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Heart attack prevention hope for rare cholesterol disorder

NCT ID NCT06832371

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

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.

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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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