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.
Studieoversikt
Studietype
Registrering (Antatt)
Kontakter og plasseringer
Studiekontakt
- Navn: Pfizer CT.gov Call Center
- Telefonnummer: 1-800-718-1021
- E-post: ClinicalTrials.gov_Inquiries@pfizer.com
Deltakelseskriterier
Kvalifikasjonskriterier
Alder som er kvalifisert for studier
Tar imot friske frivillige
Kjønn som er kvalifisert for studier
Prøvetakingsmetode
Studiepopulasjon
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
Beskrivelse
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
Studieplan
Hvordan er studiet utformet?
Designdetaljer
- Observasjonsmodeller: COHORT
- Tidsperspektiver: CROSS_SECTIONAL
Kohorter og intervensjoner
Gruppe / Kohort |
Intervensjon / Behandling |
|---|---|
|
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 |
Hva måler studien?
Primære resultatmål
Resultatmål |
Tidsramme |
|---|---|
|
Patient reported responses to Marstacimab Patient Preference Survey (MPPS)
Tidsramme: Six months after initiating marstacimab
|
Six months after initiating marstacimab
|
Sekundære resultatmål
Resultatmål |
Tidsramme |
|---|---|
|
Patient reported responses to Hemophilia Treatment Experience Measure (Hemo-TEM)
Tidsramme: Month 6 and month 12
|
Month 6 and month 12
|
|
Annualized bleed rates (ABRs)
Tidsramme: Baseline through 12 months
|
Baseline through 12 months
|
|
ABRs of all bleeding events
Tidsramme: 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)
Tidsramme: Baseline to month 3
|
Baseline to month 3
|
Samarbeidspartnere og etterforskere
Sponsor
Etterforskere
- Studieleder: Pfizer CT.gov Call Center, Pfizer
Publikasjoner og nyttige lenker
Studierekorddatoer
Studer hoveddatoer
Studiestart (Antatt)
Primær fullføring (Antatt)
Studiet fullført (Antatt)
Datoer for studieregistrering
Først innsendt
Først innsendt som oppfylte QC-kriteriene
Først lagt ut (Faktiske)
Oppdateringer av studieposter
Sist oppdatering lagt ut (Faktiske)
Siste oppdatering sendt inn som oppfylte QC-kriteriene
Sist bekreftet
Mer informasjon
Begreper knyttet til denne studien
Ytterligere relevante MeSH-vilkår
- Genetiske sykdommer, medfødte
- Hematologiske sykdommer
- Blodkoagulasjonsforstyrrelser
- Hemoragiske lidelser
- Genetiske sykdommer, X-linked
- Blodkoagulasjonsforstyrrelser, arvelig
- Koagulasjonsproteinforstyrrelser
- Medfødte, arvelige og neonatale sykdommer og abnormiteter
- Hemic og lymfatiske sykdommer
- Hemofili A
- Hemofili B
- Marstacimab
Andre studie-ID-numre
- B7841028
Plan for individuelle deltakerdata (IPD)
Planlegger du å dele individuelle deltakerdata (IPD)?
IPD-planbeskrivelse
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.
Legemiddel- og utstyrsinformasjon, studiedokumenter
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