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

Status: Recruiting
Location: See all (30) locations...
Study Type: Observational
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
Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: f
View:

• 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

Locations
Other Locations
France
CHRU Lille - Centre Hospitalier Universitaire de Lille
RECRUITING
Lille
Centre Hospitalier Universitaire de Lyon (CHU Lyon)
RECRUITING
Lyon
Hôpital La Timone - Assistance Publique Hôpitaux de Marseille (AP-HM)
RECRUITING
Marseille
Hôpital La Pitié Salpêtrière - Groupe Hospitalier La Pitié Salpêtrière - Charles Foix
RECRUITING
Paris
Strasbourg University Hospital (CHU Strasbourg)
RECRUITING
Strasbourg
Greece
University General Hospital of Ioannina
RECRUITING
Ioannina
University Hospital of Ioannina
RECRUITING
Ioannina
METROPOLITAN HOSPITAL, Piraeus
RECRUITING
Piraeus
Italy
Policlinico Sant'Orsola - Università di Bologna
RECRUITING
Bologna
Ospedale Sant'Anna e San Sebastiano
RECRUITING
Caserta
Policlinico di Catanzaro - A.O.U. Mater Domini
RECRUITING
Catanzaro
Arcispedale Sant'Anna - Università degli Studi di Ferrara
RECRUITING
Ferrara
DIMI - Dipartimento di Medicina Interna, Università di Genova
RECRUITING
Genova
Ospedale Bassini - ASST Nord Milano
RECRUITING
Milan
Ospedale Civile di Baggiovara - A.O.U. di Modena
RECRUITING
Modena
Ospedale Monaldi - AORN dei Colli, Napoli
RECRUITING
Napoli
Policlinico Federico II di Napoli
RECRUITING
Napoli
Ospedale San Luigi Gonzaga
RECRUITING
Orbassano
Policlinico di Padova - A.O.U. di Padova
NOT_YET_RECRUITING
Padua
Policlinico Paolo Giaccone - Università degli Studi di Palermo
RECRUITING
Palermo
CNR Gabriele Monasterio - Centro di Aferesi, Pisa
RECRUITING
Pisa
Policlinico Umberto I - Sapienza Università di Roma
RECRUITING
Rome
A.O.U. Città della Salute e della Scienza di Torino - Ospedale Molinette
RECRUITING
Torino
Ospedale Borgo Trento - A.O.U. Integrata Verona
RECRUITING
Verona
Netherlands
Radboud University Medical Centre
RECRUITING
Nijmegen
Rotterdam Erasmus Medical Center
RECRUITING
Rotterdam
United Kingdom
Queen Elizabeth Hospital Birmingham (QEII - Birmingham)
RECRUITING
Birmingham
Harefield Hospital - Royal Brompton & Harefield NHS Foundation Trust
RECRUITING
Harefield
Hammersmith Hospital - Imperial College Healthcare NHS Trust
RECRUITING
London
Manchester University Hospital - NHS Foundation Trust
RECRUITING
Manchester
Time Frame
Start Date: 2024-09-09
Estimated Completion Date: 2026-12
Participants
Target number of participants: 72
Treatments
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.
Sponsors
Collaborators: Amryt Pharmaceuticals DAC, CMV-Stat S.r.l., Clinical Trial Consulting
Leads: Fondazione SISA (Societa Italiana per lo Studio della Arteriosclerosi)

This content was sourced from clinicaltrials.gov