- ICH GCP
- Registro de ensaios clínicos dos EUA
- Ensaio Clínico NCT00703911
Observational Registry of NovoSeven® Used as On-demand Treatment of Bleeds in Patients With Haemophilia A and B With Inhibitors (ONE)
22 de novembro de 2016 atualizado por: Novo Nordisk A/S
A Prospective Observational Registry on the Use of NovoSeven® (Activated Recombinant Human Factor VIIa) for on Demand Treatment of Mild to Moderate Bleeds in Haemophilia A and B Patients With Inhibitors
This study was conducted in Africa, Europe, the Middle-East and South America.
The primary objective of this registry was to observe the use of single dose and multi-dose use of activated recombinant human factor VII and to compare short-term outcomes, including effectiveness, safety, quality of life and treatment satisfaction with the approved treatments.
Visão geral do estudo
Status
Concluído
Intervenção / Tratamento
Tipo de estudo
Observacional
Inscrição (Real)
102
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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Mainz, Alemanha, 55127
- Novo Nordisk Investigational Site
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Algiers, Argélia, 16035
- Novo Nordisk Investigational Site
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Riyadh, Arábia Saudita, 3542
- Novo Nordisk Investigational Site
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Brussels, Bélgica, 1070
- Novo Nordisk Investigational Site
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Paris La défense cedex, França, 92932
- Novo Nordisk Investigational Site
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Alphen a/d Rijn, Holanda
- Novo Nordisk Investigational Site
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Rome, Itália, 00144
- Novo Nordisk Investigational Site
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Istanbul, Peru, 34335
- Novo Nordisk Investigational Site
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Warszawa, Polônia, PL-02-274
- Novo Nordisk Investigational Site
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Paco de Arcos, Portugal, 2780-730
- Novo Nordisk Investigational Site
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Crawley, Reino Unido, RH11 9RT
- Novo Nordisk Investigational Site
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Prague, República Checa, 16000
- Novo Nordisk Investigational Site
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Malmö, Suécia, 202 15
- Novo Nordisk Investigational Site
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Caracas, Venezuela
- Novo Nordisk Investigational Site
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Sandton, África do Sul, 2146
- Novo Nordisk Investigational Site
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Vienna, Áustria, A-1010
- Novo Nordisk Investigational 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
- Filho
- Adulto
- Adulto mais velho
Aceita Voluntários Saudáveis
Não
Gêneros Elegíveis para o Estudo
Macho
Método de amostragem
Amostra Não Probabilística
População do estudo
Patients with haemophilia A or B with inhibitors, using activated recombinant human factor VII as on-demand treatment
Descrição
Inclusion Criteria:
- Diagnosed with haemophilia A or B with inhibitors
- Experience mild to moderate spontaneous bleeds which require on-demand treatment and who are currently prescribed activated recombinant human factor VII
- Be able and willing to provide informed consent (or proxy consent by caregiver, if applicable), as required by local research ethics committee, governmental or regulatory authorities
- Be willing to provide information on at least one alternate contact person in the event that the patient be somehow lost-to-follow-up over the course of registry participation (not applicable if patient is withdrawn)
Exclusion Criteria:
- Known hypersensitivity to the active substance or the excipients in the formulation of activated recombinant human factor VII, or to mouse, hamster or bovine protein
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
Coortes e Intervenções
Grupo / Coorte |
Intervenção / Tratamento |
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activated recombinant human factor VII
Male patients above 2 years of age with haemophilia A or B who have developed inhibitors and have been prescribed on-demand treatment of activated recombinant human factor VII at any dose for treatment of mild to moderate spontaneous bleeds
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Treatment of patients experiencing bleeds at the discretion of the physician/caregiver
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 Bleed Treatments Resulting in Effective Bleed Resolution (All Bleed Episodes)
Prazo: within 9 hours of first injection
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The percentage of bleed treatments successfully resulting in bleed resolution.
Analysis only considers the patient's opinion of effectiveness at 9 hours, with a rating of "Effective" considered as successful treatment.
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within 9 hours of first injection
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Percentage of Bleed Treatments Resulting in Effective Bleed Resolution (Spontaneous Bleed Episodes)
Prazo: within 9 hours of first injection
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The percentage of bleed treatments successfully resulting in bleed resolution.
Analysis only considers the patient's opinion of effectiveness at 9 hours, with a rating of "Effective" considered as successful treatment.
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within 9 hours of first injection
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Percentage of Bleed Treatments Resulting in Effective Pain Relief (All Bleed Episodes)
Prazo: within 9 hours of first injection
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The percentage of participants with effective pain relief.
Pain relief was a subjective assessment made by the patient during treatment of a bleed episode.
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within 9 hours of first injection
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Percentage of Bleed Treatments Resulting in Effective Pain Relief (Spontaneous Bleed Episodes)
Prazo: within 9 hours of first injection
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The percentage of participants with effective pain relief.
Pain relief was a subjective assessment made by the patient during treatment of a bleed episode.
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within 9 hours of first injection
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Medidas de resultados secundários
Medida de resultado |
Descrição da medida |
Prazo |
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Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleeds) by Time Point (All Bleed Episodes)
Prazo: 1 hour, 3 hours and 6 hours, respectively, after first injection
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Effective haemostasis at 3 different time points for all bleeds.
Patient reported outcomes are reported over 1 hour, 3 hours and 6 hours.
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1 hour, 3 hours and 6 hours, respectively, after first injection
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Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleeds) by Time Point (Spontaneous Bleed Episodes)
Prazo: 1 hour, 3 hours and 6 hours, respectively, after first injection
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Effective haemostasis at 3 different time points for all bleeds.
Patient reported outcomes are reported over 1 hour, 3 hours and 6 hours.
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1 hour, 3 hours and 6 hours, respectively, after first injection
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Percentage of Bleed Treatments Resulting in Effective Pain Relief by Time Point (All Bleed Episodes)
Prazo: 1 hour, 3 hours and 6 hours, respectively, after first injection
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Effective pain relief at 3 different time points for all bleeds.
Patient reported outcomes are reported over 1 hour, 3 hours and 6 hours.
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1 hour, 3 hours and 6 hours, respectively, after first injection
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Percentage of Bleed Treatments Resulting in Effective Pain Relief by Time Point (Spontaneous Bleed Episodes)
Prazo: 1 hour, 3 hours and 6 hours, respectively, after first injection
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Effective pain relief at 3 different time points for spontaneous bleeds.
Patient reported outcomes are reported over 1 hour, 3 hours and 6 hours.
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1 hour, 3 hours and 6 hours, respectively, after first injection
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Total Number of Injections (All Bleed Episodes)
Prazo: individual bleed episode
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The median number of injections required to treat individual bleed episodes.
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individual bleed episode
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Total Number of Injections (Spontaneous Bleed Episodes)
Prazo: individual bleed episode
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The median number of injections required to treat individual bleed episodes.
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individual bleed episode
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Total Exposure (Cumulative Dose) to Activated Recombinant Human Factor VII (All Bleed Episodes)
Prazo: individual bleed episode
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The median total cumulative dose required to treat individual bleed episodes.
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individual bleed episode
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Total Exposure (Cumulative Dose) to Activated Recombinant Human Factor VII (Spontaneous Bleed Episodes)
Prazo: individual bleed episode
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The median total cumulative dose required to treat individual bleed episodes.
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individual bleed episode
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Percentage of Patients Reporting Satisfaction With Symptom Relief (All Bleed Episodes)
Prazo: duration of bleed episode
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Patient rate of satisfaction with symptom relief for the bleed episode overall on a 7-point Likert scale.
Completion of questionnaire was voluntary.
A Likert scale is an ordered, multiple choice questionnaire from which respondents choose one option that best aligns with their view of the particular outcome being measured.
Scale answers ranged from extremely satisfied to extremely dissatisfied.
The percentage of bleed episodes for which patients reported "extremely satisfied", "very satisfied" or "satisfied" is presented.
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duration of bleed episode
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Percentage of Bleed Treatments Resulting in Patient Satisfaction With Symptom Relief (Spontaneous Bleed Episodes)
Prazo: duration of bleed episode
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Patient rate of satisfaction with symptom relief for the bleed episode overall on a 7-point Likert scale.
Completion of questionnaire was voluntary.
A Likert scale is an ordered, multiple choice questionnaire from which respondents choose one option that best aligns with their view of the particular outcome being measured.
Scale answers ranged from extremely satisfied to extremely dissatisfied.
The percentage of bleed episodes for which patients reported "extremely satisfied", "very satisfied" or "satisfied" is presented.
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duration of bleed episode
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Percentage of Bleed Treatments Resulting in Patient Satisfaction With Ease of Use (All Bleed Episodes)
Prazo: duration of bleed episode
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Rate of ease of use related to activated recombinant human factor VII on a 7-point Likert scale.
Completion of questionnaire was voluntary.
A Likert scale is an ordered, multiple choice questionnaire from which respondents choose one option that best aligns with their view of the particular outcome being measured.
Scale answers ranged from extremely difficult to extremely easy.
The percentage of bleed episodes for which patients reported "extremely easy", "very easy" or "easy" is presented.
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duration of bleed episode
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Percentage of Bleed Treatments Resulting in Patient Satisfaction With Ease of Use (Spontaneous Bleed Episodes)
Prazo: duration of bleed episode
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Rate of ease of use related to activated recombinant human factor VII on a 7-point Likert scale.
Completion of questionnaire was voluntary.
A Likert scale is an ordered, multiple choice questionnaire from which respondents choose one option that best aligns with their view of the particular outcome being measured.
Scale answers ranged from extremely difficult to extremely easy.
The percentage of bleed episodes for which patients reported "extremely easy", "very easy" or "easy" is presented.
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duration of bleed episode
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Overall Time to Cessation of Bleed/Achievement of Haemostasis (All Bleed Episodes)
Prazo: duration of bleed episode
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Median time to achievement of haemostasis/bleed cessation calculated using Kaplan Meier life table methods.
If approximately 50% or less of bleeds achieved the endpoint in a given initial dose subgroup, the median cannot be calculated and it is reported as not applicable
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duration of bleed episode
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Overall Time to Cessation of Bleed/Achievement of Haemostasis (Spontaneous Bleed Episodes)
Prazo: duration of bleed episode
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Median time to achievement of haemostasis/bleed cessation calculated using Kaplan Meier life table methods.
If approximately 50% or less of bleeds achieved the endpoint in a given initial dose subgroup, the median cannot be calculated and it is reported as not applicable
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duration of bleed episode
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Overall Time to Cessation/Achievement of Haemostasis (Spontaneous Bleed Episodes)
Prazo: duration of bleed episode
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Median time to achievement of haemostasis/bleed cessation calculated using Kaplan Meier life table methods.
If approximately 50% or less of bleeds achieved the endpoint in a given initial dose subgroup, the median cannot be calculated and it is reported as not applicable.
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duration of bleed episode
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Childrens' Health Related Quality of Life (Haemo-QoL): Overall Score
Prazo: Baseline (week 0) and and registry discontinuation (up to 28 months)
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The Haemo-QoL is a specific multidimensional validated and reliable questionnaire used to assess quality of life in patients with haemophilia.
Scores are reported on a 0 to 100 scale-higher scores indicate more impairment.
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Baseline (week 0) and and registry discontinuation (up to 28 months)
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Adults' Health Related Quality of Life (Haemo-QoL-A): Overall Score
Prazo: Baseline (week 0) and and registry discontinuation (up to 28 months)
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The adult Haemo-QoL-A is a specific multidimensional validated and reliable questionnaire used to assess quality of life in patients with haemophilia.
Scores are reported on a 0 to 100 scale-higher scores indicate more impairment.
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Baseline (week 0) and and registry discontinuation (up to 28 months)
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Outras medidas de resultado
Medida de resultado |
Descrição da medida |
Prazo |
---|---|---|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleed) by Dose Level (All Bleed Episodes)
Prazo: within 9 hours after first injection
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Percentage of bleeds with effective haemostasis within 9 hours of first injection of activated recombinant human factor VII. Patient reported assessments were sorted into 3 sub-categories: "effective" = bleed resolved or substantially improved; "partially effective" = bleed with some improvement; "ineffective" = bleed with no change or with worsening.
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within 9 hours after first injection
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Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleed) by Dose Level (Spontaneous Bleed Episodes)
Prazo: within 9 hours after first injection
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Percentage of bleeds with effective haemostasis within 9 hours of first injection of activated recombinant human factor VII. Patient reported assessments were sorted into 3 sub-categories: "effective" = bleed resolved or substantially improved; "partially effective" = bleed with some improvement; "ineffective" = bleed with no change or with worsening.
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within 9 hours after first injection
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Percentage of Bleed Treatments Resulting in Effective Pain Relief by Initial Dose (All Bleed Episodes)
Prazo: within 9 hours after first injection
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Percentage of bleeds with effective pain relief within 9 hours of first injection of activated recombinant human factor VII. Patient reported assessments were sorted into 3 sub-categories: "Better" = pain resolved or decreased substantially; "Same" = no change; "Worsened" = pain worsening.
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within 9 hours after first injection
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Percentage of Bleed Treatments Resulting in Effective Pain Relief by Initial Dose (Spontaneous Bleed Episodes)
Prazo: within 9 hours after first injection
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Percentage of bleeds with effective pain relief within 9 hours of first injection of activated recombinant human factor VII. Patient reported assessments were sorted into 3 sub-categories: "Better" = pain resolved or decreased substantially; "Same" = no change; "Worsened" = pain worsening.
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within 9 hours after first injection
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Colaboradores e Investigadores
É aqui que você encontrará pessoas e organizações envolvidas com este estudo.
Patrocinador
Publicações e links úteis
A pessoa responsável por inserir informações sobre o estudo fornece voluntariamente essas publicações. Estes podem ser sobre qualquer coisa relacionada ao estudo.
Links úteis
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 março de 2008
Conclusão Primária (Real)
1 de julho de 2010
Conclusão do estudo (Real)
1 de julho de 2010
Datas de inscrição no estudo
Enviado pela primeira vez
19 de junho de 2008
Enviado pela primeira vez que atendeu aos critérios de CQ
23 de junho de 2008
Primeira postagem (Estimativa)
24 de junho de 2008
Atualizações de registro de estudo
Última Atualização Postada (Estimativa)
11 de janeiro de 2017
Última atualização enviada que atendeu aos critérios de controle de qualidade
22 de novembro de 2016
Última verificação
1 de novembro de 2016
Mais Informações
Termos relacionados a este estudo
Termos MeSH relevantes adicionais
- Doenças cardiovasculares
- Doenças Vasculares
- Doenças Hematológicas
- Distúrbios da Coagulação Sanguínea, Herdados
- Distúrbios da Proteína de Coagulação
- Distúrbios hemorrágicos
- Doenças Genéticas, Congênitas
- Doenças Genéticas, Ligadas ao X
- Distúrbios hemostáticos
- Hemofilia A
- Hemofilia B
- Distúrbios da Coagulação Sanguínea
Outros números de identificação do estudo
- F7HAEM-3507
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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