Study of Safety And Efficacy Of ReFacto AF In Previously Untreated Hemophilia A Patients In The Usual Care Setting
10 de maio de 2019 atualizado por: 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.
Visão geral do estudo
Descrição detalhada
Regulatory Commitment
Tipo de estudo
Intervencional
Inscrição (Real)
23
Estágio
- Fase 4
Contactos e Locais
Esta seção fornece os detalhes de contato para aqueles que conduzem o estudo e informações sobre onde este estudo está sendo realizado.
Locais de estudo
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Bremen, Alemanha, 28177
- Klinikum Bremen-Mitte gGmbH, Professor Hess Kinderklinik
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Niedersachsen
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Hannover, Niedersachsen, Alemanha, 30625
- Medizinische Hochschule, Hannover
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Madrid, Espanha, 28046
- Hospital Universitario La Paz
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Valencia, Espanha, 46009
- Hospital Universitario La Fe
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Nantes, França, 44093
- CHU de Nantes
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Groningen, Holanda, 9713 GZ
- University Medical Center Groningen
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Firenze, Itália, 50134
- Agenzia per L'emofilia e Centro Regionale Riferimento Coagulopatie Congenite
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Adana, Peru, 01330
- Cukurova Universitesi Tip Fakultesi
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Istanbul, Peru, 34098
- Istanbul Universitesi Cerrahpasa Tip Fakultesi
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Izmir
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Bornova, Izmir, Peru, 35100
- Ege Universitesi Tip Fakultesi
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Lviv, Ucrânia, 79044
- Derzhavna ustanova "Instytut patolohii krovi ta transfuziinoi medytsyny Natsionalnoi akademii
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Critérios de participação
Os pesquisadores procuram pessoas que se encaixem em uma determinada descrição, chamada de critérios de elegibilidade. Alguns exemplos desses critérios são a condição geral de saúde de uma pessoa ou tratamentos anteriores.
Critérios de elegibilidade
Idades elegíveis para estudo
Não mais velho que 6 anos (Filho)
Aceita Voluntários Saudáveis
Não
Gêneros Elegíveis para o Estudo
Macho
Descrição
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).
Plano de estudo
Esta seção fornece detalhes do plano de estudo, incluindo como o estudo é projetado e o que o estudo está medindo.
Como o estudo é projetado?
Detalhes do projeto
- Finalidade Principal: Outro
- Alocação: N / D
- Modelo Intervencional: Atribuição de grupo único
- Mascaramento: Nenhum (rótulo aberto)
Armas e Intervenções
Grupo de Participantes / Braço |
Intervenção / Tratamento |
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Experimental: 1
The investigator treats subjects with ReFacto AF in the usual care setting.
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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).
Outros nomes:
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O que o estudo está medindo?
Medidas de resultados primários
Medida de resultado |
Descrição da medida |
Prazo |
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Percentage of Participants Who Developed Clinically Significant Factor VIII (FVIII) Inhibitors During the Course of the Study
Prazo: 2 years
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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.
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2 years
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Medidas de resultados secundários
Medida de resultado |
Descrição da medida |
Prazo |
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Annualized Bleeding Rate (ABR)
Prazo: 2 years
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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.
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2 years
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Total Number of Infusions to Treat a New Bleed Classified on Basis of Response to First On-Demand Treatment With Refacto AF
Prazo: 2 years
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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.
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2 years
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Total Number of Infusions Needed for Resolution of Bleeding Episodes Classified on Basis of Response to Study Drug Infusion
Prazo: Within 48 hours after infusion, up to 2 years treatment duration
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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.
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Within 48 hours after infusion, up to 2 years treatment duration
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Total Number of Breakthrough Bleeding Episodes Occurring Within 48 Hours After a Prophylaxis Infusion of ReFacto AF
Prazo: 2 years
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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.
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2 years
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Consumption of Total International Units of Factor VIII
Prazo: 2 years
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2 years
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Consumption of Total International Units of Factor VIII Per Year
Prazo: 2 years
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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.
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2 years
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Mean Dose (IU) of Study Drug Consumed Per Infusion
Prazo: 2 years
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Mean dose for each participant was calculated as participant's total factor VIII consumption (in IU) divided by the number of infusions administered.
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2 years
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Consumption of Total International Units of Factor VIII by Weight
Prazo: 2 years
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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).
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2 years
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Consumption of Total International Units of Factor VIII Per Year by Weight
Prazo: 2 years
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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).
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2 years
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Mean Dose (IU) of Study Drug Consumed Per Infusion by Weight
Prazo: 2 years
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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).
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2 years
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Mean of Total Number of Infusions of Study Drug Received
Prazo: 2 years
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2 years
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Mean of Total Number of Days Participants Exposed to Study Drug
Prazo: 2 years
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2 years
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Number of Participants Who Required Dose Escalation of Their Prescribed Prophylaxis Regimen During Their Participation in This Study
Prazo: 2 years
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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.
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2 years
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Percentage of Bleeding Episodes With Less-Than-Expected Therapeutic Effect (LETE) in On-Demand (OD) Setting
Prazo: 2 years
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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.
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2 years
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Percentage of Bleeding Episodes With Less-Than-Expected Therapeutic Effect (LETE) in the Prophylaxis Setting
Prazo: 2 years
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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.
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2 years
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Total Number of Events of Potential Less-Than-Expected Therapeutic Effect (LETE) in the Low Recovery Setting
Prazo: 2 years
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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).
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2 years
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Colaboradores e Investigadores
É aqui que você encontrará pessoas e organizações envolvidas com este estudo.
Patrocinador
Publicações e links úteis
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Datas de registro do estudo
Essas datas acompanham o progresso do registro do estudo e os envios de resumo dos resultados para ClinicalTrials.gov. Os registros do estudo e os resultados relatados são revisados pela National Library of Medicine (NLM) para garantir que atendam aos padrões específicos de controle de qualidade antes de serem publicados no site público.
Datas Principais do Estudo
Início do estudo (Real)
10 de fevereiro de 2010
Conclusão Primária (Real)
24 de novembro de 2016
Conclusão do estudo (Real)
24 de novembro de 2016
Datas de inscrição no estudo
Enviado pela primeira vez
29 de julho de 2009
Enviado pela primeira vez que atendeu aos critérios de CQ
30 de julho de 2009
Primeira postagem (Estimativa)
31 de julho de 2009
Atualizações de registro de estudo
Última Atualização Postada (Real)
19 de julho de 2019
Última atualização enviada que atendeu aos critérios de controle de qualidade
10 de maio de 2019
Última verificação
1 de maio de 2019
Mais Informações
Termos relacionados a este estudo
Palavras-chave
Termos MeSH relevantes adicionais
Outros números de identificação do estudo
- 3082B2-4434
- B1831006 (Outro identificador: Alias Study Number)
- 2008-008436-93 (Número EudraCT)
Plano para dados de participantes individuais (IPD)
Planeja compartilhar dados de participantes individuais (IPD)?
SIM
Descrição do plano 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.
Essas informações foram obtidas diretamente do site clinicaltrials.gov sem nenhuma alteração. Se você tiver alguma solicitação para alterar, remover ou atualizar os detalhes do seu estudo, entre em contato com register@clinicaltrials.gov. Assim que uma alteração for implementada em clinicaltrials.gov, ela também será atualizada automaticamente em nosso site .