Um estudo para avaliar a segurança, eficácia, farmacocinética e farmacodinâmica do emicizumabe em participantes com hemofilia A leve ou moderada sem inibidores de FVIII (HAVEN 6)
5 de agosto de 2026 atualizado por: Hoffmann-La Roche
Um estudo aberto multicêntrico para avaliar a segurança, eficácia, farmacocinética e farmacodinâmica do emicizumabe em pacientes com hemofilia A leve ou moderada sem inibidores de FVIII
Este é um estudo multicêntrico, aberto e de braço único desenhado para avaliar a segurança, eficácia, farmacocinética e farmacodinâmica do emicizumabe em participantes com hemofilia A leve ou moderada sem inibidores contra o fator VIII (FVIII).
Visão geral do estudo
Status
Concluído
Condições
Intervenção / Tratamento
Tipo de estudo
Intervencional
Inscrição (Real)
73
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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Bonn, Alemanha, 53127
- Universitätsklinikum Bonn
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München, Alemanha, 80336
- Klinikum der Universität München, Campus Innenstadt
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Brussels, Bélgica, 1200
- Cliniques Universitaires St-Luc
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Leuven, Bélgica, 3000
- UZ Leuven Gasthuisberg
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Alberta
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Edmonton, Alberta, Canadá, T6G 1Z1
- Kaye Edmonton Clinic
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Newfoundland and Labrador
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St. John's, Newfoundland and Labrador, Canadá, A1B 3V6
- Eastern Health - General Hospital
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Madrid, Espanha, 28046
- Hospital Universitario La Paz
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Seville, Espanha, 41013
- Hospital Universitario Virgen del Rocío
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California
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Los Angeles, California, Estados Unidos, 90027
- Childrens Hospital LA
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Georgia
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Atlanta, Georgia, Estados Unidos, 30308
- Hemophilia of Georgia Center for Bleeding & Clotting Disorders
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Indiana
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Indianapolis, Indiana, Estados Unidos, 46260
- Indiana Hemophilia & Thrombosis center
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Michigan
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Ann Arbor, Michigan, Estados Unidos, 48109
- University of Michigan, C.S. Mott Children's Hospital
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Washington
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Seattle, Washington, Estados Unidos, 98101
- Washington Institute for Coagulation
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Bron, França, 69677
- Hopital Cardio-vasculaire Louis Pradel
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Le Kremlin-Bicêtre, França, 94275
- CH de Bicêtre
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Paris, França, 75015
- Groupe Hospitalier Necker Enfants Malades
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Amsterdam, Holanda, 1105 AZ
- Amsterdam UMC Location AMC
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Warsaw, Polônia, 02-776
- Instytut Hematologii i Transfuzjologii
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Cardiff, Reino Unido, CF14 4XW
- Cardiff and Vale NHS Trust
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London, Reino Unido, NW3 2QG
- Royal Free Hospital
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London, Reino Unido, WC1N 3HR
- Great Ormond Street Hospital for Children NHS Foundation Trust
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Johannesburg, África do Sul, 2193
- Charlotte Maxeke Johannesburg Hospital
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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
Descrição
Critério de inclusão:
- Diagnóstico de leve (nível de FVIII entre >5% e
- Peso ≥3 quilogramas (kg)
- Necessidade de profilaxia com base na avaliação do investigador
- Um teste negativo para inibidor (ou seja,
- Nenhum inibidor documentado (ou seja,
- Documentação dos detalhes do tratamento profilático ou episódico de FVIII e do número de episódios hemorrágicos durante pelo menos as últimas 24 semanas antes da inscrição
- Função hematológica hepática e renal adequada
- Para mulheres com potencial para engravidar: concordância em permanecer abstinente ou usar contracepção (conforme definido no protocolo) durante o período de tratamento e por pelo menos 24 semanas após a dose final do medicamento do estudo
Critério de exclusão:
- Distúrbio hemorrágico hereditário ou adquirido, exceto hemofilia A congênita leve ou moderada
- Histórico de abuso de drogas ilícitas ou álcool nas 48 semanas anteriores à triagem, a critério do investigador
- Tratamento anterior (nos últimos 12 meses) ou atual para doença tromboembólica ou sinais de doença tromboembólica
- Outras condições que atualmente podem aumentar o risco de sangramento ou trombose
- História de hipersensibilidade clinicamente significativa associada a terapias com anticorpos monoclonais ou componentes da injeção de emicizumabe
- Cirurgia planejada durante a fase de dose de ataque de emicizumabe (são permitidas cirurgias em participantes em uso de emicizumabe a partir da Semana 5)
- Infecção por HIV conhecida com contagem de CD4
- Doença concomitante, condição, anormalidade significativa na avaliação de triagem ou testes laboratoriais, ou tratamento que possa interferir na condução do estudo ou que, na opinião do investigador, represente um risco inaceitável adicional na administração do medicamento do estudo ao participante
- Recebimento de qualquer um dos seguintes: Um medicamento experimental para tratar ou reduzir o risco de sangramentos hemofílicos dentro de 5 meias-vidas da última administração do medicamento, com exceção da profilaxia anterior com emicizumabe; Um medicamento experimental não relacionado à hemofilia nos últimos 30 dias ou 5 meias-vidas, o que for mais curto; ou Qualquer outro medicamento experimental atualmente sendo administrado ou planejado para ser administrado
- Incapacidade de cumprir o protocolo do estudo na opinião do investigador
- Grávida ou amamentando, ou com intenção de engravidar durante o estudo (mulheres com potencial para engravidar devem ter um resultado negativo no teste de gravidez sérica dentro de 7 dias antes do início do medicamento do estudo)
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: Emicizumabe
Os participantes com hemofilia A leve e moderada sem inibidores do fator VIII (FVIII) serão inscritos para receber o regime de dose de carga de emicizumabe seguido pela preferência do participante de um dos 3 regimes de dose de manutenção.
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Quatro doses de ataque de emicizumabe 3 miligramas por quilograma de peso corporal (mg/kg) serão administradas por via subcutânea (SC) uma vez por semana (QW) por 4 semanas, seguidas pela preferência do participante de um dos três seguintes regimes de dose SC de manutenção: 1,5 mg /kg QW, 3 mg/kg uma vez a cada 2 semanas (Q2W), ou 6 mg/kg uma vez a cada 4 semanas (Q4W).
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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Taxa de sangramento anual baseada em modelo para sangramentos tratados
Prazo: Desde o dia da primeira dose de emicizumabe até pelo menos 52 semanas de tratamento com emicizumabe (mediana [faixa, min-max] período de eficácia: 55,64 [8,6-89,9] semanas)
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O número de sangramentos tratados durante o período de eficácia foi estimado como uma taxa de sangramento anualizada (ABR) usando um modelo de regressão binomial negativo, que leva em consideração diferentes tempos de acompanhamento.
Um sangramento tratado foi definido como um sangramento seguido diretamente por um medicamento para hemofilia relatado como um "tratamento para sangramento".
A regra de 72 horas foi implementada: dois sangramentos do mesmo tipo e na mesma localização anatômica foram contados como um sangramento se o segundo sangramento ocorreu dentro de 72 horas a partir do último tratamento para o primeiro sangramento.
Sangramentos decorrentes de cirurgia/procedimento foram excluídos.
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Desde o dia da primeira dose de emicizumabe até pelo menos 52 semanas de tratamento com emicizumabe (mediana [faixa, min-max] período de eficácia: 55,64 [8,6-89,9] semanas)
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Mean Calculated ABR for Treated Bleeds
Prazo: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
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The number of treated bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
A treated bleed was defined as a bleed that was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
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From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
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Median Calculated ABR for Treated Bleeds
Prazo: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
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The number of treated bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
A treated bleed was defined as a bleed that was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
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From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
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Medidas de resultados secundários
Medida de resultado |
Descrição da medida |
Prazo |
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Taxa média de sangramento anual calculada para sangramentos espontâneos tratados
Prazo: Desde o dia da primeira dose de emicizumabe até pelo menos 52 semanas de tratamento com emicizumabe (mediana [faixa, min-max] período de eficácia: 55,64 [8,6-89,9] semanas)
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O número de sangramentos espontâneos tratados durante o período de eficácia é apresentado aqui como uma taxa de sangramento anualizada calculada (ABR) que foi anualizada para cada participante usando a seguinte fórmula: ABR = (número de sangramentos/número de dias durante o período de eficácia) x 365,25 .
Um sangramento espontâneo tratado foi definido como um sangramento tratado (sangramento seguido diretamente por um medicamento para hemofilia relatado como um "tratamento para sangramento") sem nenhum outro fator contribuinte conhecido, como trauma ou procedimento/cirurgia.
A regra de 72 horas foi implementada: dois sangramentos do mesmo tipo e na mesma localização anatômica foram contados como um sangramento se o segundo sangramento ocorreu dentro de 72 horas a partir do último tratamento para o primeiro sangramento.
Sangramentos decorrentes de cirurgia/procedimento foram excluídos.
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Desde o dia da primeira dose de emicizumabe até pelo menos 52 semanas de tratamento com emicizumabe (mediana [faixa, min-max] período de eficácia: 55,64 [8,6-89,9] semanas)
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Taxa de Sangramento Anual Calculada Mediana para Sangramentos Espontâneos Tratados
Prazo: Desde o dia da primeira dose de emicizumabe até pelo menos 52 semanas de tratamento com emicizumabe (mediana [faixa, min-max] período de eficácia: 55,64 [8,6-89,9] semanas)
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O número de sangramentos espontâneos tratados durante o período de eficácia é apresentado aqui como uma taxa de sangramento anualizada calculada (ABR) que foi anualizada para cada participante usando a seguinte fórmula: ABR = (número de sangramentos/número de dias durante o período de eficácia) x 365,25 .
Um sangramento espontâneo tratado foi definido como um sangramento tratado (sangramento seguido diretamente por um medicamento para hemofilia relatado como um "tratamento para sangramento") sem nenhum outro fator contribuinte conhecido, como trauma ou procedimento/cirurgia.
A regra de 72 horas foi implementada: dois sangramentos do mesmo tipo e na mesma localização anatômica foram contados como um sangramento se o segundo sangramento ocorreu dentro de 72 horas a partir do último tratamento para o primeiro sangramento.
Sangramentos decorrentes de cirurgia/procedimento foram excluídos.
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Desde o dia da primeira dose de emicizumabe até pelo menos 52 semanas de tratamento com emicizumabe (mediana [faixa, min-max] período de eficácia: 55,64 [8,6-89,9] semanas)
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Porcentagem de participantes que preferem o tratamento com emicizumabe SC, seu tratamento anterior para hemofilia IV ou não têm preferência, conforme avaliado pelo uso da pesquisa de preferência de emicizumabe na semana 17
Prazo: Semana 17
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A Pesquisa de Preferência de Emicizumabe é um questionário adequado à finalidade desenvolvido pelo patrocinador para registrar a preferência do participante pelo tratamento com emicizumabe subcutâneo (SC), fator VIIII (FVIII) intravenoso (IV) ou nenhuma preferência.
Os intervalos de confiança de 95% foram calculados pelo método de Pearson-Clopper.
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Semana 17
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Porcentagem de cuidadores que preferem o tratamento com emicizumabe SC, o tratamento anterior para hemofilia IV de seus filhos ou não têm preferência, conforme avaliado pelo uso da pesquisa de preferência de emicizumabe na semana 17
Prazo: Semana 17
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A Pesquisa de Preferência de Emicizumabe é um questionário adequado à finalidade desenvolvido pelo patrocinador para registrar a preferência do cuidador pelo tratamento de seu filho com emicizumabe subcutâneo (SC), fator VIIII (FVIII) intravenoso (IV) ou nenhuma preferência.
Os intervalos de confiança de 95% foram calculados pelo método de Pearson-Clopper.
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Semana 17
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Alteração da linha de base na duração média diária da atividade de pico ao longo do tempo
Prazo: Linha de base (semanas 1-2) e semanas 13 (período das semanas 12-13), 25 (período das semanas 24-25), 37 (período das semanas 36-37) e 49 (período das semanas 48-49)
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Para avaliação da atividade física, os participantes ≥5 anos de idade foram instruídos a usar o dispositivo de acelerometria do estudo no pulso continuamente (24 horas/dia) todos os dias durante os períodos designados de 2 semanas durante o estudo.
Um participante foi considerado compatível com as avaliações de atividade física se usasse o dispositivo do estudo continuamente (≥8 horas/dia) todos os dias por pelo menos 8 dias de cada um dos períodos designados de 2 semanas durante o estudo.
Se este critério de conformidade não fosse atingido em um momento específico, o participante não era incluído na análise dos períodos designados de 2 semanas em que a conformidade não era alcançada.
Medições diárias foram calculadas em média ao longo do ponto de tempo de 14 dias.
A contagem de atividade era uma medida da aceleração medida pelo dispositivo.
A duração máxima da atividade diária foi definida como a soma da atividade moderada a vigorosa por dia (em minutos).
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Linha de base (semanas 1-2) e semanas 13 (período das semanas 12-13), 25 (período das semanas 24-25), 37 (período das semanas 36-37) e 49 (período das semanas 48-49)
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Alteração da linha de base na contagem média diária de passos ao longo do tempo
Prazo: Linha de base (semanas 1-2) e semanas 13 (semanas 12-13), 25 (semanas 24-25), 37 (semanas 36-37) e 49 (semanas 48-49)
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Para avaliação da atividade física, os participantes ≥5 anos de idade foram instruídos a usar o dispositivo de acelerometria do estudo no pulso continuamente (24 horas/dia) todos os dias durante os períodos designados de 2 semanas durante o estudo.
Um participante foi considerado compatível com as avaliações de atividade física se usasse o dispositivo do estudo continuamente (≥8 horas/dia) todos os dias por pelo menos 8 dias de cada um dos períodos designados de 2 semanas durante o estudo.
Se este critério de conformidade não fosse atingido em um momento específico, o participante não era incluído na análise dos períodos designados de 2 semanas em que a conformidade não era alcançada.
Medições diárias foram calculadas em média ao longo do ponto de tempo de 14 dias.
A contagem de atividade era uma medida da aceleração medida pelo dispositivo.
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Linha de base (semanas 1-2) e semanas 13 (semanas 12-13), 25 (semanas 24-25), 37 (semanas 36-37) e 49 (semanas 48-49)
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Model-Based ABR for All Bleeds
Prazo: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
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The number of all bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times.
In this outcome measure, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded.
As "all bleeds" comprises both treated and non-treated bleeds, the 72-hour rule was implemented separately for treated and non-treated bleeds.
For treated bleeds, the 72-hour rule meant that two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
For non-treated bleeds, the 72-hour rule was implemented by calculating a treatment-free period of 72 hours from the bleed itself.
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From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
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Mean Calculated ABR for All Bleeds
Prazo: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
|
The number of all bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
In this outcome measure, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded.
As "all bleeds" comprises both treated and non-treated bleeds, the 72-hour rule was implemented separately for treated and non-treated bleeds.
For treated bleeds, the 72-hour rule meant that two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
For non-treated bleeds, the 72-hour rule was implemented by calculating a treatment-free period of 72 hours from the bleed itself.
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From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
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Median Calculated ABR for All Bleeds
Prazo: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
|
The number of all bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
In this outcome measure, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded.
As "all bleeds" comprises both treated and non-treated bleeds, the 72-hour rule was implemented separately for treated and non-treated bleeds.
For treated bleeds, the 72-hour rule meant that two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
For non-treated bleeds, the 72-hour rule was implemented by calculating a treatment-free period of 72 hours from the bleed itself.
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From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
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Model-Based ABR for Treated Joint Bleeds
Prazo: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
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The number of treated joint bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times.
A treated joint bleed was defined as a bleed with type reported as "joint" based on at least one of the following symptoms: increasing swelling or warmth of the skin over the joint and/or increasing pain, decreased range of motion, or difficulty using the joint compared with baseline, and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
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From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
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Mean Calculated ABR for Treated Joint Bleeds
Prazo: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
|
The number of treated joint bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
A treated joint bleed was defined as a bleed with type reported as "joint" based on at least one of the following symptoms: increasing swelling or warmth of the skin over the joint and/or increasing pain, decreased range of motion, or difficulty using the joint compared with baseline, and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
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From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
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Median Calculated ABR for Treated Joint Bleeds
Prazo: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
|
The number of treated joint bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
A treated joint bleed was defined as a bleed with type reported as "joint" based on at least one of the following symptoms: increasing swelling or warmth of the skin over the joint and/or increasing pain, decreased range of motion, or difficulty using the joint compared with baseline, and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
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From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
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Model-Based ABR for Treated Target Joint Bleeds
Prazo: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
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The number of treated target joint bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times.
A treated target joint bleed was defined as a joint bleed in a target joint, which is a joint location where at least 3 bleeds have occurred over the last 24 weeks prior to study entry or an unresolved target joint (target joint that does not fulfil ≤2 bleeds into this joint within a consecutive 12-month period), and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
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From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
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Mean Calculated ABR for Treated Target Joint Bleeds
Prazo: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
|
The number of treated target joint bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
A treated target joint bleed was defined as a joint bleed in a target joint, which is a joint location where at least 3 bleeds have occurred over the last 24 weeks prior to study entry or an unresolved target joint (target joint that does not fulfil ≤2 bleeds into this joint within a consecutive 12-month period), and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
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From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
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Median Calculated ABR for Treated Target Joint Bleeds
Prazo: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
|
The number of treated target joint bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
A treated target joint bleed was defined as a joint bleed in a target joint, which is a joint location where at least 3 bleeds have occurred over the last 24 weeks prior to study entry or an unresolved target joint (target joint that does not fulfil ≤2 bleeds into this joint within a consecutive 12-month period), and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
|
|
Model-Based ABR for Treated Spontaneous Bleeds
Prazo: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
|
The number of treated spontaneous bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times.
A treated spontaneous bleed was defined as a treated bleed (bleed directly followed by a hemophilia medication reported to be a "treatment for bleed") with no other known contributing factor such as trauma or procedure/surgery.
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
|
|
Long-term Efficacy of Emicizumab: Model-Based ABR for Treated Bleeds
Prazo: Median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of treated bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times.
A treated bleed was defined as a bleed that was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
Median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
Long-term Efficacy of Emicizumab: Mean Calculated ABR for Treated Bleeds
Prazo: Median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of treated bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
A treated bleed was defined as a bleed that was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
Median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
Long-term Efficacy of Emicizumab: Median Calculated ABR for Treated Bleeds
Prazo: Median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of treated bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
A treated bleed was defined as a bleed that was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
Median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
Long-term Efficacy of Emicizumab: Model-Based ABR for All Bleeds
Prazo: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of all bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times.
In this outcome measure, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded.
As "all bleeds" comprises both treated and non-treated bleeds, the 72-hour rule was implemented separately for treated and non-treated bleeds.
For treated bleeds, the 72-hour rule meant that two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
For non-treated bleeds, the 72-hour rule was implemented by calculating a treatment-free period of 72 hours from the bleed itself.
|
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
Long-term Efficacy of Emicizumab: Mean Calculated ABR for All Bleeds
Prazo: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of all bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
In this outcome measure, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded.
As "all bleeds" comprises both treated and non-treated bleeds, the 72-hour rule was implemented separately for treated and non-treated bleeds.
For treated bleeds, the 72-hour rule meant that two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
For non-treated bleeds, the 72-hour rule was implemented by calculating a treatment-free period of 72 hours from the bleed itself.
|
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
Long-term Efficacy: Median Calculated ABR for All Bleeds
Prazo: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of all bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
In this outcome measure, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded.
As "all bleeds" comprises both treated and non-treated bleeds, the 72-hour rule was implemented separately for treated and non-treated bleeds.
For treated bleeds, the 72-hour rule meant that two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
For non-treated bleeds, the 72-hour rule was implemented by calculating a treatment-free period of 72 hours from the bleed itself.
|
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
Long-term Efficacy of Emicizumab: Model-Based ABR for Treated Joint Bleeds
Prazo: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of treated joint bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times.
A treated joint bleed was defined as a bleed with type reported as "joint" based on at least one of the following symptoms: increasing swelling or warmth of the skin over the joint and/or increasing pain, decreased range of motion, or difficulty using the joint compared with baseline, and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
Long-term Efficacy of Emicizumab: Mean Calculated ABR for Treated Joint Bleeds
Prazo: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of treated joint bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
A treated joint bleed was defined as a bleed with type reported as "joint" based on at least one of the following symptoms: increasing swelling or warmth of the skin over the joint and/or increasing pain, decreased range of motion, or difficulty using the joint compared with baseline, and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
Long-term Efficacy of Emicizumab: Median Calculated ABR for Treated Joint Bleeds
Prazo: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of treated joint bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
A treated joint bleed was defined as a bleed with type reported as "joint" based on at least one of the following symptoms: increasing swelling or warmth of the skin over the joint and/or increasing pain, decreased range of motion, or difficulty using the joint compared with baseline, and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
Long-term Efficacy of Emicizumab: Model-Based ABR for Treated Target Joint Bleeds
Prazo: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of treated target joint bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times.
A treated target joint bleed was defined as a joint bleed in a target joint, which is a joint location where at least 3 bleeds have occurred over the last 24 weeks prior to study entry or an unresolved target joint (target joint that does not fulfil ≤2 bleeds into this joint within a consecutive 12-month period), and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
Long-term Efficacy of Emicizumab: Mean Calculated ABR for Treated Target Joint Bleeds
Prazo: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of treated target joint bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
A treated target joint bleed was defined as a joint bleed in a target joint, which is a joint location where at least 3 bleeds have occurred over the last 24 weeks prior to study entry or an unresolved target joint (target joint that does not fulfil ≤2 bleeds into this joint within a consecutive 12-month period), and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
Long-term Efficacy of Emicizumab: Median Calculated ABR for Treated Target Joint Bleeds
Prazo: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of treated target joint bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
A treated target joint bleed was defined as a joint bleed in a target joint, which is a joint location where at least 3 bleeds have occurred over the last 24 weeks prior to study entry or an unresolved target joint (target joint that does not fulfil ≤2 bleeds into this joint within a consecutive 12-month period), and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
Long-term Efficacy of Emicizumab: Model-Based ABR for Treated Spontaneous Bleeds
Prazo: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of treated spontaneous bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times.
A treated spontaneous bleed was defined as a treated bleed (bleed directly followed by a hemophilia medication reported to be a "treatment for bleed") with no other known contributing factor such as trauma or procedure/surgery.
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
Long-term Efficacy of Emicizumab: Mean Calculated ABR for Treated Spontaneous Bleeds
Prazo: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of treated spontaneous bleeds over the efficacy period is presented here as a calculated annualized bleed rate (ABR) that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
A treated spontaneous bleed was defined as a treated bleed (bleed directly followed by a hemophilia medication reported to be a "treatment for bleed") with no other known contributing factor such as trauma or procedure/surgery.
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
Long-term Efficacy of Emicizumab: Median Calculated ABR for Treated Spontaneous Bleeds
Prazo: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of treated spontaneous bleeds over the efficacy period is presented here as a calculated annualized bleed rate (ABR) that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
A treated spontaneous bleed was defined as a treated bleed (bleed directly followed by a hemophilia medication reported to be a "treatment for bleed") with no other known contributing factor such as trauma or procedure/surgery.
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
CATCH Questionnaire for Adult Participants: Change From Baseline in the Daily Activity Risk Perception (RP) and Impact Domain Scores Over Time
Prazo: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and Study completion (SC)/Early discontinuation (ED) (up to approximately 274 weeks)
|
Comprehensive Assessment Tool of Challenges in Hemophilia (CATCH) was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥ 18 years).
The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain.
Of this, the daily activity RP and impact domain has 48 items with each item scored on 4 or 5 points ordinal scales.
Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept and applying a linear transformation.
The transformed scores range from 0-100 scale for both the RP and Impact.
Higher scores indicating higher perceived risk of having a bleed while doing daily activities and higher impact of hemophilia on daily activities.
|
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and Study completion (SC)/Early discontinuation (ED) (up to approximately 274 weeks)
|
|
CATCH Questionnaire for Adult Participants: Change From Baseline in the Social Activity RP and Impact Domain Scores Over Time
Prazo: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
|
CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥ 18 years).
The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain.
Of this, the social activity RP and impact domain has 22 items with each item scored on 4 or 5 points ordinal scales.
Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept and applying a linear transformation.
The transformed scores range from 0-100 scale for both the RP and Impact.
Higher scores indicating higher perceived risk of having a bleed while doing social activities and higher impact of hemophilia on social activities.
|
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
|
|
CATCH Questionnaire for Adult Participants: Change From Baseline in the Recreational Activity RP and Impact Domain Scores Over Time
Prazo: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
|
CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥ 18 years).
The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain.
Of this, the recreational activity RP and impact domain has 32 items and a larger bank of additional items with each item scored on 4 or 5 points ordinal scales.
Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept and applying a linear transformation.
The transformed scores range from 0-100 scale for both the RP and Impact.
Higher scores indicating higher perceived risk of having a bleed while doing recreational activities and higher impact of hemophilia on recreational activities.
|
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
|
|
CATCH Questionnaire for Adult Participants: Change From Baseline in the Work Impact (WI) and College Impact (CI) Domains Score Over Time
Prazo: WI: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks); CI: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, and SC/ED (up to approximately 274 weeks)
|
CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥ 18 years).
The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain.
Of this, work and college impact has 10 items and the items were scored on 4 or 5 points ordinal scales.
Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept & applying a linear transformation.
The transformed scores range from 0-100 scale.
Higher scores indicated impact of hemophilia on work and college activities.
|
WI: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks); CI: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, and SC/ED (up to approximately 274 weeks)
|
|
CATCH Questionnaire for Adult Participants: Change From Baseline in the Preoccupation Domain Score Over Time
Prazo: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
|
CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥18 years).
The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain.
Of this, preoccupation has 10 items which were scored on 4 or 5 points ordinal scales.
Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept & applying a linear transformation.
The transformed scores range from 0-100 scale.
Higher scores indicated greater preoccupation related to hemophilia.
|
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
|
|
CATCH Questionnaire for Adult Participants: Change From Baseline in the Treatment Burden Domain Score Over Time
Prazo: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
|
CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥18 years).
The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain.
Of this, treatment burden has 8 items which were scored on 4 or 5 points ordinal scales.
Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept & applying a linear transformation.
The transformed scores range from 0-100 scale.
Higher scores indicated greater perceived burden of the hemophilia treatment.
|
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
|
|
CATCH Questionnaire for Adult Participants: Number of Participants by Responses to Their Level of Pain Associated With a Bleed Over Time
Prazo: Baseline, Weeks 25, 49, 61, 85, 121, 133, 157 and 169 and SC/ED (up to approximately 274 weeks)
|
CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥18 years).
The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain.
Of this, the pain domain consisting of five items, assessed using an 11-point numeric rating scale where 0 = no pain and 10 = pain as bad as you can imagine.
The data reported here are from participants who answered the following question: "Did you get a bleed in the past 7 days?"
If the response was yes, participants were asked to rate their pain by selecting the number that best described the pain associated with the bleed.
Data is reported only for categories/timepoints with non-zero values.
|
Baseline, Weeks 25, 49, 61, 85, 121, 133, 157 and 169 and SC/ED (up to approximately 274 weeks)
|
|
CATCH Questionnaire for Adult Participants: Number of Participants by Responses to Their Level of Pain in Target Joints Over Time
Prazo: Baseline, Weeks 1, 13, 25, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
|
CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥18 years).
The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain.
Of this, the pain domain consists of five items, assessed using an 11-point numeric rating scale where 0 = no pain and 10 = pain as bad as you can imagine.
The data reported here are from participants who answered the following question "Do you have target joints?"
(If yes, please rate your pain by picking the number that best describes your pain in your target joint(s) over the past 7 days).
Data is reported only for categories/timepoints with non-zero values.
|
Baseline, Weeks 1, 13, 25, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
|
|
CATCH Questionnaire for Adult Participants: Number of Participants by Responses to Their Level of Pain at Its Worst Over Time
Prazo: Baseline, Weeks 1, 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
|
CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥18 years).
The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain.
Of this, pain domain consists of five items, assessed using an 11-point numeric rating scale where 0 = no pain and 10 = pain as bad as you can imagine.
The data reported here are from participants who answered the following question "Please rate your pain by picking the number that best describes your pain at its WORST over the past 7 days".
Data is reported only for categories/timepoints with non-zero values.
|
Baseline, Weeks 1, 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
|
|
CATCH Questionnaire for Adult Participants: Number of Participants by Responses to Their Level of Pain at Its Least Over Time
Prazo: Baseline, Weeks 1, 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
|
CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥18 years).
The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain.
Of this, pain domain consists of five items, assessed using an 11-point numeric rating scale where 0 = no pain and 10 = pain as bad as you can imagine.
The data reported here are from participants who answered the following question "Please rate your pain by picking the number that best describes your pain at its LEAST over the past 7 days."
Data is reported only for categories/timepoints with non-zero values.
|
Baseline, Weeks 1, 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
|
|
CATCH Questionnaire for Adult Participants: Number of Participants by Responses to Their Level of Pain at Its Least on the Average Over Time
Prazo: Baseline, Weeks 1, 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
|
CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥18 years).
The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain.
Of this, the pain domain consists of five items, assessed using an 11-point numeric rating scale where 0 = no pain and 10 = pain as bad as you can imagine.
The data reported here are from participants who answered the following question "Please rate your pain by picking the number that best describes your pain at its average over the past 7 days".
Data is reported only for categories/timepoints with non-zero values.
|
Baseline, Weeks 1, 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
|
|
CATCH Questionnaire for Pediatric Participants: Change From Baseline in the Daily Activity RP and Impact Domain Score Over Time
Prazo: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
|
CATCH was used to measure the effect of hemophilia and its treatment on pediatric participants (aged 8-17 years).
The pediatric version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, school impact, preoccupation, treatment burden, and pain).
Of this, daily activity RP and impact has 38 items with each item scored on 3 or 4 points ordinal scale.
Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept and applying a linear transformation.
The transformed scores range from 0-100 scale.
Higher scores indicating higher perceived risk of having a bleed while doing daily activities and higher impact of hemophilia on daily activities.
|
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
|
|
CATCH Questionnaire for Pediatric Participants: Change From Baseline in the Social Activity Risk Perception and Impact Domain Score Over Time
Prazo: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
|
CATCH was used to measure the effect of hemophilia and its treatment on pediatric participants (aged 8-17 years).
The pediatric version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, school impact, preoccupation, treatment burden, and pain).
Of this, social activity RP and impact has 16 items which were scored on 3 or 4 points ordinal scale.
Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept & applying a linear transformation.
The transformed scores range from 0-100 scale.
Higher scores indicated higher perceived risk of having a bleed while doing social activities and higher impact of hemophilia on social activities.
|
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
|
|
CATCH Questionnaire for Pediatric Participants: Change From Baseline in the Recreational Activity RP and Impact Domain Score Over Time
Prazo: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
|
CATCH was used to measure the effect of hemophilia and its treatment on pediatric participants (aged 8-17 years).
The pediatric version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, school impact, preoccupation, treatment burden, and pain.
Of this, recreational activity RP & impact has 34 items + larger bank of additional items.
The items were scored on 3 or 4 points ordinal scales.
Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept & applying a linear transformation.
The transformed scores range from 0-100 scale.
Higher scores indicated higher perceived risk of having a bleed while doing recreational activities and higher impact of hemophilia on recreational activities.
|
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
|
|
CATCH Questionnaire for Pediatric Participants: Change From Baseline in the School Impact Domain Score Over Time
Prazo: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
|
CATCH was used to measure the effect of hemophilia and its treatment on pediatric participants (aged 8-17 years).
The pediatric version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, school impact, preoccupation, treatment burden, and pain.
Of this, school impact has 11 items and the items were scored on 3 or 4 points ordinal scales.
Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept & applying a linear transformation.
The transformed scores range from 0-100 scale.
Higher scores indicated higher impact of hemophilia on school activities.
|
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
|
|
CATCH Questionnaire for Pediatric Participants: Change From Baseline in the Preoccupation Domain Score Over Time
Prazo: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
|
CATCH was used to measure the effect of hemophilia and its treatment on pediatric participants (aged 8-17 years).
The pediatric version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, school impact, preoccupation, treatment burden, and pain.
Of this, preoccupation has 3 items which were scored on 3 or 4 points ordinal scales.
Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept & applying a linear transformation.
The transformed scores range from 0-100 scale.
Higher scores indicated greater preoccupation related to hemophilia.
|
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
|
|
CATCH Questionnaire for Pediatric Participants: Change From Baseline in the Treatment Burden Domain Score Over Time
Prazo: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
|
CATCH was used to measure the effect of hemophilia and its treatment on pediatric participants (aged 8-17 years).
The pediatric version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, school impact, preoccupation, treatment burden, and pain.
Of this, treatment burden has 7 items which were scored on 3 or 4 points ordinal scales.
Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept & applying a linear transformation.
The transformed scores range from 0-100 scale.
Higher scores indicated lower perceived burden of the hemophilia treatment.
|
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
|
|
CATCH Questionnaire for Pediatric Participants: Number of Participants by Responses to Their Level of Hurt Associated With a Bleed Over Time
Prazo: Baseline, Weeks 13, 25, 37, 49, 61, 85, 97, 109, 121, 133, 145 and 157
|
CATCH was used to measure the effect of hemophilia and its treatment on pediatric participants (aged 8-17 years).
The pediatric version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, school impact, preoccupation, treatment burden, and pain).
Of this, pain domain (Hurt) consists of two items, assessed using an 11-point numeric rating scale where 0 = no hurt and 10 = a lot of hurt.
The data reported here are from participants who answered the following question "Did you get a bleed in the past 7 days?
( If yes, how much did the bleed hurt?)."
Data is reported only for categories/timepoints with non-zero values.
|
Baseline, Weeks 13, 25, 37, 49, 61, 85, 97, 109, 121, 133, 145 and 157
|
|
CATCH Questionnaire for Pediatric Participants: Number of Participants by Responses to Their Level of Hurt at Its Worst Over Time
Prazo: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145,157, 169 and SC/ED (up to approximately 274 weeks)
|
CATCH was used to measure the effect of hemophilia and its treatment on pediatric participants (aged 8-17 years).
The pediatric version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, school impact, preoccupation, treatment burden, and pain.
Of this, pain domain (Hurt) consists of two items, assessed using an 11-point numeric rating scale where 0 = no hurt and 10 = a lot of hurt.
The data reported here are from participants who answered the following question "What is the WORST that your body has hurt over the past 7 days".
Data is reported only for categories/timepoints with non-zero values.
|
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145,157, 169 and SC/ED (up to approximately 274 weeks)
|
|
CATCH Questionnaire for Caregivers: Change From Baseline in the Preoccupation Domain Score Over Time
Prazo: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
|
CATCH was used to measure the effect of hemophilia and its treatment on caregivers of pediatric participants (aged <8 years).
The caregiver version has 2 domains (preoccupation and treatment burden).
Of this, preoccupation has 13 items with each of the items were scored on 4 or 5 ordinal scales.
Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept and applying a linear transformation.
The transformed scores range from 0-100 scale.
Higher scores indicated greater preoccupation related to hemophilia.
|
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
|
|
CATCH Questionnaire for Caregivers: Change From Baseline in the Treatment Burden Domain Score Over Time
Prazo: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
|
CATCH was used to measure the effect of hemophilia and its treatment on caregivers of pediatric participants (aged <8 years).
The caregiver version has 2 domains (preoccupation and treatment burden).
Of this, treatment burden has 8 items which were scored on 4 or 5 ordinal scales.
Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept and applying a linear transformation.
The transformed scores range from 0-100 scale.
Higher scores indicated greater perceived burden of the hemophilia treatment.
|
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
|
|
Change From Baseline in Hemophilia Joint Health Scores Over Time
Prazo: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
|
The HJHS measures joint health of the joints (knees, ankles, and elbows) most commonly affected by bleeding in hemophilia.
The HJHS 2.1 provides joint specific total scores which are added to obtain the sum of joints totals and also a global gait score.
These two scores are then added to obtain HJHS total score.
The minimum score per joint is 0, the maximum score is 20.
In addition, Gait is scored on a scale from 0 to 4 based on the number of skills that are not within the normal limits.
The total score is the sum of scores across all six joints plus the gait score (range from 0 to 124, with 0 being normal and 124 being the most severe disease).
A higher score indicates worse joint health.
|
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
|
|
Menstrual Bleed Questionnaire (MBQ) for Female Participants of Childbearing Potential: Change From Baseline in the MBQ Total Score Over Time
Prazo: Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 85, 89, 93, 97, 101, 104, 109, 113, 117, 121, 125, 129, 133, 137, 141 and145
|
The MBQ is a validated measure for menorrhagia and used to assess impact on the menstrual bleed-related heaviness, pain, irregularity, and quality of life (QoL).
The MBQ is a 20-item measure covering four aspects regarding heavy menstrual bleeding: heaviness (8 items), QoL (8 items), irregularity (3 item), and pain (1 item).
The MBQ score ranges from 0 to 75.
Higher scores indicate a worse quality of life and more severe symptoms.
|
Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 85, 89, 93, 97, 101, 104, 109, 113, 117, 121, 125, 129, 133, 137, 141 and145
|
|
MBQ for Female Participants of Childbearing Potential: Change From Baseline in the Heaviness Subscale Score Over Time
Prazo: Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 89, 93, 97, 101, 104, 109, 113, 121, 125, 129, 133 and 137 (up to approximately 274 weeks)
|
The MBQ is a validated measure for menorrhagia and used to assess impact on the menstrual bleed-related heaviness, pain, irregularity, and quality of life (QoL).
The MBQ is a 20-item measure covering four aspects regarding heavy menstrual bleeding: heaviness (8 items), QoL (8 items), irregularity (3 item), and pain (1 item).
The total score for heaviness subscale ranges from 0 to 29.
Higher score indicates more heavy bleeding.
|
Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 89, 93, 97, 101, 104, 109, 113, 121, 125, 129, 133 and 137 (up to approximately 274 weeks)
|
|
MBQ for Female Participants of Childbearing Potential: Change From Baseline in the QoL Subscale Score Over Time
Prazo: Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 85, 89, 93, 97, 101, 104, 109, 113, 117, 121, 125, 129, 133, 137, 141 and 145
|
The MBQ is a validated measure for menorrhagia and used to assess impact on the menstrual bleed-related heaviness, pain, irregularity, and quality of life (QoL).
The MBQ is a 20-item measure covering four aspects regarding heavy menstrual bleeding: heaviness (8 items), QoL (8 items), irregularity (3 item), and pain (1 item).
The total score for QoL subscale ranges from 0 to 37. Higher scores indicate a worse QoL.
|
Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 85, 89, 93, 97, 101, 104, 109, 113, 117, 121, 125, 129, 133, 137, 141 and 145
|
|
MBQ for Female Participants of Childbearing Potential: Change From Baseline in the Irregularity Subscale Score Over Time
Prazo: Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 85, 89, 93, 97, 101, 104, 109, 113, 117, 121, 125, 129, 133, 137, 141 and 145
|
The MBQ is a validated measure for menorrhagia and used to assess impact on the menstrual bleed-related heaviness, pain, irregularity, and quality of life (QoL).
The MBQ is a 20-item measure covering four aspects regarding heavy menstrual bleeding: heaviness (8 items), QoL (8 items), irregularity (3 item), and pain (1 item).
The total score for irregularity subscale ranges from 0 to 6. Higher score indicates more irregularity.
|
Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 85, 89, 93, 97, 101, 104, 109, 113, 117, 121, 125, 129, 133, 137, 141 and 145
|
|
MBQ for Female Participants of Childbearing Potential: Change From Baseline in the Pain Subscale Score Over Time
Prazo: Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 89, 93, 97, 101, 104, 109, 113, 121, 125, 129, 133 and 137
|
The MBQ is a validated measure for menorrhagia and used to assess impact on the menstrual bleed-related heaviness, pain, irregularity, and quality of life (QoL).
The MBQ is a 20-item measure covering four aspects regarding heavy menstrual bleeding: heaviness (8 items), QoL (8 items), irregularity (3 item), and pain (1 item).
The total score for pain subscale ranges from 0 to 3. Higher score indicates severe pain.
|
Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 89, 93, 97, 101, 104, 109, 113, 121, 125, 129, 133 and 137
|
|
Menstruation Diary With the Pictorial Blood Assessment Chart (PBAC) for Female Participants of Childbearing Potential: PBAC Scores Over Time
Prazo: Baseline and monthly (on days of menstruation) until Study Completion (up to approximately 247 weeks)
|
The Menstruation Diary used a PBAC, which has shown good correlation with menstrual blood loss.
The PBAC is a chart that records the occurrence and size of clots, and the number of episodes of heavy bleeding (flooding), as well as depicts the amount of blood loss during a cycle.The PBAC records pad and tampon use (as either light [1 point], medium [5 points], or heavy [10 points] flow), clots (small [1 point] or large [5 points]), and flooding episodes (1 point each) which can be recorded as many times as necessary any day of the month.
The PBAC is scored from 0 (no bleeding) onwards, with a score of >100 defining abnormal coagulation and heavy menstrual bleeding (corresponds to >80ml of blood loss per menstrual cycle).
|
Baseline and monthly (on days of menstruation) until Study Completion (up to approximately 247 weeks)
|
|
Number of Participants With at Least One Adverse Event by Severity, According to the World Health Organization (WHO) Toxicity Grading Scale
Prazo: From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
|
An AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment.
An AE can therefore be any unfavorable and unintended sign (including abnormal laboratory values or abnormal clinical test results), symptoms, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product.
As per WHO toxicity grading, Grade 1= mild, 2= moderate, 3=severe, 4=life threatening.
|
From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
|
|
Number of Participants With Adverse Events Leading to Study Drug Discontinuation
Prazo: From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
|
An AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment.
An AE can therefore be any unfavorable and unintended sign (including abnormal laboratory values or abnormal clinical test results), symptoms, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product.
|
From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
|
|
Number of Participants With at Least One Thromboembolic Event
Prazo: From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
|
An AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment.
An AE can therefore be any unfavorable and unintended sign (including abnormal laboratory values or abnormal clinical test results), symptoms, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product.
The following signs and symptoms were recognized as potential thromboembolism (i.e., dyspnea, chest pain, leg pain, or swelling; or if in the head, headache, numbness in the face, eye pain or swelling, or vision impairment).
|
From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
|
|
Number of Participants With at Least One Event of Thrombotic Microangiopathy
Prazo: From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
|
An AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment.
An AE can therefore be any unfavorable and unintended sign (including abnormal laboratory values or abnormal clinical test results), symptoms, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product.
TMA is used to describe a group of disorders with clinical features of microangiopathic hemolytic anemia, thrombocytopenia, and organ damage that can include the kidneys, gastrointestinal system, central nervous system, etc.
|
From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
|
|
Number of Participants With at Least One Injection-Site Reaction by Severity, According to the WHO Toxicity Grading Scale
Prazo: From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
|
An AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment.
An AE can therefore be any unfavorable and unintended sign (including abnormal laboratory values or abnormal clinical test results), symptoms, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product.
Injection-site reactions are defined as AEs related that occur within 24 hours after study drug administration and are judged to be related to the study drug injection.
Local injection-site reactions included erythema, hematoma, rash, discomfort, pain, and pruritus and were mostly of mild and moderate intensity.
As per WHO toxicity grading, Grade 1= mild, 2= moderate, 3=severe, 4=life threatening.
|
From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
|
|
Number of Participants With at Least One Severe Hypersensitivity, Anaphylaxis, and Anaphylactoid Event
Prazo: From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
|
An AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment.
An AE can therefore be any unfavorable and unintended sign (including abnormal laboratory values or abnormal clinical test results), symptoms, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product.
|
From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
|
|
Number of Participants With at Least One Shifts in Clinical Laboratory Parameters From Baseline WHO Toxicity Scale Grade 0-2 to Post-baseline Grade 3 or 4
Prazo: Up to approximately 274 weeks
|
An abnormal laboratory value was defined as a laboratory test result outside of the normal range for hematology or serum chemistry parameters.
The WHO toxicity grading scale, which ranges from Grades 1 to 4 (least severe to most severe, respectively; Grade 0 is within normal range), was used for assessing the severity of laboratory abnormalities and adverse events (WHO 2003).
Not every laboratory abnormality qualified as an adverse event; an abnormality was reported as an adverse event if it met any of the following criteria: was accompanied by clinical symptoms; resulted in a change in study treatment (e.g., dosage modification, treatment interruption or discontinuation); resulted in a medical intervention or a change in concomitant therapy; or was clinically significant in the investigator's judgment.
|
Up to approximately 274 weeks
|
|
Number of Participants With at Least One Adverse Events of Changes From Baseline in Vital Signs
Prazo: Up to approximately 274 weeks
|
The number of participants with adverse events of changes from baseline in vital signs is reported here.
Vital signs measurements consisted of heart and respiratory rate, temperature, and systolic and diastolic blood pressures, with an abnormal vital sign value being outside of the normal range.
An abnormal vital sign result was reported as an adverse event if it met any of the following criteria: was accompanied by clinical symptoms; resulted in a change in study treatment (e.g., dosage modification, treatment interruption or discontinuation); resulted in a medical intervention or a change in concomitant therapy; or was clinically significant in the investigator's judgment.
All of the adverse events reported here were assessed independently by the investigator as not related to treatment with emicizumab.
|
Up to approximately 274 weeks
|
|
Change From Baseline in Electrocardiogram (ECG) Parameters Over Time: QT, QTcB, QTcF, RR, PR, and QRS Intervals
Prazo: Baseline, Weeks 5, 25, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181, SC/ED (up to approximately 274 weeks)
|
Baseline, Weeks 5, 25, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181, SC/ED (up to approximately 274 weeks)
|
|
|
Change From Baseline in Heart Rate Over Time, as Measured by ECG
Prazo: Baseline, Weeks 5, 25, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181, SC/ED (up to approximately 274 weeks)
|
Baseline, Weeks 5, 25, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181, SC/ED (up to approximately 274 weeks)
|
|
|
Plasma Trough Concentration (Ctrough) of Emicizumab Over Time
Prazo: Pre-dose at Weeks 1, 2, 3, 4, 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169 and 181 (up to approximately 274 weeks)
|
Pre-dose at Weeks 1, 2, 3, 4, 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169 and 181 (up to approximately 274 weeks)
|
|
|
Number of Participants With Anti-Drug Antibodies Against Emicizumab at Baseline and Post-Baseline
Prazo: Baseline, Weeks 5, 13, 25, 33, 41, 49, 61, 73, 85, 97, 109, 121, 133, 145, 169, 181 and SC/ED (up to approximately 274 weeks)
|
Participants were considered to be ADA positive if they were ADA negative at baseline but develop an ADA response following study drug administration (treatment-induced ADA response), or if they were ADA positive at baseline and the titer of one or more post-baseline samples is at least 4-fold greater than the titer of the baseline sample (treatment-enhanced ADA response).
The sum of all visits has been reported here.
|
Baseline, Weeks 5, 13, 25, 33, 41, 49, 61, 73, 85, 97, 109, 121, 133, 145, 169, 181 and SC/ED (up to approximately 274 weeks)
|
|
Number of Participants Who Develop Anti-FVIII Inhibitors Over Time
Prazo: Up to approximately 274 weeks
|
Central laboratory measurement of anti-FVIII inhibitory antibodies (inhibitors) was performed using a Chromogenic Bethesda Assay (CBA).
Participants who develop anti-FVIII inhibitors (titer ≥ 0.6 Bethesda units per milliliter [BU/mL]) following study drug administration were summarized.
|
Up to approximately 274 weeks
|
Colaboradores e Investigadores
É aqui que você encontrará pessoas e organizações envolvidas com este estudo.
Patrocinador
Investigadores
- Diretor de estudo: Clinical Trials, Hoffmann-La Roche
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.
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 2020
Conclusão Primária (Real)
30 de outubro de 2021
Conclusão do estudo (Real)
19 de dezembro de 2025
Datas de inscrição no estudo
Enviado pela primeira vez
7 de novembro de 2019
Enviado pela primeira vez que atendeu aos critérios de CQ
7 de novembro de 2019
Primeira postagem (Real)
12 de novembro de 2019
Atualizações de registro de estudo
Última Atualização Postada (Real)
27 de agosto de 2026
Última atualização enviada que atendeu aos critérios de controle de qualidade
5 de agosto de 2026
Última verificação
1 de agosto de 2026
Mais Informações
Termos relacionados a este estudo
Palavras-chave
Termos MeSH relevantes adicionais
- Doenças Genéticas, Congênitas
- Doenças Hematológicas
- Distúrbios da Coagulação Sanguínea
- Distúrbios hemorrágicos
- Distúrbios da Coagulação Sanguínea, Herdados
- Distúrbios da Proteína de Coagulação
- Doenças e Anormalidades Congênitas, Hereditárias e Neonatais
- Doenças hemic e linfáticas
- Hemofilia A
- emicizumab
Outros números de identificação do estudo
- BO41423
- 2019 (Concessão/Contrato do NIH dos EUA: Chief Medical Office (CMO) Alberta Health Services)
- 2023 (Concessão/Contrato do NIH dos EUA: GRAMMY Museum Foundation)
- 2019-002179-32 (Número EudraCT)
- 2023-506610-52-00 (Identificador de registro: EU CT Number)
Plano para dados de participantes individuais (IPD)
Planeja compartilhar dados de participantes individuais (IPD)?
SIM
Descrição do plano IPD
Pesquisadores qualificados podem solicitar acesso a dados individuais do paciente por meio da plataforma de solicitação (www.vivli.org). Mais detalhes sobre os critérios da Roche para estudos elegíveis estão disponíveis aqui (https://vivli.org/ourmember/roche/).
Para obter mais detalhes sobre a Política Global da Roche sobre Compartilhamento de Informações de Estudos Clínicos e como solicitar acesso a documentos de estudos clínicos relacionados, consulte aqui (https://www.roche.com/research_and_development/who_we_are_how_we_work/clinical_trials/our_commitment_to_data_sharing.htm).
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 .