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

Last updated: February 14, 2025
Sponsor: Fondazione SISA (Societa Italiana per lo Studio della Arteriosclerosi)
Overall Status: Active - Recruiting

Phase

N/A

Condition

Hypercholesterolemia

Dyslipidemia

Familial Hypercholesterolemia

Treatment

N/A

Clinical Study ID

NCT06832371
LILITH
  • Ages > 18
  • All Genders

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.

Eligibility Criteria

Inclusion

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 confirmMACE

  • Giving written informed consent

Exclusion

Exclusion Criteria:

  • Patients who were prescribed lomitapide outside of the marketing authorization or incontraindicated patients

  • Patients receiving lomitapide in clinical trials

  • Patients receiving an investigational agent, defined as any drug or biologic agentother than lomitapide that has not received Market Authorization in the country ofparticipation, at the time of enrolment

Study Design

Total Participants: 72
Study Start date:
September 09, 2024
Estimated Completion Date:
December 31, 2026

Study Description

This is a multicenter, international, long-term observational study investigating the real-world impact of lomitapide on Major Adverse Cardiovascular Events (MACE) in patients with Homozygous Familial Hypercholesterolemia (HoFH).

Study Design:

Observational, open-label, retrospective and prospective study Data will be collected from >26 lipid centers across Europe Patients will serve as their own control, with comparisons between pre-treatment (3 years before lomitapide) and post-treatment (first 3 years of lomitapide therapy) periods

Study Population:

Approximately 72 adult patients (≥18 years) diagnosed with HoFH Patients must have received lomitapide for at least 12 months Availability of 3 years of pre-treatment clinical records

Objectives:

Primary Objective: Evaluate the incidence of MACE before and after lomitapide treatment

Secondary Objectives: Assess changes in LDL-C, total cholesterol, liver function tests (ALT, AST, GGT), and lipid-lowering therapy usage (e.g., discontinuation of LDL apheresis, addition of PCSK9 inhibitors)

Endpoints:

Primary Endpoint: Change in MACE incidence over the 3-year treatment period

Secondary Endpoints: Changes in lipid levels, liver safety markers, and adherence to treatment protocols

Safety Considerations:

The study follows real-world clinical practice, with monitoring of adverse events, including liver-related safety concerns associated with lomitapide

Data will be collected in an electronic Case Report Form (eCRF) and analyzed following Good Clinical Practice (GCP) guidelines

This study aims to generate real-world evidence on the cardiovascular impact of lomitapide in HoFH patients, addressing an unmet clinical need for data on long-term outcomes.

Connect with a study center

  • CHRU Lille - Centre Hospitalier Universitaire de Lille

    Lille,
    France

    Active - Recruiting

  • Centre Hospitalier Universitaire de Lyon (CHU Lyon)

    Lyon,
    France

    Active - Recruiting

  • Hôpital La Timone - Assistance Publique Hôpitaux de Marseille (AP-HM)

    Marseille,
    France

    Active - Recruiting

  • Hôpital La Pitié Salpêtrière - Groupe Hospitalier "La Pitié Salpêtrière - Charles Foix"

    Paris,
    France

    Active - Recruiting

  • Strasbourg University Hospital (CHU Strasbourg)

    Strasbourg,
    France

    Active - Recruiting

  • University General Hospital of Ioannina

    Ioannina,
    Greece

    Active - Recruiting

  • University Hospital of Ioannina

    Ioannina,
    Greece

    Active - Recruiting

  • METROPOLITAN HOSPITAL, Piraeus

    Piraeus,
    Greece

    Active - Recruiting

  • Policlinico di Catanzaro - A.O.U. Mater Domini

    Catanzaro, Calabria 88100
    Italy

    Active - Recruiting

  • Ospedale Sant'Anna e San Sebastiano

    Caserta, Campania 81100
    Italy

    Active - Recruiting

  • Ospedale Monaldi - AORN dei Colli, Napoli

    Napoli, Campania 80131
    Italy

    Active - Recruiting

  • Policlinico Federico II di Napoli

    Napoli, Campania 80131
    Italy

    Active - Recruiting

  • Policlinico Sant'Orsola - Università di Bologna

    Bologna, Emilia-Romagna 40138
    Italy

    Active - Recruiting

  • Arcispedale Sant'Anna - Università degli Studi di Ferrara

    Ferrara, Emilia-Romagna 44124
    Italy

    Active - Recruiting

  • Ospedale Civile di Baggiovara - A.O.U. di Modena

    Modena, Emilia-Romagna 41124
    Italy

    Active - Recruiting

  • DIMI - Dipartimento di Medicina Interna, Università di Genova

    Genova, GE 16132
    Italy

    Active - Recruiting

  • Policlinico Umberto I - Sapienza Università di Roma

    Roma, Lazio 00161
    Italy

    Active - Recruiting

  • Ospedale Bassini - ASST Nord Milano

    Milano, Lombardia 20092
    Italy

    Active - Recruiting

  • Policlinico Paolo Giaccone - Università degli Studi di Palermo

    Palermo, Sicilia 90127
    Italy

    Active - Recruiting

  • Ospedale San Luigi Gonzaga

    Orbassano, Torino 10043
    Italy

    Active - Recruiting

  • CNR Gabriele Monasterio - Centro di Aferesi, Pisa

    Pisa, Toscana 56124
    Italy

    Active - Recruiting

  • Policlinico di Padova - A.O.U. di Padova

    Padova, Veneto 35128
    Italy

    Site Not Available

  • Ospedale Borgo Trento - A.O.U. Integrata Verona

    Verona, Veneto 37126
    Italy

    Active - Recruiting

  • A.O.U. Città della Salute e della Scienza di Torino - Ospedale Molinette

    Torino, 10126
    Italy

    Active - Recruiting

  • Radboud University Medical Centre

    Nijmegen,
    Netherlands

    Active - Recruiting

  • Rotterdam Erasmus Medical Center

    Rotterdam,
    Netherlands

    Active - Recruiting

  • Queen Elizabeth Hospital Birmingham (QEII - Birmingham)

    Birmingham, England
    United Kingdom

    Active - Recruiting

  • Harefield Hospital - Royal Brompton & Harefield NHS Foundation Trust

    Harefield, England
    United Kingdom

    Active - Recruiting

  • Hammersmith Hospital - Imperial College Healthcare NHS Trust

    London, England W12 0HS
    United Kingdom

    Active - Recruiting

  • Manchester University Hospital - NHS Foundation Trust

    Manchester, England
    United Kingdom

    Active - Recruiting

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