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NCT06832371Active, not recruiting

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

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

Source: ClinicalTrials.gov · Last updated 3 Nov 2026

Brief summary (registry, in English)

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.

Status (registry)
Active, not recruiting
Study type
Observational
Enrollment
73 (actual)
First submitted
10 Feb 2025
Start date
9 Sept 2024
Primary completion date
Feb 2028
Lead sponsor
Fondazione SISA (Societa Italiana per lo Studio della Arteriosclerosi)
NCT number
NCT06832371

What you commit to

  • Study type: Observational
  • Participants enrolled: 73
  • From 9 Sept 2024 to the primary completion date, Feb 2028
  • 30 sites in 5 countries
  • Visits and tests: ask the trial team, the registry does not list them

Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.

Who cannot take part

Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.

Prior treatment

  • 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

Other

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

Who the study is looking for

  • 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

Age in the registry: 18 years to - · Sex: All · Does not accept healthy volunteers

Full criteria on ClinicalTrials.gov

Sites (30)

Sites in registry order. Pick your city on the disease page to sort them by distance.

SiteCityCountrySite status
CHRU Lille - Centre Hospitalier Universitaire de Lille
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LilleFranceUnknown status
Centre Hospitalier Universitaire de Lyon (CHU Lyon)
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LyonFranceUnknown status
Hôpital de la Conception - Assistance Publique Hôpitaux de Marseille (AP-HM)
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MarseilleFranceUnknown status
Hôpital La Pitié Salpêtrière - Groupe Hospitalier "La Pitié Salpêtrière - Charles Foix"
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ParisFranceUnknown status
Strasbourg University Hospital (CHU Strasbourg)
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StrasbourgFranceUnknown status
Queen Elizabeth Hospital Birmingham (QEII - Birmingham)
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BirminghamUnited KingdomUnknown status
Harefield Hospital - Royal Brompton & Harefield NHS Foundation Trust
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HarefieldUnited KingdomUnknown status
Hammersmith Hospital, Imperial College Healthcare NHS Trust
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LondonUnited KingdomUnknown status
Manchester University Hospital - NHS Foundation Trust
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ManchesterUnited KingdomUnknown status
University General Hospital of Ioannina
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IoanninaGreeceUnknown status
METROPOLITAN HOSPITAL, Piraeus
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PiraeusGreeceUnknown status
Ospedale regionale generale "F. Miulli"
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BariItalyUnknown status
Policlinico Sant'Orsola - Università di Bologna
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BolognaItalyUnknown status
Ospedale Sant'Anna e san Sebastiano
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CasertaItalyUnknown status
Arnas Garibaldi
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CataniaItalyUnknown status
Policlinico di Catanzaro - A.O.U. Mater Domini
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CatanzaroItalyUnknown status
Arcispedale Sant'Anna - Università degli Studi di Ferrara
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FerraraItalyUnknown status
DIMI - Dipartimento di Medicina Interna, Università di Genova
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GenovaItalyUnknown status
Ospedale Bassini - ASST Nord Milano
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MilanItalyUnknown status
Ospedale Civile di Baggiovara - A.O.U. di Modena
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ModenaItalyUnknown status
Policlinico Federico II di Napoli
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NaplesItalyUnknown status
Ospedale San Luigi Gonzaga
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OrbassanoItalyUnknown status
Policlinico di Padova - A.O.U. di Padova
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PadovaItalyUnknown status
Policlinico Paolo Giaccone - Università degli Studi di Palermo
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PalermoItalyUnknown status
CNR Gabriele Monasterio - Centro di Aferesi, Pisa
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PisaItalyUnknown status
Policlinico Umberto I - Sapienza Università di Roma
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RomeItalyUnknown status
A.O.U. Citta della Salute e della Scienza di Torino - Ospedale Molinette
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TorinoItalyUnknown status
Ospedale Borgo Trento - A.O.U. Integrata Verona
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VeronaItalyUnknown status
Radboud university medical centre
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NijmegenNetherlandsUnknown status
Rotterdam Erasmus Medical Center
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RotterdamNetherlandsUnknown status

This page is information, not medical advice. Discuss any trial with your doctor.