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.
Aperçu de l'étude
Statut
Les conditions
Intervention / Traitement
Type d'étude
Inscription (Estimé)
Contacts et emplacements
Coordonnées de l'étude
- Nom: Pfizer CT.gov Call Center
- Numéro de téléphone: 1-800-718-1021
- E-mail: ClinicalTrials.gov_Inquiries@pfizer.com
Critères de participation
Critère d'éligibilité
Âges éligibles pour étudier
Accepte les volontaires sains
Sexes éligibles pour l'étude
Méthode d'échantillonnage
Population étudiée
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
La description
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 d'étude
Comment l'étude est-elle conçue ?
Détails de conception
- Modèles d'observation: COHORT
- Perspectives temporelles: CROSS_SECTIONAL
Cohortes et interventions
Groupe / Cohorte |
Intervention / Traitement |
|---|---|
|
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 |
Que mesure l'étude ?
Principaux critères de jugement
Mesure des résultats |
Délai |
|---|---|
|
Patient reported responses to Marstacimab Patient Preference Survey (MPPS)
Délai: Six months after initiating marstacimab
|
Six months after initiating marstacimab
|
Mesures de résultats secondaires
Mesure des résultats |
Délai |
|---|---|
|
Patient reported responses to Hemophilia Treatment Experience Measure (Hemo-TEM)
Délai: Month 6 and month 12
|
Month 6 and month 12
|
|
Annualized bleed rates (ABRs)
Délai: Baseline through 12 months
|
Baseline through 12 months
|
|
ABRs of all bleeding events
Délai: 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)
Délai: Baseline to month 3
|
Baseline to month 3
|
Collaborateurs et enquêteurs
Parrainer
Les enquêteurs
- Directeur d'études: Pfizer CT.gov Call Center, Pfizer
Publications et liens utiles
Dates d'enregistrement des études
Dates principales de l'étude
Début de l'étude (Estimé)
Achèvement primaire (Estimé)
Achèvement de l'étude (Estimé)
Dates d'inscription aux études
Première soumission
Première soumission répondant aux critères de contrôle qualité
Première publication (Réel)
Mises à jour des dossiers d'étude
Dernière mise à jour publiée (Réel)
Dernière mise à jour soumise répondant aux critères de contrôle qualité
Dernière vérification
Plus d'information
Termes liés à cette étude
Termes MeSH pertinents supplémentaires
- Maladies génétiques, innées
- Maladies hématologiques
- Troubles de la coagulation sanguine
- Troubles hémorragiques
- Maladies génétiques liées à l'X
- Troubles de la coagulation sanguine, héréditaires
- Troubles des protéines de la coagulation
- Maladies et anomalies congénitales, héréditaires et néonatales
- Maladies hémiques et lymphatiques
- Hémophilie A
- Hémophilie B
- marstacimab
Autres numéros d'identification d'étude
- B7841028
Plan pour les données individuelles des participants (IPD)
Prévoyez-vous de partager les données individuelles des participants (DPI) ?
Description du régime 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.
Informations sur les médicaments et les dispositifs, documents d'étude
Étudie un produit pharmaceutique réglementé par la FDA américaine
produit fabriqué et exporté des États-Unis.
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