A Study to Learn About the Study Medicine (Called Marstacimab) in People With Hemophilia A or B
ConditionHemophilia
Trial statusNot yet recruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age6+
SponsorPfizer
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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
Disqualifiers
None
Trial population
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
Trial design
Design model
Cohort
Time perspective
Cross-sectional
Treatments tested in this trial
Marstacimab
DrugAs provided in real world practice
Treatment groups
100 Participants
are divided into 1 treatment groupGroup A: Real World Data participants1 intervention
Trial outcomes
Primary outcomes
1
Patient reported responses to Marstacimab Patient Preference Survey (MPPS)
Time frame
Six months after initiating marstacimabSecondary outcomes
1
Patient reported responses to Hemophilia Treatment Experience Measure (Hemo-TEM)
Time frame
Month 6 and month 122
Annualized bleed rates (ABRs)
Time frame
Baseline through 12 months3
ABRs of all bleeding events
Time frame
Baseline through 36 months of martstacimab treatment4
Reponses to the hemophilia prophylaxis experience survey, second version (HPES-II)
Time frame
Baseline to month 3Locations
Source ClinicalTrials.gov
This trial is not recruiting at the moment. You can still explore other options: