LILITH- Homozygous familial hypercholesterolemia observational study

• Research type

  Research Study

• Full title

  Evaluation of the effect of lomitapide treatment on major adverse cardiovascular events (MACE) in patients with homozygous familial hypercholesterolemia.

• IRAS ID

  345905

• Contact name

  Alberico Luigi Catapano

• Contact email

  fondazione@sisa.it

• Sponsor organisation

  Fondazione SISA

• Duration of Study in the UK

  2 years, 10 months, 5 days

• Research summary

  This is an observational study of Homozygous familial hypercholesterolemia (HoFH), which is a rare life-threatening medical condition characterised by a severe increase
  of LDL-C (bad cholesterol) and accelerated atherosclerosis (making the blood flow through arteries more difficult due to fatty material build up). These individuals are at hight risk for Coronary heart conditions if their cholesterol levels are not low enough.
  Approved drug, Lomitapide, can reduce one of the important proteins call microsomal triglycerides transferase protein (MTP) and has shown to be very effective in reducing LDL-cholesterol in HoFH patients. The drug is already available on the market. Many Lipid centres are known to be routinely treating HoFH patients with lomitapide.
  This observational study will collect patient data generated as part of their standard of care treatment. The study design involves collecting already available patient data and new patient data to evaluate the occurrence of major adverse cardiovascular events (MACE). More than 26 centres from Europe and other regions will participate. The Sponsor (Fondazione SISA, Italy) of the study is academic organisation. The maximum duration of the study will be 37 months (~3 yrs.) Enrolment period will be 12 months and the new data collection will last maximum of 24 months followed by 1 month follow-up. The old data collection involves collection of at least 4 years retrospective data (3 years prior to lomitapide and 1 year on lomitapide). In patients that have already completed 37 months treatment with lomitapide, observation data collection can be extended to 5 years of treatment. The data related to the further 2 years of observation can be collected prospectively or retrospectively.

• REC name

  East Midlands - Leicester Central Research Ethics Committee

• REC reference

  24/EM/0275

• Date of REC Opinion

  30 Jan 2025

• REC opinion

  Further Information Favourable Opinion