LOWER: Lomitapide Observational Worldwide Evaluation Registry (AEGR-733-025)

Study type

• Study topic:
  Disease /health condition

  Human medicinal product

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

  Drug utilisation

  Effectiveness study (incl. comparative)

  Safety study (incl. comparative)

Non-interventional study

• Non-interventional study design:
  Cohort

  Other
• Non-interventional study design, other:
  This registry study is a multi-centre, long-term, prospective, observational cohort study

Study drug and medical condition

• Medicinal product name:
  LOJUXTA
• Medicinal product name, other:
  Juxtapid
• Study drug International non-proprietary name (INN) or common name:
  LOMITAPIDE
• Anatomical Therapeutic Chemical (ATC) code:
  (C10AX12) lomitapide

  lomitapide
• Medical condition to be studied:
  Type IIa hyperlipidaemia
• Additional medical condition(s):
  MedDRA code for homozygous familial hypercholesterolaemia (HoFH) is 10057100

Population studied

• Short description of the study population:
  patients treated with lomitapide
• Age groups:
  • Preterm newborn infants (0 – 27 days)
  • Term newborn infants (0 – 27 days)
  • Infants and toddlers (28 days – 23 months)
  • Children (2 to < 12 years)
  • 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:
  Hepatic impaired

  Renal impaired
• Estimated number of subjects:
  300

Study design details

• Main study objective:
  The main objectives of the study are to evaluate the long-term safety and effectiveness of lomitapide under conditions of usual clinical practice, and to evaluate the effectiveness of risk minimisation interventions in mitigating the serious risks of lomitapide in countries where such efforts are in place. ; The registry is designed to evaluate the long-term safety and effectiveness of lomitapide in clinical practice. Objectives: To evaluate the occurrence of the following in patients treated with lomitapide: Hepatic abnormalities, GI events, Small bowel, hepatic, colorectal and pancreatic tumours, Events associated with coagulopathy, MACE events, Death (including cause of death) and Pregnancy.
• Outcomes:
  The safety-related events of special interest include: Hepatic abnormalities, GI events, Tumours, Events associated with coagulopathy, Other safety events of interest include: MACE events, Death and cause of death, Pregnancy (both prospectively and retrospectively reported pregnancies will be collected). Effectiveness evaluations: Magnitude of reduction in serum LDL-C from baseline, Absolute and percent change from baseline in total cholesterol, non-HDL-C, apolipoprotein B (apo B), triglycerides (TG), lipoprotein a (Lp(a)), HDL-C, apolipoprotein AI (apo- AI) and very-low-density lipoprotein cholesterol (VLDL-C), Changes in concomitant medications or apheresis treatments.
• Data analysis plan:
  Summary tabulations will be presented that will display the number of observations, mean, standard deviation, median, minimum and maximum for continuous variables and the number and percentage per category for categorical or ordered categorical data. In addition, two-sided 95% confidence intervals will be calculated for all outcomes.