is a validated measure for menorrhagia and used to assess impact on the menstrual bleed-related heaviness, pain, irregularity, and quality of life (QoL).
The MBQ is a 20-item measure covering four aspects regarding heavy menstrual bleeding: heaviness (8 items), QoL (8 items), irregularity (3 item), and pain (1 item).
The MBQ score ranges from 0 to 75.
Higher scores indicate a worse quality of life and more severe symptoms.
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Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 85, 89, 93, 97, 101, 104, 109, 113, 117, 121, 125, 129, 133, 137, 141 and145
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MBQ for Female Participants of Childbearing Potential: Change From Baseline in the Heaviness Subscale Score Over Time
Временное ограничение: Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 89, 93, 97, 101, 104, 109, 113, 121, 125, 129, 133 and 137 (up to approximately 274 weeks)
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The MBQ is a validated measure for menorrhagia and used to assess impact on the menstrual bleed-related heaviness, pain, irregularity, and quality of life (QoL).
The MBQ is a 20-item measure covering four aspects regarding heavy menstrual bleeding: heaviness (8 items), QoL (8 items), irregularity (3 item), and pain (1 item).
The total score for heaviness subscale ranges from 0 to 29.
Higher score indicates more heavy bleeding.
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Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 89, 93, 97, 101, 104, 109, 113, 121, 125, 129, 133 and 137 (up to approximately 274 weeks)
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MBQ for Female Participants of Childbearing Potential: Change From Baseline in the QoL Subscale Score Over Time
Временное ограничение: Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 85, 89, 93, 97, 101, 104, 109, 113, 117, 121, 125, 129, 133, 137, 141 and 145
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The MBQ is a validated measure for menorrhagia and used to assess impact on the menstrual bleed-related heaviness, pain, irregularity, and quality of life (QoL).
The MBQ is a 20-item measure covering four aspects regarding heavy menstrual bleeding: heaviness (8 items), QoL (8 items), irregularity (3 item), and pain (1 item).
The total score for QoL subscale ranges from 0 to 37. Higher scores indicate a worse QoL.
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Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 85, 89, 93, 97, 101, 104, 109, 113, 117, 121, 125, 129, 133, 137, 141 and 145
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MBQ for Female Participants of Childbearing Potential: Change From Baseline in the Irregularity Subscale Score Over Time
Временное ограничение: Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 85, 89, 93, 97, 101, 104, 109, 113, 117, 121, 125, 129, 133, 137, 141 and 145
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The MBQ is a validated measure for menorrhagia and used to assess impact on the menstrual bleed-related heaviness, pain, irregularity, and quality of life (QoL).
The MBQ is a 20-item measure covering four aspects regarding heavy menstrual bleeding: heaviness (8 items), QoL (8 items), irregularity (3 item), and pain (1 item).
The total score for irregularity subscale ranges from 0 to 6. Higher score indicates more irregularity.
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Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 85, 89, 93, 97, 101, 104, 109, 113, 117, 121, 125, 129, 133, 137, 141 and 145
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MBQ for Female Participants of Childbearing Potential: Change From Baseline in the Pain Subscale Score Over Time
Временное ограничение: Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 89, 93, 97, 101, 104, 109, 113, 121, 125, 129, 133 and 137
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The MBQ is a validated measure for menorrhagia and used to assess impact on the menstrual bleed-related heaviness, pain, irregularity, and quality of life (QoL).
The MBQ is a 20-item measure covering four aspects regarding heavy menstrual bleeding: heaviness (8 items), QoL (8 items), irregularity (3 item), and pain (1 item).
The total score for pain subscale ranges from 0 to 3. Higher score indicates severe pain.
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Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 89, 93, 97, 101, 104, 109, 113, 121, 125, 129, 133 and 137
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Menstruation Diary With the Pictorial Blood Assessment Chart (PBAC) for Female Participants of Childbearing Potential: PBAC Scores Over Time
Временное ограничение: Baseline and monthly (on days of menstruation) until Study Completion (up to approximately 247 weeks)
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The Menstruation Diary used a PBAC, which has shown good correlation with menstrual blood loss.
The PBAC is a chart that records the occurrence and size of clots, and the number of episodes of heavy bleeding (flooding), as well as depicts the amount of blood loss during a cycle.The PBAC records pad and tampon use (as either light [1 point], medium [5 points], or heavy [10 points] flow), clots (small [1 point] or large [5 points]), and flooding episodes (1 point each) which can be recorded as many times as necessary any day of the month.
The PBAC is scored from 0 (no bleeding) onwards, with a score of >100 defining abnormal coagulation and heavy menstrual bleeding (corresponds to >80ml of blood loss per menstrual cycle).
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Baseline and monthly (on days of menstruation) until Study Completion (up to approximately 247 weeks)
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Number of Participants With at Least One Adverse Event by Severity, According to the World Health Organization (WHO) Toxicity Grading Scale
Временное ограничение: From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
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An AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment.
An AE can therefore be any unfavorable and unintended sign (including abnormal laboratory values or abnormal clinical test results), symptoms, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product.
As per WHO toxicity grading, Grade 1= mild, 2= moderate, 3=severe, 4=life threatening.
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From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
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Number of Participants With Adverse Events Leading to Study Drug Discontinuation
Временное ограничение: From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
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An AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment.
An AE can therefore be any unfavorable and unintended sign (including abnormal laboratory values or abnormal clinical test results), symptoms, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product.
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From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
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Number of Participants With at Least One Thromboembolic Event
Временное ограничение: From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
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An AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment.
An AE can therefore be any unfavorable and unintended sign (including abnormal laboratory values or abnormal clinical test results), symptoms, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product.
The following signs and symptoms were recognized as potential thromboembolism (i.e., dyspnea, chest pain, leg pain, or swelling; or if in the head, headache, numbness in the face, eye pain or swelling, or vision impairment).
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From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
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Number of Participants With at Least One Event of Thrombotic Microangiopathy
Временное ограничение: From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
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An AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment.
An AE can therefore be any unfavorable and unintended sign (including abnormal laboratory values or abnormal clinical test results), symptoms, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product.
TMA is used to describe a group of disorders with clinical features of microangiopathic hemolytic anemia, thrombocytopenia, and organ damage that can include the kidneys, gastrointestinal system, central nervous system, etc.
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From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
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Number of Participants With at Least One Injection-Site Reaction by Severity, According to the WHO Toxicity Grading Scale
Временное ограничение: From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
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An AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment.
An AE can therefore be any unfavorable and unintended sign (including abnormal laboratory values or abnormal clinical test results), symptoms, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product.
Injection-site reactions are defined as AEs related that occur within 24 hours after study drug administration and are judged to be related to the study drug injection.
Local injection-site reactions included erythema, hematoma, rash, discomfort, pain, and pruritus and were mostly of mild and moderate intensity.
As per WHO toxicity grading, Grade 1= mild, 2= moderate, 3=severe, 4=life threatening.
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From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
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Number of Participants With at Least One Severe Hypersensitivity, Anaphylaxis, and Anaphylactoid Event
Временное ограничение: From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
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An AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment.
An AE can therefore be any unfavorable and unintended sign (including abnormal laboratory values or abnormal clinical test results), symptoms, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product.
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From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
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Number of Participants With at Least One Shifts in Clinical Laboratory Parameters From Baseline WHO Toxicity Scale Grade 0-2 to Post-baseline Grade 3 or 4
Временное ограничение: Up to approximately 274 weeks
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An abnormal laboratory value was defined as a laboratory test result outside of the normal range for hematology or serum chemistry parameters.
The WHO toxicity grading scale, which ranges from Grades 1 to 4 (least severe to most severe, respectively; Grade 0 is within normal range), was used for assessing the severity of laboratory abnormalities and adverse events (WHO 2003).
Not every laboratory abnormality qualified as an adverse event; an abnormality was reported as an adverse event if it met any of the following criteria: was accompanied by clinical symptoms; resulted in a change in study treatment (e.g., dosage modification, treatment interruption or discontinuation); resulted in a medical intervention or a change in concomitant therapy; or was clinically significant in the investigator's judgment.
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Up to approximately 274 weeks
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Number of Participants With at Least One Adverse Events of Changes From Baseline in Vital Signs
Временное ограничение: Up to approximately 274 weeks
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The number of participants with adverse events of changes from baseline in vital signs is reported here.
Vital signs measurements consisted of heart and respiratory rate, temperature, and systolic and diastolic blood pressures, with an abnormal vital sign value being outside of the normal range.
An abnormal vital sign result was reported as an adverse event if it met any of the following criteria: was accompanied by clinical symptoms; resulted in a change in study treatment (e.g., dosage modification, treatment interruption or discontinuation); resulted in a medical intervention or a change in concomitant therapy; or was clinically significant in the investigator's judgment.
All of the adverse events reported here were assessed independently by the investigator as not related to treatment with emicizumab.
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Up to approximately 274 weeks
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Change From Baseline in Electrocardiogram (ECG) Parameters Over Time: QT, QTcB, QTcF, RR, PR, and QRS Intervals
Временное ограничение: Baseline, Weeks 5, 25, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181, SC/ED (up to approximately 274 weeks)
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Baseline, Weeks 5, 25, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181, SC/ED (up to approximately 274 weeks)
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Change From Baseline in Heart Rate Over Time, as Measured by ECG
Временное ограничение: Baseline, Weeks 5, 25, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181, SC/ED (up to approximately 274 weeks)
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Baseline, Weeks 5, 25, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181, SC/ED (up to approximately 274 weeks)
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Plasma Trough Concentration (Ctrough) of Emicizumab Over Time
Временное ограничение: Pre-dose at Weeks 1, 2, 3, 4, 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169 and 181 (up to approximately 274 weeks)
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Pre-dose at Weeks 1, 2, 3, 4, 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169 and 181 (up to approximately 274 weeks)
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Number of Participants With Anti-Drug Antibodies Against Emicizumab at Baseline and Post-Baseline
Временное ограничение: Baseline, Weeks 5, 13, 25, 33, 41, 49, 61, 73, 85, 97, 109, 121, 133, 145, 169, 181 and SC/ED (up to approximately 274 weeks)
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Participants were considered to be ADA positive if they were ADA negative at baseline but develop an ADA response following study drug administration (treatment-induced ADA response), or if they were ADA positive at baseline and the titer of one or more post-baseline samples is at least 4-fold greater than the titer of the baseline sample (treatment-enhanced ADA response).
The sum of all visits has been reported here.
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Baseline, Weeks 5, 13, 25, 33, 41, 49, 61, 73, 85, 97, 109, 121, 133, 145, 169, 181 and SC/ED (up to approximately 274 weeks)
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Number of Participants Who Develop Anti-FVIII Inhibitors Over Time
Временное ограничение: Up to approximately 274 weeks
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Central laboratory measurement of anti-FVIII inhibitory antibodies (inhibitors) was performed using a Chromogenic Bethesda Assay (CBA).
Participants who develop anti-FVIII inhibitors (titer ≥ 0.6 Bethesda units per milliliter [BU/mL]) following study drug administration were summarized.
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Up to approximately 274 weeks
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Соавторы и исследователи
Здесь вы найдете людей и организации, участвующие в этом исследовании.
Спонсор
Следователи
- Директор по исследованиям: Clinical Trials, Hoffmann-La Roche
Публикации и полезные ссылки
Лицо, ответственное за внесение сведений об исследовании, добровольно предоставляет эти публикации. Это может быть что угодно, связанное с исследованием.
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)
10 февраля 2020 г.
Первичное завершение (Действительный)
30 октября 2021 г.
Завершение исследования (Действительный)
19 декабря 2025 г.
Даты регистрации исследования
Первый отправленный
7 ноября 2019 г.
Впервые представлено, что соответствует критериям контроля качества
7 ноября 2019 г.
Первый опубликованный (Действительный)
12 ноября 2019 г.
Обновления учебных записей
Последнее опубликованное обновление (Действительный)
27 августа 2026 г.
Последнее отправленное обновление, отвечающее критериям контроля качества
5 августа 2026 г.
Последняя проверка
1 августа 2026 г.
Дополнительная информация
Термины, связанные с этим исследованием
Ключевые слова
Дополнительные соответствующие термины MeSH
- Генетические заболевания, врожденные
- Гематологические заболевания
- Нарушения свертывания крови
- Геморрагические расстройства
- Нарушения свертывания крови, наследственные
- Нарушения белковой коагуляции
- Врожденные, наследственные и неонатальные заболевания и аномалии
- Гемики и лимфатические заболевания
- Гемофилия А
- эмицизумаб
Другие идентификационные номера исследования
- BO41423
- 2019 (Грант/контракт NIH США: Chief Medical Office (CMO) Alberta Health Services)
- 2023 (Грант/контракт NIH США: GRAMMY Museum Foundation)
- 2019-002179-32 (Номер EudraCT)
- 2023-506610-52-00 (Идентификатор реестра: EU CT Number)
Планирование данных отдельных участников (IPD)
Планируете делиться данными об отдельных участниках (IPD)?
ДА
Описание плана IPD
Квалифицированные исследователи могут запросить доступ к данным на уровне отдельных пациентов через платформу запросов (www.vivli.org). Дополнительные сведения о критериях «Рош» для приемлемых исследований доступны здесь (https://vivli.org/ourmember/roche/).
Дополнительные сведения о Глобальной политике компании «Рош» в отношении обмена информацией о клинических исследованиях и о том, как запросить доступ к соответствующим документам клинических исследований, см. здесь (https://www.roche.com/research_and_development/who_we_are_how_we_work/clinical_trials/our_commitment_to_data_sharing.htm).
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. .