Evaluation of the Effect of Lomitapide Treatment on Major Adverse Cardiovascular Events (MACE) in Patients With Homozygous Familial Hypercholesterolemia
NCT ID: NCT06832371
Last Updated: 2025-02-18
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
72 participants
OBSERVATIONAL
2024-09-09
2026-12-31
Brief Summary
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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.
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Detailed Description
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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.
Conditions
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Study Design
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COHORT
OTHER
Study Groups
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HoFH Patients Treated with Lomitapide
This cohort consists of adult patients (≥18 years) diagnosed with Homozygous Familial Hypercholesterolemia (HoFH) who have been treated with lomitapide for at least 12 months. The study will retrospectively analyze the incidence of Major Adverse Cardiovascular Events (MACE) in the three years before lomitapide treatment, and prospectively track MACE incidence during the first three years of lomitapide therapy.
No interventions assigned to this group
Eligibility Criteria
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Inclusion Criteria
* 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 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
18 Years
ALL
No
Sponsors
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Amryt Pharmaceuticals DAC
UNKNOWN
Clinical Trial Consulting
UNKNOWN
CMV-Stat S.r.l.
UNKNOWN
Fondazione SISA (Societa Italiana per lo Studio della Arteriosclerosi)
OTHER
Responsible Party
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Locations
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CHRU Lille - Centre Hospitalier Universitaire de Lille
Lille, , France
Centre Hospitalier Universitaire de Lyon (CHU Lyon)
Lyon, , France
Hôpital La Timone - Assistance Publique Hôpitaux de Marseille (AP-HM)
Marseille, , France
Hôpital La Pitié Salpêtrière - Groupe Hospitalier "La Pitié Salpêtrière - Charles Foix"
Paris, , France
Strasbourg University Hospital (CHU Strasbourg)
Strasbourg, , France
University General Hospital of Ioannina
Ioannina, , Greece
University Hospital of Ioannina
Ioannina, , Greece
METROPOLITAN HOSPITAL, Piraeus
Piraeus, , Greece
Policlinico di Catanzaro - A.O.U. Mater Domini
Catanzaro, Calabria, Italy
Ospedale Sant'Anna e San Sebastiano
Caserta, Campania, Italy
Ospedale Monaldi - AORN dei Colli, Napoli
Napoli, Campania, Italy
Policlinico Federico II di Napoli
Napoli, Campania, Italy
Policlinico Sant'Orsola - Università di Bologna
Bologna, Emilia-Romagna, Italy
Arcispedale Sant'Anna - Università degli Studi di Ferrara
Ferrara, Emilia-Romagna, Italy
Ospedale Civile di Baggiovara - A.O.U. di Modena
Modena, Emilia-Romagna, Italy
DIMI - Dipartimento di Medicina Interna, Università di Genova
Genova, GE, Italy
Policlinico Umberto I - Sapienza Università di Roma
Rome, Lazio, Italy
Ospedale Bassini - ASST Nord Milano
Milan, Lombardy, Italy
Policlinico Paolo Giaccone - Università degli Studi di Palermo
Palermo, Sicily, Italy
Ospedale San Luigi Gonzaga
Orbassano, Torino, Italy
A.O.U. Città della Salute e della Scienza di Torino - Ospedale Molinette
Torino, Torino, Italy
CNR Gabriele Monasterio - Centro di Aferesi, Pisa
Pisa, Tuscany, Italy
Policlinico di Padova - A.O.U. di Padova
Padua, Veneto, Italy
Ospedale Borgo Trento - A.O.U. Integrata Verona
Verona, Veneto, Italy
Radboud University Medical Centre
Nijmegen, , Netherlands
Rotterdam Erasmus Medical Center
Rotterdam, , Netherlands
Queen Elizabeth Hospital Birmingham (QEII - Birmingham)
Birmingham, England, United Kingdom
Harefield Hospital - Royal Brompton & Harefield NHS Foundation Trust
Harefield, England, United Kingdom
Hammersmith Hospital - Imperial College Healthcare NHS Trust
London, England, United Kingdom
Manchester University Hospital - NHS Foundation Trust
Manchester, England, United Kingdom
Countries
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Facility Contacts
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Fabio Pellegatta, MD
Role: backup
Maurizio Averna, Prof.
Role: primary
Baldassarre Cefalù, Prof.
Role: backup
Deena Patel
Role: backup
Other Identifiers
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LILITH
Identifier Type: -
Identifier Source: org_study_id
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