A Post-Authorisation Safety Study to Evaluate the Safety of Marstacimab Among Patients with Haemophilia A or B using Real-World Data in Haemophilia Registers

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Disease epidemiology

  Safety study (incl. comparative)
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Medicinal product name:
  HYMPAVZI
• Study drug International non-proprietary name (INN) or common name:
  MARSTACIMAB
• Anatomical Therapeutic Chemical (ATC) code:
  (B02BX11) marstacimab

  marstacimab

Population studied

• Short description of the study population:
  Two patient cohorts will be included in the study:; - Patients 12 years of age and older with haemophilia A or B with or without inhibitors who have been treated with marstacimab in a routine care setting after marstacimab has become commercially available in the UK and the US.; - Patients 12 years of age and older with haemophilia A or B with or without inhibitors who are unexposed to marstacimab and are receiving routine prophylaxis in a routine care setting.; ; Patients in the unexposed cohort will be matched within their originating register in a 1:1 ratio to the marstacimab exposure cohort by age group, index date, haemophilia severity, inhibitor status, history of TE, and history of cardiovascular disease.; ; Inclusion Criteria:; The study inclusion criteria are specific for each cohort, as indicated below. The inclusion and exclusion criteria are subject to amendments, and final criteria will be based on the approved local labels. These eligibility criteria are intended to make the two cohorts as similar as possible. ; Patients must meet all the following inclusion criteria specific to the applicable cohort to be eligible for inclusion in the study.; Marstacimab Exposure Cohort:; - Patients 12 years of age and older with haemophilia A or B with or without inhibitors who have been treated with commercial marstacimab in a routine care setting in the UK or the US.; Unexposed Cohort:; - Patients 12 years of age and older with haemophilia A or B with or without inhibitors who have not been treated with marstacimab and are receiving routine prophylaxis in a routine care setting in the UK or the US.; Exclusion Criteria:; There are no exclusion criteria.
• Age groups:
  • Adolescents (12 to < 18 years)
  • Adult and elderly population (≥18 years)

    • Adults (18 to < 65 years)

      • Adults (18 to < 46 years)
      • Adults (46 to < 65 years)
    • Elderly (≥ 65 years)

      • Adults (65 to < 75 years)
      • Adults (75 to < 85 years)
      • Adults (85 years and over)
• Estimated number of subjects:
  220

Study design details

• Study design:
  This is a multi-country, non-interventional, prospective cohort study to evaluate the incidence of TEs in patients with haemophilia A or B with or without inhibitors treated with marstacimab in real-world settings in the UK and the US.
• Main study objective:
  To describe the incidence rates of TEs among patients with haemophilia A or B with or without inhibitors in the patient cohort treated with marstacimab during routine clinical care.
• Setting:
  Routine clinical care in real-world settings in the UK and the US.
• Outcomes:
  New diagnoses of selected medical conditions; TEs (deep vein thrombosis, myocardial infarction, pulmonary embolism, stroke, angina, thrombotic microangiopathy, pulmonary infarction); All Serious Adverse Events; All-Cause Mortality, including cause and date of death when available; - Intracranial haemorrhage, Bleeding (excluding intracranial); - Thromboembolic event; - Liver disease, specify; - Cancer, specify; - Cardiac; - Infection, including pneumonia; - HIV ; - Other, specify.
• Data analysis plan:
  This is a descriptive study with a focus on estimation and not hypothesis testing. Descriptive statistics will be presented for each cohort. For categorical variables, the frequency and percentage will be reported. For continuous variables, the number of patients with available data, mean, median, standard deviation, range, minimum, and maximum values will be reported. In addition, the number of patients with missing data will be summarized for each variable. No imputation of missing values will be performed, except in the case of partial dates which will be outlined in the SAP. Patients who switch between treatments during the study will contribute patient years to each cohort as applicable. See Protocol for additional details