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

NCT06832371 · Status: ACTIVE_NOT_RECRUITING · Type: OBSERVATIONAL · Enrollment: 73

Last updated 2026-02-20

No results posted yet for this study

Summary

This observational, multicenter, retrospective and prospective study aims to evaluate the effect of lomitapide treatment on Major Adverse Cardiovascular Events (MACE) in patients with Homozygous Familial Hypercholesterolemia (HoFH).

HoFH is a rare genetic disorder characterized by extremely high levels of LDL cholesterol (LDL-C), leading to an increased risk of early cardiovascular diseases. Lomitapide is an approved medication that lowers LDL-C levels by inhibiting microsomal triglyceride transfer protein (MTP).

The study will collect data from patients who have been treated with lomitapide for at least 12 months and will compare the incidence of MACE during the first three years of treatment with the three years before treatment initiation. The study includes data collection from multiple lipid centers across Europe.

The primary objective is to assess the impact of lomitapide on MACE, while secondary objectives include evaluating changes in lipid profiles, liver function tests, and lipid-lowering treatments.

Conditions

  • Homozygous Familial Hypercholesterolemia (HoFH)
  • Major Adverse Cardiovascular Events (MACE)
  • Dyslipidemia

Sponsors & Collaborators

  • Amryt Pharmaceuticals DAC

    collaborator UNKNOWN

  • Clinical Trial Consulting

    collaborator UNKNOWN

  • CMV-Stat S.r.l.

    collaborator UNKNOWN

  • Fondazione SISA (Societa Italiana per lo Studio della Arteriosclerosi)

    lead OTHER

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-09-09
Primary Completion
2026-12-31
Completion
2026-12-31

Countries

  • France
  • Greece
  • Italy
  • Netherlands
  • United Kingdom

Study Locations

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Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06832371 on ClinicalTrials.gov