A Study to Learn About the Study Medicine (Called Marstacimab) in People With Hemophilia A or B
MARSTACIMAB REAL-WORLD EVALUATION OF EFFECTIVENESS IN HEMOPHILIA: A UNITED STATES MEDICAL RECORD AND PARTICIPANT REPORTED OUTCOME STUDY (MARVL-US)
The purpose of this non-interventional, observational, real-world study is to find out what are the real-world experiences of people living with hemophilia A or B[OK1.1] who start the study medicine (called marstacimab) in routine clinical practice. Hemophilia is a rare genetic condition where the blood does not clot normally, meaning people bleed longer than usual after an injury.
This study is seeking participants who are living with hemophilia A or B and:
- Have a medical record documenting hemophilia A or hemophilia B. A hematologist must have confirmed the diagnosis before they join the study.
- Are under current treatment with marstacimab or have a prescription to start the treatment with marstacimab
- Are 6 years of age or older and the recipient of medical care in the United States All participants in this study will receive their usual treatment for hemophilia A or hemophilia B Participants will take part in this study for 1 year. During this time, they will complete up to six surveys. These surveys will be given between enrollment and about one year after marstacimab is started.
Some participants in the study may be asked to take part in interviews. During these interviews they will be asked about their experience with hemophilia or marstacimab.
The study will provide information about how well the marstacimab treatment works. It will also assess treatment burden, and treatment preferences. This information will help to determine its benefit compared to existing treatment options. It will also help doctors make treatment decisions and optimize hemophilia care.
Visão geral do estudo
Status
Condições
Intervenção / Tratamento
Tipo de estudo
Inscrição (Estimado)
Contactos e Locais
Contato de estudo
- Nome: Pfizer CT.gov Call Center
- Número de telefone: 1-800-718-1021
- E-mail: ClinicalTrials.gov_Inquiries@pfizer.com
Critérios de participação
Critérios de elegibilidade
Idades elegíveis para estudo
Aceita Voluntários Saudáveis
Gêneros Elegíveis para o Estudo
Método de amostragem
População do estudo
This observational study is designed as a virtual-site study to take place in the United States (US), and will collect real-world data from participants' documented routine interactions with the healthcare system, surveys, and interviews. Participants will be recruited from US-based healthcare sites that care for people living with hemophilia, or through digital outreach
Descrição
Medical record documentation of hematologist-confirmed diagnosis of hereditary factor VIII deficiency disease (Hemophilia A) or hereditary factor IX deficiency disease (Hemophilia B) prior to enrollment
- Current treatment with marstacimab or documentation of a prescription to initiate marstacimab
- Aged 6 or older at the time of consenting into the study
- Receipt of medical care in the United States
- Evidence of a personally signed and dated informed consent document indicating that the participant (or a legally authorized representative) has been informed of all pertinent aspects of the study ○ If the participant is between the ages of 7 and 17, evidence of a personally signed and dated informed assent indicating that the participant has been informed of and assents to the study
Plano de estudo
Como o estudo é projetado?
Detalhes do projeto
- Modelos de observação: COHORT
- Perspectivas de Tempo: CROSS_SECTIONAL
Coortes e Intervenções
Grupo / Coorte |
Intervenção / Tratamento |
|---|---|
|
Real World Data participants
This observational study is designed as a virtual-site study to take place in the United States (US), and will collect real-world data from participants' documented routine interactions with the healthcare system, surveys, and interviews. Participants will be recruited from US-based healthcare sites that care for people living with hemophilia, or through digital outreach |
As provided in real world practice |
O que o estudo está medindo?
Medidas de resultados primários
Medida de resultado |
Prazo |
|---|---|
|
Patient reported responses to Marstacimab Patient Preference Survey (MPPS)
Prazo: Six months after initiating marstacimab
|
Six months after initiating marstacimab
|
Medidas de resultados secundários
Medida de resultado |
Prazo |
|---|---|
|
Patient reported responses to Hemophilia Treatment Experience Measure (Hemo-TEM)
Prazo: Month 6 and month 12
|
Month 6 and month 12
|
|
Annualized bleed rates (ABRs)
Prazo: Baseline through 12 months
|
Baseline through 12 months
|
|
ABRs of all bleeding events
Prazo: Baseline through 36 months of martstacimab treatment
|
Baseline through 36 months of martstacimab treatment
|
|
Reponses to the hemophilia prophylaxis experience survey, second version (HPES-II)
Prazo: Baseline to month 3
|
Baseline to month 3
|
Colaboradores e Investigadores
Patrocinador
Investigadores
- Diretor de estudo: Pfizer CT.gov Call Center, Pfizer
Publicações e links úteis
Datas de registro do estudo
Datas Principais do Estudo
Início do estudo (Estimado)
Conclusão Primária (Estimado)
Conclusão do estudo (Estimado)
Datas de inscrição no estudo
Enviado pela primeira vez
Enviado pela primeira vez que atendeu aos critérios de CQ
Primeira postagem (Real)
Atualizações de registro de estudo
Última Atualização Postada (Real)
Última atualização enviada que atendeu aos critérios de controle de qualidade
Última verificação
Mais Informações
Termos relacionados a este estudo
Palavras-chave
Termos MeSH relevantes adicionais
- Doenças Genéticas, Congênitas
- Doenças Hematológicas
- Distúrbios da Coagulação Sanguínea
- Distúrbios hemorrágicos
- Doenças Genéticas, Ligadas ao X
- Distúrbios da Coagulação Sanguínea, Herdados
- Distúrbios da Proteína de Coagulação
- Doenças e Anormalidades Congênitas, Hereditárias e Neonatais
- Doenças hemic e linfáticas
- Hemofilia A
- Hemofilia B
- Marstacimab
Outros números de identificação do estudo
- B7841028
Plano para dados de participantes individuais (IPD)
Planeja compartilhar dados de participantes individuais (IPD)?
Descrição do plano IPD
Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.
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