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Contact the study team using the details below to take part. If there are no contact details below please ask your doctor in the first instance.
Prof
Alberico
Catapano
+39 (0)2 49637591
alberico.catapano@unimi.it
Dr
Jaimini
Cegla
+44 (0)7775557295
j.cegla@imperial.ac.uk
MACE in patients with familial hypercholesterolemia
This information is provided directly by researchers, and we recognise that it isn't always easy to understand. We are working with researchers to improve the accessibility of this information. In some summaries, you may come across links to external websites. These websites will have more information to help you better understand the study.
Homozygous familial hypercholesterolemia (HoFH) is a rare, life-threatening condition characterized by a severe elevation of LDL cholesterol (LDL-C) and accelerated atherosclerosis. In these patients, an aggressive therapy to reduce LDL-C is mandatory to control the high risk of CHD associated with this disease. Lomitapide has been demonstrated to be very effective in reducing LDL-C in HoFH in both clinical trial and real-world experience. However, limited information is available on how this drug affects cardiovascular risk. Due to the rarity of the disease, a randomized controlled trial testing the effect of lomitapide on the incidence of major adverse cardiovascular events (MACE) is not feasible.
To overcome this, an observational study with the aim of analyzing the occurrence of MACE in HoFH patients exposed to lomitapide will be performed. In the Italian network of lipid centres, information about MACE in HoFH patients exposed to lomitapide is available for more than 30 patients. The duration of follow-up among these patients was not homogenous. In fact, there was a group of patients with barely 1 year of treatment and this may not represent a sufficient time to observe any detectable benefit on cardiovascular risk, especially in adult HoFH patients exposed to high levels of LDL-C since birth. Therefore, to provide a better estimation of the effect of lomitapide therapy on MACE, we have designed this observational study with a retrospective phase in which the data available will be collected, followed by a prospective phase where all patients will be followed up to completion of at least 3 years of treatment. As a parallel cohort of untreated HoFH is not available, we have decided to compare the occurrence of MACE during the 3-year period of lomitapide treatment with that which occurred in the same cohort during the 3-year period before initiation of lomitapide.
Start dates may differ between countries and research sites. The research team are responsible for keeping the information up-to-date.
The recruitment start and end dates are as follows:
You can take part if:
You may not be able to take part if:
1. Patients who were prescribed lomitapide outside of the marketing authorization or in contraindicated patients2. Patients who are receiving lomitapide in clinical trials3. 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 time of enrolment
Below are the locations for where you can take part in the trial. Please note that not all sites may be open.
Dr
Jaimini
Cegla
+44 (0)7775557295
j.cegla@imperial.ac.uk
Prof
Alberico
Catapano
+39 (0)2 49637591
alberico.catapano@unimi.it
The study is sponsored by Fondazione S.I.S.A. and funded by Investigator initiated and funded.
Your feedback is important to us. It will help us improve the quality of the study information on this site. Please answer both questions.
You can print or share the study information with your GP/healthcare provider or contact the research team directly.
