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Heart attack prevention hope for rare cholesterol disorder

NCT ID NCT06832371

Disease control Sponsor: Fondazione SISA (Societa Italiana per lo Studio della Arteriosclerosi) Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tracks 73 adults with homozygous familial hypercholesterolemia (HoFH), a rare genetic disorder causing extremely high cholesterol and early heart disease. Researchers will compare major cardiovascular events (like heart attacks and strokes) during the first three years of lomitapide treatment to the three years before starting the drug. The goal is to see if this approved cholesterol-lowering medication reduces serious heart problems.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
lomitapide
What this could lead to
If successful, this study could confirm that lomitapide reduces heart attacks and strokes in people with this rare genetic condition.
What could go wrong
This is an observational study, not a controlled trial, so results may be influenced by other factors. The small size (73 patients) limits how broadly findings can be applied.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

73 people

The number who actually took part.

Started

Sep 2024

Expected to finish

Dec 2026

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

Patients with a clinical or genetic diagnosis of homozygous familial hypercholesterolemia (HoFH) who have been treated with lomitapide for at least 12 months.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

atherosclerosis cardiovascular disorder Dyslipidemias homozygous familial hypercholesterolemia Hypercholesterolemia inherited lipid metabolism disorder Lipid Metabolism Disorders

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Dyslipidemia Homozygous familial hypercholesterolemia (HOFH) Major adverse cardiovascular events (MACE)

Contacts and locations

Locations

  • A.O.U. Città della Salute e della Scienza di Torino - Ospedale Molinette

    Torino, Torino, 10126, Italy

  • ARNAS Garibaldi

    Catania, Italy, Italy

  • Arcispedale Sant'Anna - Università degli Studi di Ferrara

    Ferrara, Emilia-Romagna, 44124, Italy

  • CHRU Lille - Centre Hospitalier Universitaire de Lille

    Lille, France

  • CNR Gabriele Monasterio - Centro di Aferesi, Pisa

    Pisa, Tuscany, 56124, Italy

  • Centre Hospitalier Universitaire de Lyon (CHU Lyon)

    Lyon, France

  • DIMI - Dipartimento di Medicina Interna, Università di Genova

    Genova, GE, 16132, Italy

  • Hammersmith Hospital - Imperial College Healthcare NHS Trust

    London, England, W12 0HS, United Kingdom

  • Harefield Hospital - Royal Brompton & Harefield NHS Foundation Trust

    Harefield, England, United Kingdom

  • Hôpital La Pitié Salpêtrière - Groupe Hospitalier "La Pitié Salpêtrière - Charles Foix"

    Paris, France

  • Hôpital de la Conception - Assistance Publique Hôpitaux de Marseille (AP-HM)

    Marseille, France

  • METROPOLITAN HOSPITAL, Piraeus

    Piraeus, Greece

  • Manchester University Hospital - NHS Foundation Trust

    Manchester, England, United Kingdom

  • Ospedale Bassini - ASST Nord Milano

    Milan, Lombardy, 20092, Italy

  • Ospedale Borgo Trento - A.O.U. Integrata Verona

    Verona, Veneto, 37126, Italy

  • Ospedale Civile di Baggiovara - A.O.U. di Modena

    Modena, Emilia-Romagna, 41124, Italy

  • Ospedale San Luigi Gonzaga

    Orbassano, Torino, 10043, Italy

  • Ospedale Sant'Anna e San Sebastiano

    Caserta, Campania, 81100, Italy

  • Ospedale regionale generale "F. Miulli"

    Bari, Italy

  • Policlinico Federico II di Napoli

    Naples, Campania, 80131, Italy

  • Policlinico Paolo Giaccone - Università degli Studi di Palermo

    Palermo, Sicily, 90127, Italy

  • Policlinico Sant'Orsola - Università di Bologna

    Bologna, Emilia-Romagna, 40138, Italy

  • Policlinico Umberto I - Sapienza Università di Roma

    Rome, Lazio, 00161, Italy

  • Policlinico di Catanzaro - A.O.U. Mater Domini

    Catanzaro, Calabria, 88100, Italy

  • Policlinico di Padova - A.O.U. di Padova

    Padova, Veneto, 35128, Italy

  • Queen Elizabeth Hospital Birmingham (QEII - Birmingham)

    Birmingham, England, United Kingdom

  • Radboud University Medical Centre

    Nijmegen, Netherlands

  • Rotterdam Erasmus Medical Center

    Rotterdam, Netherlands

  • Strasbourg University Hospital (CHU Strasbourg)

    Strasbourg, France

  • University General Hospital of Ioannina

    Ioannina, Greece

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