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New registry tracks Real-World safety of cholesterol drug lomitapide

NCT ID NCT02135705

Disease control Sponsor: Amryt Pharma 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
Running, but no longer taking on new participants.
Completed This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This observational registry followed 260 patients with homozygous familial hypercholesterolemia (a severe genetic condition causing very high cholesterol) who were taking lomitapide. The goal was to monitor long-term safety, especially liver problems and gastrointestinal issues, and to see how well the drug works over time. Participants were already prescribed lomitapide by their doctor before joining.

What this could mean

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

Active substance
lomitapide (also known as Juxtapid or Lojuxta)
What this could lead to
If successful, this registry could confirm that lomitapide is safe and effective for long-term use in managing extremely high cholesterol.
What could go wrong
This is an observational study, not a controlled trial, so results may be less definitive. Liver and gastrointestinal side effects are known risks.

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

260 people

The number who actually took part.

Started

Mar 2014

Finished

Apr 2026

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

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 treated with lomitapide who agree to participate in the study.

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

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: * Patients of all ages, including minors, who have initiated commercial treatment with lomitapide prior to or at time of registry enrolment. * Patients who have the ability to understand the requirements of the study and provide written informed consent to comply with the study data collection procedures or paediatric patients with the consent of a parent or legal guardian. Patients ≥7 years of age (or above the age determined by the IRB/EC and in accordance with the local regulations and requirements) must also provide written informed assent forms. Exclusion Criteria: * Patients who are receiving lomitapide in clinical trials or through compassionate use, where patients are followed under a separate protocol. * 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 lomitapide initiation and continuing to receive an investigational agent at time of registry enrolment. These patients may be enrolled if receiving lomitapide when the investigational agent is discontinued..

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Conditions

The condition(s) this trial relates to.

homozygous familial hypercholesterolemia

As listed by the trial registrant

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

Homozygous familial hypercholesterolemia

Contacts and locations

Locations

  • 201 Route 17 North

    Rutherford, New Jersey, 07070, United States

  • 317 Saint Francis Dr.

    Greenville, South Carolina, 29601, United States

  • ASST Grande Ospedale Metropolitano Niguarda

    Milan, MI, 20162, Italy

  • Alfieri Cardiology

    Newark, Delaware, 19713, United States

  • Arkansas Heart Center

    Fort Smith, Arkansas, 72901, United States

  • Ascension

    Milwaukee, Wisconsin, 53211, United States

  • Aspirus Research Institute

    Wausau, Wisconsin, 54401, United States

  • Atlanta Heart Specialists

    Cumming, Georgia, 30041, United States

  • Azienda Ospedaliera G. Brotzu

    Cagliari, CA, 09134, Italy

  • Azienda Ospedaliera Specialistica Dei Colli

    Naples, 80131, Italy

  • Azienda Ospedaliera Universita di Padova

    Padova, PD, 35128, Italy

  • Azienda Ospedaliera Universitaria Federico II

    Naples, 80131, Italy

  • Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone

    Palermo, PA, 90127, Italy

  • Azienda Ospedaliero Universi consorziale policlinico di Bari

    Bari, Bari, 70120, Italy

  • Azienda Ospedaliero-Universitaria San Luigi Gonzaga

    Orbassano, TO, 10043, Italy

  • Baylor Scott & White Health

    Dallas, Texas, 75226, United States

  • Brookwood Baptist Medical Center

    Birmingham, Alabama, 35209, United States

  • CNR Regione Toscana - Fondazione Toscana Gabriele Monasterio

    Pisa, Pisa, 56124, Italy

  • Cardiology Consultants of Philadelphia

    Philadelphia, Pennsylvania, 19107, United States

  • Cardiology Specialists Of Carolina

    Charlotte, North Carolina, 28211, United States

  • Cardiology Specialists of the Carolinas PA

    Charlotte, North Carolina, 28204, United States

  • Cardiovascular Institute of the South

    Houma, Louisiana, 70360, United States

  • Cardiovascular Specialists of Texas

    Austin, Texas, 78758, United States

  • Cedars-Sinai Medical Center

    Los Angeles, California, 90048, United States

  • Centre Hospitalier Régional Universitaire de Lille

    Lille, Cedex, 59037, France

  • Centre Hospitalier Universitaire Lyon

    Bron, Lyon, 69677, France

  • Centre Hospitalier Universitaire Strasbourg

    Strasbourg, 67000, France

  • Clinica de Salud de Chaco

    Reconquista, Chaco CPA, H3500BJS, Argentina

  • Community Health Network

    Indianapolis, Indiana, 46250, United States

  • Complete Family Care Cholesterol Treatment Center

    Sterling Heights, Michigan, 48314, United States

  • Comprehensive Cardiovascular Care

    Gurnee, Illinois, 60031, United States

  • Ecogene-21

    Chicoutimi, Quebec, G7H7K9, Canada

  • Erasmus Medisch Centrum

    Rotterdam, South Holland, 3015 GD, Netherlands

  • Florida Lipid Institute

    Winter Park, Florida, 32792-2223, United States

  • Get Well Immediate Care

    Towson, Maryland, 21204, United States

  • HealthEast Care System

    Saint Paul, Minnesota, 55102, United States

  • Henry County Medical Center

    New Castle, Kentucky, 40050, United States

  • Imperial College Healthcare - NHS Trust

    London, London, W12 0NN, United Kingdom

  • Lehigh Valley Hospital And Health Network

    East Stroudsburg, Pennsylvania, 18301, United States

  • McGill University

    Montreal, Quebec, H4A3J1, Canada

  • Medical College of Wisconsin

    Milwaukee, Wisconsin, 53226, United States

  • Methodist Physicians Clinic

    Omaha, Nebraska, 68114, United States

  • Metropolitan Hospital

    Piraeus, Piraeus, 18547, Greece

  • Minneapolis Heart Institute Foundation

    Minneapolis, Minnesota, 55407, United States

  • Murray Hill Medical Group

    New York, New York, 10016, United States

  • NY Heart Center

    Syracuse, New York, 13210, United States

  • Northern Arizona Healthcare

    Cottonwood, Arizona, 86326, United States

  • Northside Hospital, Inc.

    Cumming, Georgia, 30041, United States

  • Nova Scotia Health Authority

    Halifax, Nova Scotia, B3H2Y9, Canada

  • Palmetto Health

    Columbia, South Carolina, 29203, United States

  • Pediatric Cardiology Associates

    Syracuse, New York, 13210, United States

  • Piedmont Heart Institute

    Atlanta, Georgia, 30309, United States

  • Pima Heart

    Tucson, Arizona, 85718, United States

  • Policlinico S. Orsola-Malpighi

    Bologna, BO, 40138, Italy

  • Preventative Cardiology, Inc.

    Boca Raton, Florida, 33434, United States

  • Radboud Universiteit Medisch Centrum

    Nijmegen, Gelderland, 6525 GA, Netherlands

  • Robarts Research Institute

    London, Ontario, N6A5B7, Canada

  • Scottsdale Family Health

    Phoenix, Arizona, 85018, United States

  • Springhill Physician Practices

    Mobile, Alabama, 36608, United States

  • St. Luke's University Health Network - Pennsylvania

    Bethlehem, Pennsylvania, 18015, United States

  • St. Vincent Medical Group, Inc.

    Indianapolis, Indiana, 46290, United States

  • Stern Cardiovascular Foundation

    Southaven, Mississippi, 38671, United States

  • The Heart Care Group, PC

    Allentown, Pennsylvania, 18103, United States

  • The University of Vermont Health Network

    Burlington, Vermont, 05401-1473, United States

  • Thomas Jefferson University Health System

    Philadelphia, Pennsylvania, 19148, United States

  • University General Hospital of Ioannina

    Ioannina, 45500, Greece

  • University of Colorado Health

    Aurora, Colorado, 80045, United States

  • Università degli Studi di Roma La Sapienza

    Roma, RM, 00161, Italy

  • Università degli Studi di Roma La Sapienza

    Roma, Rome, 00161, Italy

  • Valley Medical Group

    Wyckoff, New Jersey, 07481, United States

  • West Virginia University Medicine

    Morgantown, West Virginia, 26506, United States

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