Study of Biostate® in Children With Von Willebrand Disease
2 de outubro de 2017 atualizado por: CSL Behring
A Phase III Open-label, Multi-centre Study to Assess the Pharmacokinetics, Efficacy, and Safety of Biostate® in Paediatric Subjects With Von Willebrand Disease
This is an open-label study to investigate the pharmacokinetics (PK), efficacy, and safety of a von Willebrand Factor/Factor VIII (VWF/FVIII), Biostate, in children with Von Willebrand disease (VWD) in whom treatment with a VWF product is required for prophylactic therapy, haemostatic control during surgery, or control of a non-surgical, spontaneous, or traumatic bleeding event.
Visão geral do estudo
Tipo de estudo
Intervencional
Inscrição (Real)
17
Estágio
- Fase 3
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
- Study Site
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Homel, Bielorrússia, 246040
- Study Site
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Minsk, Bielorrússia, 223040
- Study Site
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Tbilisi, Geórgia, 0179
- Study Site
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CP
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Guatemala, CP, Guatemala, 01010
- Study Site
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Beirut, Líbano
- Study Site
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Lviv, Ucrânia
- Study Site
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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 12 anos (Filho)
Aceita Voluntários Saudáveis
Não
Gêneros Elegíveis para o Estudo
Tudo
Descrição
Inclusion Criteria:
- Male and female subjects between 0 and <12 years of age
- Diagnosed with VWD Type 1, 2A, or 3
- Desmopressin acetate (DDAVP) treatment is ineffective, contraindicated, or not available for subject
- von Willebrand factor: ristocetin cofactor (VWF:RCo) is <20% at screening or the subject has a history of VWF:RCo <10%
- Evidence of vaccination against hepatitis A and B or presence of antibodies against hepatitis A and B due to either a previous infection or prior immunization
- Written informed consent given
Exclusion Criteria:
- Active bleeding immediately prior to initial PK period
- Received treatment with DDAVP or a VWF concentrate product for their VWD in the 5 days prior to their first study treatment
- Have received aspirin or other non-steroidal anti-inflammatory drugs (NSAIDs) within 7 days of commencing the PK period.
- Known history or suspicion of having VWF or FVIII inhibitors
- Acute or chronic medical condition, other than VWD, which may affect the conduct of the study
- Known or suspected hypersensitivity or previous evidence of severe side effects to other FVIII/VWF concentrates
- Participation in a clinical study or use of an investigational compound in another study in the 3 months preceding study start
- Unwillingness and/or inability to comply with the 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: Tratamento
- 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: Biostate
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PK component: Single bolus infusion of 80 IU VWF:RCo/kg administered intravenously on Day 1, and approximately Day 180 in Type 3 VWD subjects only. Efficacy component: Repeated bolus doses over 12 months as required to manage VWD condition. |
O que o estudo está medindo?
Medidas de resultados primários
Medida de resultado |
Prazo |
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Half-life of FVIII
Prazo: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
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Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
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Mean residence time (MRT) of FVIII
Prazo: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
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Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
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Haemostatic efficacy
Prazo: From Day 1 until final study visit
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From Day 1 until final study visit
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Incremental Recovery of VWF
Prazo: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
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Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
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Incremental Recovery of FVIII
Prazo: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
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Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
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Half-life of VWF
Prazo: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
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Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
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Area under the concentration curve (AUC) of VWF
Prazo: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
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Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
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AUC of FVIII
Prazo: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
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Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
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Maximum plasma concentration (Cmax) of VWF
Prazo: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
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Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
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Maximum plasma concentration (Cmax) of FVIII
Prazo: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
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Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
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Minimum plasma concentration (Cmin) of VWF
Prazo: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
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Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
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Minimum plasma concentration (Cmin) of FVIII
Prazo: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
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Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
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Time to maximum concentration (tmax) of VWF
Prazo: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
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Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
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Time to maximum concentration (tmax) of FVIII
Prazo: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
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Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
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Mean residence time (MRT) of VWF
Prazo: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
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Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
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Clearance (CL) of VWF
Prazo: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
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Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
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Clearance (CL) of FVIII
Prazo: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
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Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
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Volume of distribution of steady state (Vss) of VWF
Prazo: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
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Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
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Volume of distribution of steady state (Vss) of FVIII
Prazo: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
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Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
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Medidas de resultados secundários
Medida de resultado |
Prazo |
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Frequency of adverse events (AEs) per infusion
Prazo: 13 months
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13 months
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Severity of AEs per infusion
Prazo: 13 months
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13 months
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Severity of AEs per subject
Prazo: 13 months
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13 months
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Relatedness of AEs per infusion
Prazo: 13 months
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13 months
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Relatedness of AEs per subject
Prazo: 13 months
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13 months
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Development of VWF inhibitors
Prazo: Sample taken at baseline, then every 3 months up to 12 months
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Sample taken at baseline, then every 3 months up to 12 months
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Development of FVIII inhibitors
Prazo: Sample taken at baseline, then every 3 months up to 12 months
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Sample taken at baseline, then every 3 months up to 12 months
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Frequency of adverse events (AEs) per subject
Prazo: 13 months
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13 months
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Colaboradores e Investigadores
É aqui que você encontrará pessoas e organizações envolvidas com este estudo.
Patrocinador
Colaboradores
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
1 de agosto de 2010
Conclusão Primária (Real)
1 de agosto de 2013
Conclusão do estudo (Real)
1 de agosto de 2013
Datas de inscrição no estudo
Enviado pela primeira vez
1 de outubro de 2010
Enviado pela primeira vez que atendeu aos critérios de CQ
1 de outubro de 2010
Primeira postagem (Estimativa)
4 de outubro de 2010
Atualizações de registro de estudo
Última Atualização Postada (Real)
3 de outubro de 2017
Última atualização enviada que atendeu aos critérios de controle de qualidade
2 de outubro de 2017
Última verificação
1 de outubro de 2017
Mais Informações
Termos relacionados a este estudo
Palavras-chave
Termos MeSH relevantes adicionais
Outros números de identificação do estudo
- CSLCT-BIO-08-52
- 1494 (CSL Behring)
- 2009-017753-34 (Número EudraCT)
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