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Study of Safety And Efficacy Of ReFacto AF In Previously Untreated Hemophilia A Patients In The Usual Care Setting

10 мая 2019 г. обновлено: Pfizer

AN OPEN-LABEL STUDY OF THE SAFETY AND EFFICACY OF REFACTO AF IN PREVIOUSLY UNTREATED PATIENTS IN USUAL CARE SETTINGS

Study to evaluate the safety and effectiveness of ReFacto AF for the treatment of severe hemophilia A in patients who have not yet received treatment for their hemophilia. Study subjects will be males less than 6 years old who have not taken any clotting factor or other blood products before the study. The safety and effectiveness of ReFacto AF will be determined in this study by tests and procedures done at the doctor's office.

Обзор исследования

Статус

Завершенный

Условия

Вмешательство/лечение

Подробное описание

Regulatory Commitment

Тип исследования

Интервенционный

Регистрация (Действительный)

23

Фаза

  • Фаза 4

Контакты и местонахождение

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

Места учебы

  • Германия
      • Bremen, Германия, 28177
        • Klinikum Bremen-Mitte gGmbH, Professor Hess Kinderklinik
    • Niedersachsen
      • Hannover, Niedersachsen, Германия, 30625
        • Medizinische Hochschule, Hannover
  • Испания
      • Madrid, Испания, 28046
        • Hospital Universitario La Paz
      • Valencia, Испания, 46009
        • Hospital Universitario La Fe
  • Италия
      • Firenze, Италия, 50134
        • Agenzia per L'emofilia e Centro Regionale Riferimento Coagulopatie Congenite
  • Нидерланды
      • Groningen, Нидерланды, 9713 GZ
        • University Medical Center Groningen
  • Турция
      • Adana, Турция, 01330
        • Cukurova Universitesi Tip Fakultesi
      • Istanbul, Турция, 34098
        • Istanbul Universitesi Cerrahpasa Tip Fakultesi
    • Izmir
      • Bornova, Izmir, Турция, 35100
        • Ege Universitesi Tip Fakultesi
  • Украина
      • Lviv, Украина, 79044
        • Derzhavna ustanova "Instytut patolohii krovi ta transfuziinoi medytsyny Natsionalnoi akademii
  • Франция
      • Nantes, Франция, 44093
        • CHU de Nantes

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

Не старше 6 лет (Ребенок)

Принимает здоровых добровольцев

Нет

Полы, имеющие право на обучение

Мужской

Описание

Inclusion Criteria:

  • Male subjects <6 years of age with severe hemophilia A (FVIII:C <1%) based on clinical records, including newborns.
  • No prior exposure to factor products or any blood products.

Exclusion Criteria:

  • Presence of any bleeding disorder in addition to hemophilia A.
  • Treatment with any investigational agent or device within the past 30 days.
  • Any condition(s) that compromises the ability to collect study-related observations, or that poses a contraindication to study participation (these conditions include, but are not limited to, inadequate medical history to assure study eligibility; and expectation of poor adherence to study requirements).

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: Другой
  • Распределение: Н/Д
  • Интервенционная модель: Одногрупповое задание
  • Маскировка: Нет (открытая этикетка)

Оружие и интервенции

Группа участников / Армия
Вмешательство/лечение
Экспериментальный: 1
The investigator treats subjects with ReFacto AF in the usual care setting.
Процедура: Laboratory Tests
Blood draws to determine the level of factor VIII activity before and after dosing at appropriate visits, and blood draws to determine levels of factor VIII inhibitor (antibody to factor VIII).
Другие имена:
  • РеФакто АФ

Что измеряет исследование?

Первичные показатели результатов

Мера результата
Мера Описание
Временное ограничение
Percentage of Participants Who Developed Clinically Significant Factor VIII (FVIII) Inhibitors During the Course of the Study
Временное ограничение: 2 years
Percentage of participants who developed clinically significant FVIII inhibitors: those persistent over a defined period with clinically impactful effects like breakthrough bleed, low recovery, etc., during the course of the study.
2 years

Вторичные показатели результатов

Мера результата
Мера Описание
Временное ограничение
Annualized Bleeding Rate (ABR)
Временное ограничение: 2 years
Annualized bleeding rate was calculated as the number of bleeds divided by the treatment interval duration (enrollment visit to final visit) and then multiplied by 365.25. If there was more than 1 bleed location (like ankle and joint) with identical bleed start date and time, it was treated as 1 bleed occurrence.
2 years
Total Number of Infusions to Treat a New Bleed Classified on Basis of Response to First On-Demand Treatment With Refacto AF
Временное ограничение: 2 years
Number of infusions of Refacto AF required to treat a new bleed were classified on basis of the response to at 4-point response scale of assessment (excellent, good, moderate and no response). Assessment was completed each time a participant experienced a new bleed requiring an 'on-demand' IV infusion. Excellent: definite pain relief and/or improvement in bleeding signs within 8 hours (hr) after infusion, no additional infusion administered; Good: definite pain relief and/or improvement in bleeding signs within 8 hr after infusion, at least 1 additional infusion administered for complete resolution or with no additional infusion administered; Moderate: probable or slight improvement starting after 8 hr following infusion, at least 1 additional infusion administered for complete resolution; No Response: no improvement at all between infusions or during 24 hr interval following infusion or condition worsen. Bleeds for which response not recorded, reported as: Data Not Recorded.
2 years
Total Number of Infusions Needed for Resolution of Bleeding Episodes Classified on Basis of Response to Study Drug Infusion
Временное ограничение: Within 48 hours after infusion, up to 2 years treatment duration
Number of infusions of Refacto AF required for resolution of a bleeding episodes were classified on basis of the response at 4-point response scale of assessment (excellent, good, moderate and no response). Excellent: definite pain relief and/or improvement in bleeding signs within 8 hours (hr) after infusion, no additional infusion administered; Good: definite pain relief and/or improvement in bleeding signs within 8 hr after infusion, at least 1 additional infusion administered for complete resolution or with no additional infusion administered; Moderate: probable or slight improvement starting after 8 hr following infusion, at least 1 additional infusion administered for complete resolution; No Response: no improvement at all between infusions or during 24 hr interval following infusion or condition worsen. Bleeds for which response not recorded, reported as: Data Not Recorded.
Within 48 hours after infusion, up to 2 years treatment duration
Total Number of Breakthrough Bleeding Episodes Occurring Within 48 Hours After a Prophylaxis Infusion of ReFacto AF
Временное ограничение: 2 years
The number of breakthrough bleeds (spontaneous or traumatic) within 48 hours following a prophylaxis dose of ReFacto AF are summarized. If there was more than 1 bleed location (like ankle and joint) with identical bleed start date and time, it was treated as 1 bleed occurrence.
2 years
Consumption of Total International Units of Factor VIII
Временное ограничение: 2 years
2 years
Consumption of Total International Units of Factor VIII Per Year
Временное ограничение: 2 years
Consumption of total international units of Factor VIII per year was calculated for a participant: dividing the total consumption of factor VIII by participant's treatment interval duration (in days), then multiplying by 365.25.
2 years
Mean Dose (IU) of Study Drug Consumed Per Infusion
Временное ограничение: 2 years
Mean dose for each participant was calculated as participant's total factor VIII consumption (in IU) divided by the number of infusions administered.
2 years
Consumption of Total International Units of Factor VIII by Weight
Временное ограничение: 2 years
Consumption of total international units of Factor VIII by weight was calculated for a participant: dividing the total consumption of factor VIII by participant's weight (the most recently recorded).
2 years
Consumption of Total International Units of Factor VIII Per Year by Weight
Временное ограничение: 2 years
Consumption of total international units of Factor VIII per year by weight was calculated for a participant: the total consumption of factor VIII divided by participant's treatment interval duration (in days), then multiplying by 365.25 and then dividing by participant's weight (the most recently recorded).
2 years
Mean Dose (IU) of Study Drug Consumed Per Infusion by Weight
Временное ограничение: 2 years
Mean dose for each participant was calculated as participant's total factor consumption (in IU) divided by the number of infusions administered and then dividing by participant's weight (the most recently recorded).
2 years
Mean of Total Number of Infusions of Study Drug Received
Временное ограничение: 2 years
2 years
Mean of Total Number of Days Participants Exposed to Study Drug
Временное ограничение: 2 years
2 years
Number of Participants Who Required Dose Escalation of Their Prescribed Prophylaxis Regimen During Their Participation in This Study
Временное ограничение: 2 years
The number of participants who met the dose escalation criteria were prescribed a higher dose and/or were prescribed more frequent doses. When dose escalation was required, the specific dose and dosing schedule was at the investigator's discretion.
2 years
Percentage of Bleeding Episodes With Less-Than-Expected Therapeutic Effect (LETE) in On-Demand (OD) Setting
Временное ограничение: 2 years
LETE occurs in OD setting if participant recorded 2 successive "No Response" (no improvement at all between infusions, or condition worsens) ratings after 2 successive infusions of study drug. Infusions must have been given within 24 hours (hr) of each other for treatment of same bleeding event in absence of confounding factors (known presence or subsequent identification of a FVIII inhibitor, known inadequate dose for type and/or severity of bleed in opinion of investigator, delay of greater than (>) 4 hr between onset of bleed to infusion, delay of >24 hr before administration of a follow-up infusion, known compromised study drug, faulty administration of study drug, participant had an underlying, predisposing condition responsible for bleed in opinion of investigator, ongoing trauma responsible for continued bleeding.
2 years
Percentage of Bleeding Episodes With Less-Than-Expected Therapeutic Effect (LETE) in the Prophylaxis Setting
Временное ограничение: 2 years
LETE in prophylaxis setting if there was a spontaneous bleed within 48 hours after a regularly scheduled prophylactic dose of study drug (which was not used to treat a bleed) in the absence of confounding factors (known presence or subsequent identification of a FVIII inhibitor, known inadequate prophylactic dose [a dose less than that prescribed in participant's regimen], known lack of adherence to the prescribed prophylaxis regimen, known compromised study drug, faulty administration of study drug, participant had an underlying, predisposing condition responsible for the bleed in the opinion of the investigator, traumatic injury responsible for bleeding.
2 years
Total Number of Events of Potential Less-Than-Expected Therapeutic Effect (LETE) in the Low Recovery Setting
Временное ограничение: 2 years
LETE was lower than expected recovery of FVIII in the opinion of the investigator following infusion of study drug in the absence of confounding factors (known presence or subsequent identification of a FVIII inhibitor, known compromised study drug, faulty administration of study drug including inadequate dosing).
2 years

Соавторы и исследователи

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

Спонсор

Pfizer

Публикации и полезные ссылки

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

Полезные ссылки

Даты записи исследования

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

Изучение основных дат

Начало исследования (Действительный)

10 февраля 2010 г.

Первичное завершение (Действительный)

24 ноября 2016 г.

Завершение исследования (Действительный)

24 ноября 2016 г.

Даты регистрации исследования

Первый отправленный

29 июля 2009 г.

Впервые представлено, что соответствует критериям контроля качества

30 июля 2009 г.

Первый опубликованный (Оценивать)

31 июля 2009 г.

Обновления учебных записей

Последнее опубликованное обновление (Действительный)

19 июля 2019 г.

Последнее отправленное обновление, отвечающее критериям контроля качества

10 мая 2019 г.

Последняя проверка

1 мая 2019 г.

Дополнительная информация

Термины, связанные с этим исследованием

Ключевые слова

Дополнительные соответствующие термины MeSH

Другие идентификационные номера исследования

  • 3082B2-4434
  • B1831006 (Другой идентификатор: Alias Study Number)
  • 2008-008436-93 (Номер EudraCT)

Планирование данных отдельных участников (IPD)

Планируете делиться данными об отдельных участниках (IPD)?

ДА

Описание плана IPD

Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

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. .

Клинические исследования Гемофилия А

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