Evaluation of the Effect of Lomitapide Treatment on Major Adverse Cardiovascular Events (MACE) in… (NCT06832371) | Clinical Trial Compass
Active — Not RecruitingNot Applicable
Evaluation of the Effect of Lomitapide Treatment on Major Adverse Cardiovascular Events (MACE) in Patients With Homozygous Familial Hypercholesterolemia
France, Greece, Italy73 participantsStarted 2024-09-09
Plain-language 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.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* 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
Exclusion Criteria:
* Patients who were prescribed lomitapide outside of the marketing authorization or in contraindicated patients
* 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
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Since this trial is specifically looking at whether lomitapide reduces major adverse cardiovascular events in people with homozygous familial hypercholesterolemia — the exact condition I have — is this study relevant to my situation, and could the findings change how you'd approach my treatment?
2The trial is listed as 'active, not recruiting,' which means I can't enroll — but do you think the data being collected here could affect your recommendations for me once results are published, and how soon might that be?
3Lomitapide is already an approved medication for HoFH — so how does what this trial is measuring about real-world cardiovascular outcomes compare to what we already know about its safety and effectiveness, and does that change how confident you'd be prescribing it for me?
4Given that this is a Phase NA observational-style study focused on MACE incidence before and after lomitapide treatment, are there any known cardiovascular risks or side effects from lomitapide that I should be weighing against potential benefits in my specific case?
5Are there other current treatment options or trials for homozygous familial hypercholesterolemia that I should be considering alongside or instead of lomitapide, especially since I can't join this particular study right now?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Incidence of Major Adverse Cardiovascular Events (MACE) Before and After Lomitapide Treatment
Timeframe: 3 years before treatment vs. 3 years during treatment
Trial details
NCT IDNCT06832371
SponsorFondazione SISA (Societa Italiana per lo Studio della Arteriosclerosi)