- ICH GCP
- Registro de ensaios clínicos dos EUA
- Ensaio Clínico NCT02954575
Clinical Study to Investigate the PK, Efficacy, and Safety of Wilate in Patients With Severe Hemophilia A
21 de dezembro de 2020 atualizado por: Octapharma
Clinical Study to Investigate the Pharmacokinetics, Efficacy, Safety, and Immunogenicity of Wilate in Previously Treated Patients With Severe Hemophilia A
The purpose of this study is to obtain additional data on the safety and efficacy of Wilate in PTPs with hemophilia A with at least 150 previous exposure days (EDs) to a FVIII concentrate who undergo prophylactic treatment with Wilate for 6 months and at least 50 EDs, thus supplementing the existing database to obtain approval of Wilate for the indication hemophilia A in the USA.
Visão geral do estudo
Tipo de estudo
Intervencional
Inscrição (Real)
57
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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Sofia, Bulgária
- Specialized Hospital for Active Treatment "Joan Pavel"
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Barnaul, Federação Russa
- Barnaul Branch of RAMS hematology center
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Moscow, Federação Russa
- Federal Scientific Hematology Center
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Budapest, Hungria
- National Haemophilia Centre
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Krakow, Polônia
- Krakowskie Centrum Medyczne
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Rzeszow, Polônia
- Korczowski Bartosz Gabinet Lekarski
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Bucharest, Romênia
- Centrul Medical Unirea -Policlinica Enescu
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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
12 anos e mais velhos (Filho, Adulto, Adulto mais velho)
Aceita Voluntários Saudáveis
Não
Gêneros Elegíveis para o Estudo
Macho
Descrição
Inclusion Criteria:
- Severe hemophilia A (<1% FVIII:C) according to medical history
- Male patients aged ≥12 years
- Previous treatment with a FVIII concentrate for at least 150 exposure days (EDs)
- Immunocompetence (CD4+ count >200/µL)
- Good documentation of the historical bleeding rate (at least for the 6 months preceding study start)
- Voluntarily given, fully informed written and signed consent obtained by the patient (or parent/legal guardian in case of adolescents) before any study-related procedures are conducted
Whenever possible, the interval between the Screening Visit and the PK or Non-PK Visit should not exceed 30 days. If the 30-day interval is exceeded, determination of the CD4+ count is to be repeated and must be >200/µL for patients to be enrolled (i.e., exclusion criterion no. 4).
Exclusion Criteria:
- Any coagulation disorders other than hemophilia A
- History of FVIII inhibitor activity (≥0.6 BU) or detectable FVIII inhibitory anti-bodies (≥0.6 BU using the Nijmegen modification of the Bethesda assay) at screening, as determined by the central laboratory
- Severe liver or kidney diseases (alanine aminotransferase [ALAT] and aspartate transaminase [ASAT] levels >5 times of upper limit of normal, creatinine>120 µmol/L)
- Patients receiving or scheduled to receive immunomodulating drugs (other than anti-retroviral chemotherapy) such as alpha-interferon, prednisone (equivalent to >10 mg/day), or similar drugs
- Treatment with any investigational medicinal product in another interventional clinical study currently or within 4 weeks before enrollment
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: All patients
All patients will receive Wilate for prophylactic treatment
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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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Total Annualized Bleeding Rate (TABR)
Prazo: 6 months
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The total number of bleeding events (BEs) was documented by patients in a patient diary (together with the investigator in case of on-site treatments), which was reviewed at each follow-up visit by site personnel.
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6 months
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Medidas de resultados secundários
Medida de resultado |
Descrição da medida |
Prazo |
---|---|---|
Spontaneous Annualized Bleeding Rate (SABR)
Prazo: 6 months
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The number of spontaneous bleeding events (BEs) was documented by patients in a patient diary (together with the investigator in case of on-site treatments), which was reviewed at each follow-up visit by site personnel.
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6 months
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Efficacy of Wilate in the Treatment of Breakthrough BEs
Prazo: 6 months
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The proportion of BEs successfully treated with Wilate were documented by the patient (together with the investigator in case of on-site treatments) in the patient diary for all BEs according to a 4-point hemostatic efficacy scale including the four items: 'excellent,' 'good,' moderate,' and 'none', where 'excellent' was defined as "Abrupt pain relief and/or unequivocal improvement in objective signs of bleeding within approximately 8 hours after a single injection" (best outcome) and 'none' was defined as "No improvement within 12 hours, or worsening of symptoms, requiring more than two injections for complete resolution" (worst outcome).
All efficacy ratings assessed as either 'excellent' or 'good' were considered 'successfully treated.'
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6 months
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Wilate Consumption Data (Average Total Normdose of FVIII IU/kg Per Month of Study) for Prophylaxis
Prazo: 6 months
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The average consumption of Wilate per month of study (IU/kg) for all patients receiving prophylaxis.
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6 months
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Pharmacokinetic (PK) Assessment (Area Under the Curve [AUC] Norm) of FVIII:C
Prazo: Initial PK visit (Day -1) and PK study completion visit (6 months); data collected 1 h prior to injection and 15 min, 1 h, 3 h, 6 h, 9 h, 24 h, 30 h and 48 h after the end of injection
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PK assessments of FVIII:C were conducted using the one-stage (OS) assay.
The value of the AUCnorm of FVIII:C was calculated based on the FVIII:C values measured in the patients participating in the PK study.
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Initial PK visit (Day -1) and PK study completion visit (6 months); data collected 1 h prior to injection and 15 min, 1 h, 3 h, 6 h, 9 h, 24 h, 30 h and 48 h after the end of injection
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Pharmacokinetic (PK) Assessment (in Vivo Half-Life (t1/2)) of FVIII:C
Prazo: Initial PK assessment (Day -1) and PK study completion visit (6 months); data collected 1 h prior to infusion and 15 min, 1 h, 3 h, 6 h, 9 h, 24 h, 30 h and 48 h after the end of injection
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PK assessments of FVIII:C were conducted using the one-stage (OS) assay.
The in vivo half-life of FVIII:C was calculated based on the FVIII:C values measured in the patients participating in the PK study.
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Initial PK assessment (Day -1) and PK study completion visit (6 months); data collected 1 h prior to infusion and 15 min, 1 h, 3 h, 6 h, 9 h, 24 h, 30 h and 48 h after the end of injection
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Pharmacokinetic (PK) Assessment (Maximum Plasma Concentration [Cmax]) of FVIII:C
Prazo: Initial PK assessment (Day -1) and 6 months
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PK assessments of FVIII:C were conducted using the one-stage (OS) assay.
The maximum plasma concentration of FVIII:C was calculated based on the FVIII:C values measured in the patients participating in the PK study.
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Initial PK assessment (Day -1) and 6 months
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Incremental in Vivo Recovery (IVR) of Wilate Over Time
Prazo: Baseline, 3 and 6 months
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The rise in FVIII activity in IU/dl per unit dose administered in IU/kg was determined from all patients at baseline, 3 and 6 months, using the OS assay.
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Baseline, 3 and 6 months
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Association Between ABO Blood Type and the FVIII:C Half-life of Wilate (OS Assay)
Prazo: 6 months
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Analysis of variance (ANOVA) was used in an exploratory sense to assess an association between ABO blood type and the FVIII:C half-life of Wilate.
This was analyzed by calculating the mean square in a one-stage assay.
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6 months
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Association Between VWF:Ag Concentration and the FVIII:C Half-life of Wilate
Prazo: 6 months
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ANOVA was used in an exploratory sense to assess an association between VWF:Ag with the FVIII:C half-life of Wilate.
This was analyzed by calculating the mean square in a one-stage assay.
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6 months
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Safety and Tolerability of Wilate by Monitoring Adverse Events (AEs) Throughout the Study
Prazo: 6 months
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At each (scheduled or unscheduled) study visit, AEs were documented by the investigator throughout the study.
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6 months
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Immunogenicity of Wilate by Testing for FVIII Inhibitors
Prazo: 6 months
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FVIII inhibitor activity was determined at each study visit before the injection of Wilate using the modified Bethesda assay (Nijmegen modification).
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6 months
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Virus Safety Measured by the Number of Parvovirus B19 Seroconversions Between Baseline (BL) and End of Study
Prazo: 6 months
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Virus safety was evaluated by taking a plasma sample for parvovirus B19 antibody testing before the first injection of Wilate.
All patients negative at screening were tested again at the study completion visit.
The number with Parvovirus B19 seroconversions between BL and end of study was recorded.
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6 months
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Outras medidas de resultado
Medida de resultado |
Descrição da medida |
Prazo |
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Efficacy of Wilate in Surgical Prophylaxis
Prazo: 6 months
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Hemostatic efficacy was assessed at the end of surgery by the surgeon and at end of the postoperative period by the hematologist, using a 4-point hemostatic efficacy scale including the four items: 'excellent' (best possible outcome), 'good', 'moderate' and 'none' (worst outcome).
Overall efficacy was assessed by the investigator, taking both the intra and postoperative assessments into account, and using the 'excellent,' 'good,' moderate,' and 'none' scale.
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6 months
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Colaboradores e Investigadores
É aqui que você encontrará pessoas e organizações envolvidas com este estudo.
Patrocinador
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)
1 de dezembro de 2016
Conclusão Primária (Real)
29 de março de 2018
Conclusão do estudo (Real)
29 de março de 2018
Datas de inscrição no estudo
Enviado pela primeira vez
27 de outubro de 2016
Enviado pela primeira vez que atendeu aos critérios de CQ
1 de novembro de 2016
Primeira postagem (Estimativa)
3 de novembro de 2016
Atualizações de registro de estudo
Última Atualização Postada (Real)
19 de janeiro de 2021
Última atualização enviada que atendeu aos critérios de controle de qualidade
21 de dezembro de 2020
Última verificação
1 de dezembro de 2020
Mais Informações
Termos relacionados a este estudo
Termos MeSH relevantes adicionais
Outros números de identificação do estudo
- WIL-27
Plano para dados de participantes individuais (IPD)
Planeja compartilhar dados de participantes individuais (IPD)?
NÃO
Informações sobre medicamentos e dispositivos, documentos de estudo
Estuda um medicamento regulamentado pela FDA dos EUA
Sim
Estuda um produto de dispositivo regulamentado pela FDA dos EUA
Não
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 .
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