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)
- Diseases
- Familial hypercholesterolaemia
- 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
Sites (30)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| CHRU Lille - Centre Hospitalier Universitaire de Lille Coordinator of this site? Confirm its status | Lille | France | Unknown status |
| Centre Hospitalier Universitaire de Lyon (CHU Lyon) Coordinator of this site? Confirm its status | Lyon | France | Unknown status |
| Hôpital de la Conception - Assistance Publique Hôpitaux de Marseille (AP-HM) Coordinator of this site? Confirm its status | Marseille | France | Unknown status |
| Hôpital La Pitié Salpêtrière - Groupe Hospitalier "La Pitié Salpêtrière - Charles Foix" Coordinator of this site? Confirm its status | Paris | France | Unknown status |
| Strasbourg University Hospital (CHU Strasbourg) Coordinator of this site? Confirm its status | Strasbourg | France | Unknown status |
| Queen Elizabeth Hospital Birmingham (QEII - Birmingham) Coordinator of this site? Confirm its status | Birmingham | United Kingdom | Unknown status |
| Harefield Hospital - Royal Brompton & Harefield NHS Foundation Trust Coordinator of this site? Confirm its status | Harefield | United Kingdom | Unknown status |
| Hammersmith Hospital, Imperial College Healthcare NHS Trust Coordinator of this site? Confirm its status | London | United Kingdom | Unknown status |
| Manchester University Hospital - NHS Foundation Trust Coordinator of this site? Confirm its status | Manchester | United Kingdom | Unknown status |
| University General Hospital of Ioannina Coordinator of this site? Confirm its status | Ioannina | Greece | Unknown status |
| METROPOLITAN HOSPITAL, Piraeus Coordinator of this site? Confirm its status | Piraeus | Greece | Unknown status |
| Ospedale regionale generale "F. Miulli" Coordinator of this site? Confirm its status | Bari | Italy | Unknown status |
| Policlinico Sant'Orsola - Università di Bologna Coordinator of this site? Confirm its status | Bologna | Italy | Unknown status |
| Ospedale Sant'Anna e san Sebastiano Coordinator of this site? Confirm its status | Caserta | Italy | Unknown status |
| Arnas Garibaldi Coordinator of this site? Confirm its status | Catania | Italy | Unknown status |
| Policlinico di Catanzaro - A.O.U. Mater Domini Coordinator of this site? Confirm its status | Catanzaro | Italy | Unknown status |
| Arcispedale Sant'Anna - Università degli Studi di Ferrara Coordinator of this site? Confirm its status | Ferrara | Italy | Unknown status |
| DIMI - Dipartimento di Medicina Interna, Università di Genova Coordinator of this site? Confirm its status | Genova | Italy | Unknown status |
| Ospedale Bassini - ASST Nord Milano Coordinator of this site? Confirm its status | Milan | Italy | Unknown status |
| Ospedale Civile di Baggiovara - A.O.U. di Modena Coordinator of this site? Confirm its status | Modena | Italy | Unknown status |
| Policlinico Federico II di Napoli Coordinator of this site? Confirm its status | Naples | Italy | Unknown status |
| Ospedale San Luigi Gonzaga Coordinator of this site? Confirm its status | Orbassano | Italy | Unknown status |
| Policlinico di Padova - A.O.U. di Padova Coordinator of this site? Confirm its status | Padova | Italy | Unknown status |
| Policlinico Paolo Giaccone - Università degli Studi di Palermo Coordinator of this site? Confirm its status | Palermo | Italy | Unknown status |
| CNR Gabriele Monasterio - Centro di Aferesi, Pisa Coordinator of this site? Confirm its status | Pisa | Italy | Unknown status |
| Policlinico Umberto I - Sapienza Università di Roma Coordinator of this site? Confirm its status | Rome | Italy | Unknown status |
| A.O.U. Citta della Salute e della Scienza di Torino - Ospedale Molinette Coordinator of this site? Confirm its status | Torino | Italy | Unknown status |
| Ospedale Borgo Trento - A.O.U. Integrata Verona Coordinator of this site? Confirm its status | Verona | Italy | Unknown status |
| Radboud university medical centre Coordinator of this site? Confirm its status | Nijmegen | Netherlands | Unknown status |
| Rotterdam Erasmus Medical Center Coordinator of this site? Confirm its status | Rotterdam | Netherlands | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.