Active not recruitingOBSERVATIONAL

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

This study focuses on people in Europe who have a rare inherited condition called Homozygous Familial Hypercholesterolemia (HoFH). HoFH means you have extremely high cholesterol from birth, which can lead to serious heart problems at a young age. The research aims to understand if a medicine called lomitapide, which is used to lower cholesterol, helps prevent or reduce these heart-related issues. The study will look at the health records of adult patients who have been taking lomitapide for at least a year. Researchers will compare heart problems these patients had in the three years before starting lomitapide with the heart problems they had during the first three years of taking the medicine. This will help doctors learn more about the long-term benefits of lomitapide in real-life situations.

At a glance

Status

Active not recruiting

Sponsor

Fondazione SISA (Societa Italiana per lo Studio della Arteriosclerosi)

Enrolment target

Start

09 Sep 2024

Estimated completion

01 Dec 2026

Homozygous Familial Hypercholesterolemia (HoFH)Major Adverse Cardiovascular Events (MACE)Dyslipidemia

What is this study about?

This study is about a very specific and rare health condition called Homozygous Familial Hypercholesterolemia, often shortened to HoFH. If you have HoFH, it means your body has extremely high levels of 'bad' cholesterol (LDL cholesterol) right from birth because of a faulty gene you inherited. This high cholesterol can cause serious heart and blood vessel problems, like heart attacks or strokes, much earlier in life than usual.

Doctors are interested in a medicine called lomitapide, which is approved to treat HoFH. This medicine works by helping to lower those very high cholesterol levels. The main goal of this research is to see if taking lomitapide can help reduce the chances of people with HoFH having serious heart-related events. These events are sometimes called 'Major Adverse Cardiovascular Events' or MACE for short, and they include things like heart attacks, strokes, or needing heart surgery.

To figure this out, researchers are looking at the medical records of adults with HoFH who are already taking lomitapide. They will compare a patient's health during the three years before they started taking lomitapide with their health during the first three years they were on the medicine. This helps doctors understand how the medicine works in a real-world setting, rather than in a controlled lab environment. They'll also check other things like how the medicine affects liver health and other cholesterol levels.

Key takeaways

This study looks at a rare condition called HoFH.
It aims to see if lomitapide helps prevent heart problems in HoFH patients.
Researchers will review existing medical records, not conduct new tests.
Participation will not change your current medical care.
The study helps doctors understand real-world effects of lomitapide.
Your health records from before and after starting lomitapide will be used.

Who may be eligible?

To be considered for this study, you must be an adult, aged 18 or older, and have a confirmed diagnosis of Homozygous Familial Hypercholesterolemia (HoFH). It's also important that you have been taking the medicine lomitapide for at least 12 months.

Additionally, the study needs to be able to look back at your health records for the three years before you started taking lomitapide. This helps the researchers compare your health before and after starting the medicine. Finally, you would need to agree to take part in the study by giving your written consent.

You cannot join the study if you received lomitapide in a way that wasn't approved by doctors, or if you were previously involved in other clinical trials for lomitapide. You also can't be taking any experimental drugs that haven't been approved for use yet in your country.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 18 years old or older?
Do you have a diagnosis of HoFH?
Have you been taking lomitapide for at least 12 months?
Can your doctor provide your medical records from the 3 years before you started lomitapide?
Are you willing to provide written permission for your de-identified data to be used?

Answer every question to see your result.

What does participation involve?

If you take part in this study, you won't need to change your current treatment plan with lomitapide or attend extra clinic visits specifically for the study. This is an 'observational' study, which means researchers will gather information from your existing medical records from your doctor's office or hospital.

They will look at data collected during your regular check-ups, so there will be no new tests or medications given to you as part of the study. The study involves reviewing information over a long period: the three years before you started lomitapide and the first three years you were taking it. Your doctor will continue to monitor your health and any side effects as part of your standard care.

Potential risks and benefits

Since this is an observational study using existing medical records, there are very few direct risks associated with simply participating. You won't be given any new treatments or asked to do anything different. However, the study involves collecting your health data, which will be handled carefully to protect your privacy. Lomitapide itself can have side effects, including liver-related issues, which your doctor should already be monitoring as part of your ongoing care. A potential benefit of taking part is that the information gathered from your experience, combined with others, could help doctors better understand how lomitapide works in the long term, potentially improving care for future patients with HoFH. Remember, you have the right to withdraw your consent and stop participating at any time without it affecting your medical care.

Locations (30)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

CHRU Lille - Centre Hospitalier Universitaire de Lille
Verified postcode
Lille, France
Centre Hospitalier Universitaire de Lyon (CHU Lyon)
Verified postcode
Lyon, France
Hôpital de la Conception - Assistance Publique Hôpitaux de Marseille (AP-HM)
Verified postcode
Marseille, France
Hôpital La Pitié Salpêtrière - Groupe Hospitalier "La Pitié Salpêtrière - Charles Foix"
Verified postcode
Paris, France
Strasbourg University Hospital (CHU Strasbourg)
Verified postcode
Strasbourg, France
University General Hospital of Ioannina
Verified postcode
Ioannina, Greece
METROPOLITAN HOSPITAL, Piraeus
Verified postcode
Piraeus, Greece
Policlinico di Catanzaro - A.O.U. Mater Domini
Verified postcode
Catanzaro, Italy
Ospedale Sant'Anna e San Sebastiano
Verified postcode
Caserta, Italy
Policlinico Federico II di Napoli
Verified postcode
Naples, Italy
Policlinico Sant'Orsola - Università di Bologna
Verified postcode
Bologna, Italy
Arcispedale Sant'Anna - Università degli Studi di Ferrara
Verified postcode
Ferrara, Italy

Common questions

What is HoFH?

HoFH (Homozygous Familial Hypercholesterolemia) is a rare inherited condition that causes extremely high cholesterol levels from birth, increasing the risk of early heart problems.

What is Lomitapide?

Lomitapide is a medicine approved to help lower cholesterol in people with HoFH.

What will I have to do if I join?

You won't have to do anything extra. The study will look at your existing medical records from before and after you started taking lomitapide.

Will I get a new medicine?

No, this study is about how your current lomitapide treatment works. You won't receive any new medications for the study.

Is my information kept private?

Yes, all your personal health information collected for the study will be handled with strict privacy and confidentiality.