An observational study to evaluate the long-term effects of evinacumab treatment in patients with homozygous familial hypercholesterolemia (HoFH)

Study type

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

  Disease epidemiology

  Effectiveness study (incl. comparative)

Non-interventional study

• Non-interventional study design:
  Cohort

  Other
• Non-interventional study design, other:
  Registry study of HoFH patients treated with evinacumab in real world setting

Study drug and medical condition

• Study drug International non-proprietary name (INN) or common name:
  EVINACUMAB
• Anatomical Therapeutic Chemical (ATC) code:
  (C10AX17) evinacumab

  evinacumab
• Medical condition to be studied:
  Type IIa hyperlipidaemia

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)
• Special population of interest:
  Pregnant women
• Estimated number of subjects:
  50

Study design details

• Main study objective:
  To evaluate the safety, disease progression, and pregnancy outcomes in participants with HoFH who are treated with evinacumab.
• Outcomes:
  The study will evaluate long-term safety outcomes, the frequency and outcomes of pregnancy in female participants, changes in the atherosclerosis process over time and frequency of cardiovascular imaging in participants with HoFH and treated with evinacumab.
• Data analysis plan:
  Descriptive analysis of safety data with annual interim analysis and final report at 5 years.