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Evaluating Lomitapide's Effect on Heart Events in Patients with a Rare Cholesterol Disorder

Evaluation of the Effect of Lomitapide Treatment on Major Adverse Cardiovascular Events (MACE) in Patients With Homozygous Familial Hypercholesterolemia: A Multicenter, Retrospective and Prospective Observational Study

Observational Fondazione SISA (Societa Italiana per lo Studio della Arteriosclerosi) · NCT06832371

This study is testing if the cholesterol medication lomitapide can help prevent serious heart problems in adults with a rare cholesterol disorder called Homozygous Familial Hypercholesterolemia.

Quick facts

Study typeObservational
Enrollment72 (estimated)
Ages18 Years and up
SexAll
SponsorFondazione SISA (Societa Italiana per lo Studio della Arteriosclerosi) Academic / other
Locations30 sites (Lille and 29 other locations)
Trial IDNCT06832371 on ClinicalTrials.gov

What this trial studies

This observational study investigates the impact of lomitapide treatment on Major Adverse Cardiovascular Events (MACE) in patients with Homozygous Familial Hypercholesterolemia (HoFH). It collects data from over 26 lipid centers across Europe, comparing the incidence of MACE during the first three years of lomitapide treatment to the three years prior. The study includes approximately 72 adult patients who have been treated with lomitapide for at least 12 months and have three years of pre-treatment clinical records. The primary objective is to assess the effect of lomitapide on MACE, while secondary objectives include evaluating changes in lipid profiles and liver function tests.

Who should consider this trial

Good fit: Ideal candidates for this study are adults aged 18 and older diagnosed with Homozygous Familial Hypercholesterolemia who have been treated with lomitapide for at least 12 months.

Not a fit: Patients who have not been treated with lomitapide or those with contraindications to the medication may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide insights into the effectiveness of lomitapide in reducing cardiovascular events in patients with HoFH.

How similar studies have performed: While this approach is observational, similar studies evaluating the impact of lipid-lowering therapies on cardiovascular events have shown promising results.

Eligibility criteria

Show full inclusion / exclusion 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

Where this trial is running

Lille and 29 other locations

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Homozygous Familial HypercholesterolemiaMajor Adverse Cardiovascular EventsDyslipidemiaLomitapideHypercholesterolemiaCardiovascular DiseaseLDL CholesterolLipid-Lowering Therapy
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.