Evaluation of the Effect of Lomitapide Treatment on Major Adverse Cardiovascular Events (MACE) in Patients With Homozygous Familial Hypercholesterolemia

Launched by FONDAZIONE SISA (SOCIETA ITALIANA PER LO STUDIO DELLA ARTERIOSCLEROSI) · Feb 14, 2025

Keywords

ClinConnect Summary

This clinical trial is studying how effective the medication lomitapide is in reducing major heart-related problems (called Major Adverse Cardiovascular Events or MACE) in patients with a rare genetic condition known as Homozygous Familial Hypercholesterolemia (HoFH). People with HoFH have very high levels of LDL cholesterol, which can lead to early heart issues. The trial will look at data from patients who have been taking lomitapide for at least 12 months, comparing their heart health during the first three years of treatment to the three years before they started taking the medication.

To be part of this study, participants need to be adults (18 years or older) who have been diagnosed with HoFH and have been on lomitapide for at least a year. They also need to provide three years of medical records before starting the treatment to help researchers understand their health history. The trial is currently recruiting participants from various lipid treatment centers across Europe, and participants can expect to have their cholesterol levels, liver function, and overall treatment effectiveness monitored throughout the study. This research aims to provide valuable information about the benefits of lomitapide for heart health in individuals with HoFH.

Gender

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Eligibility criteria

• Inclusion Criteria:
• • Adult patients (age ≥18 years)
• • Clinical or genetic diagnosis of HoFH
• • Treated with lomitapide at any dosage
• • On treatment with lomitapide for at least 12 months at the time of enrollment
• • Availability of 3 years of medical records prior to lomitapide treatment to confirm MACE
• • Giving written informed consent
• Exclusion Criteria:
• • Patients who were prescribed lomitapide outside of the marketing authorization or in contraindicated patients
• • Patients receiving lomitapide in clinical trials
• • Patients receiving an investigational agent, defined as any drug or biologic agent other than lomitapide that has not received Market Authorization in the country of participation, at the time of enrolment