Observational study evaluating long-term safety of real-world treatment with damoctocog alfa pegol in previously treated patients with hemophilia A (HA-SAFE)

Study type

• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine:
  JIVI
• Medical condition to be studied:
  Haemophilia A without inhibitors

Population studied

• Age groups:
  Adolescents (12 to < 18 years)

  Adults (18 to < 46 years)

  Adults (46 to < 65 years)

  Adults (65 to < 75 years)

  Adults (75 to < 85 years)

  Adults (85 years and over)
• Estimated number of subjects:
  50

Study design details

• Main study objective:
  To assess the long-term safety of prophylaxis with damoctocog alfa pegol in patients with hemophilia A in the real-world setting through the collection and analysis of adverse events (AEs) of special interest including those potentially indicative of PEG accumulation), AEs, serious adverse events (SAEs), and adverse reactions (ARs).
• Outcomes:
  1. Number of subjects with safety events 2. Duration of safety events 3. Number of subjects with safety events leading to a change of treatment 4. Number of subjects with safety events per intensity 5. Number of subjects with safety events with outcome of death 6. Number of subjects with safety events related to inhibitor development, 1.Number of adverse reactions (ARs) that are defined within the system organ classes nervous system and psychiatric disorders 2.Number of ARs related to hepatic or renal function 3.Change from baseline in creatinine, eGFR, ALT, AST, bilirubin 4.Testing for PEG plasma levels (baseline and end of study, if collected) 5.Number of patients with abnormal findings as assessed by neurological examination
• Data analysis plan:
  Statistical analyses will be of an explorative and descriptive nature. The study is not aimed to confirm or reject predefined hypotheses, hence no formal hypothesis testing will be performed.