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.
Descripción general del estudio
Tipo de estudio
Inscripción (Estimado)
Contactos y Ubicaciones
Estudio Contacto
- Nombre: Pfizer CT.gov Call Center
- Número de teléfono: 1-800-718-1021
- Correo electrónico: ClinicalTrials.gov_Inquiries@pfizer.com
Criterios de participación
Criterio de elegibilidad
Edades elegibles para estudiar
Acepta Voluntarios Saludables
Géneros elegibles para el estudio
Método de muestreo
Población de estudio
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
Descripción
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
Plan de estudios
¿Cómo está diseñado el estudio?
Detalles de diseño
- Modelos observacionales: COHORT
- Perspectivas temporales: CROSS_SECTIONAL
Cohortes e Intervenciones
Grupo / Cohorte |
Intervención / Tratamiento |
|---|---|
|
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 |
¿Qué mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Periodo de tiempo |
|---|---|
|
Patient reported responses to Marstacimab Patient Preference Survey (MPPS)
Periodo de tiempo: Six months after initiating marstacimab
|
Six months after initiating marstacimab
|
Medidas de resultado secundarias
Medida de resultado |
Periodo de tiempo |
|---|---|
|
Patient reported responses to Hemophilia Treatment Experience Measure (Hemo-TEM)
Periodo de tiempo: Month 6 and month 12
|
Month 6 and month 12
|
|
Annualized bleed rates (ABRs)
Periodo de tiempo: Baseline through 12 months
|
Baseline through 12 months
|
|
ABRs of all bleeding events
Periodo de tiempo: 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)
Periodo de tiempo: Baseline to month 3
|
Baseline to month 3
|
Colaboradores e Investigadores
Patrocinador
Investigadores
- Director de estudio: Pfizer CT.gov Call Center, Pfizer
Publicaciones y enlaces útiles
Fechas de registro del estudio
Fechas importantes del estudio
Inicio del estudio (Estimado)
Finalización primaria (Estimado)
Finalización del estudio (Estimado)
Fechas de registro del estudio
Enviado por primera vez
Primero enviado que cumplió con los criterios de control de calidad
Publicado por primera vez (Actual)
Actualizaciones de registros de estudio
Última actualización publicada (Actual)
Última actualización enviada que cumplió con los criterios de control de calidad
Última verificación
Más información
Términos relacionados con este estudio
Palabras clave
Términos MeSH relevantes adicionales
- Enfermedades Genéticas Congénitas
- Enfermedades hematológicas
- Trastornos de la coagulación de la sangre
- Trastornos hemorrágicos
- Enfermedades Genéticas, Ligadas al X
- Trastornos de la coagulación de la sangre, hereditarios
- Trastornos de proteínas de coagulación
- Enfermedades y anomalías congénitas, hereditarias y neonatales
- Enfermedades hemic y linfáticas
- Hemofilia A
- Hemofilia B
- marstacimab
Otros números de identificación del estudio
- B7841028
Plan de datos de participantes individuales (IPD)
¿Planea compartir datos de participantes individuales (IPD)?
Descripción del plan 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.
Información sobre medicamentos y dispositivos, documentos del estudio
Estudia un producto farmacéutico regulado por la FDA de EE. UU.
producto fabricado y exportado desde los EE. UU.
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