LOWER: Lomitapide Observational Worldwide Evaluation Registry (LOWER)

May 7, 2026 updated by: Amryt Pharma
This global product exposure registry is a multicentre, long-term, prospective, observational cohort study (exposure registry), designed to evaluate the long term safety and effectiveness of lomitapide.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

To evaluate the occurrence of adverse events of special interest, long term effectiveness of lomitapide, and to evaluate whether prescribers of lomitapide are following screening and monitoring recommendations as specified in product labeling.

Study Type

Observational

Enrollment (Actual)

260

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Argentina
    • Chaco CPA
      • Reconquista, Chaco CPA, Argentina, H3500BJS
        • Clinica de Salud de Chaco
  • Canada
    • Nova Scotia
      • Halifax, Nova Scotia, Canada, B3H2Y9
        • Nova Scotia Health Authority
    • Ontario
      • London, Ontario, Canada, N6A5B7
        • Robarts Research Institute
    • Quebec
      • Chicoutimi, Quebec, Canada, G7H7K9
        • Ecogene-21
      • Montreal, Quebec, Canada, H4A3J1
        • McGill University
  • France
      • Strasbourg, France, 67000
        • Centre Hospitalier Universitaire Strasbourg
    • Cedex
      • Lille, Cedex, France, 59037
        • Centre Hospitalier Régional Universitaire de Lille
    • Lyon
      • Bron, Lyon, France, 69677
        • Centre Hospitalier Universitaire Lyon
  • Greece
      • Ioannina, Greece, 45500
        • University General Hospital of Ioannina
    • Piraeus
      • Piraeus, Piraeus, Greece, 18547
        • Metropolitan Hospital
  • Italy
      • Naples, Italy, 80131
        • Azienda Ospedaliera Universitaria Federico II
      • Naples, Italy, 80131
        • Azienda Ospedaliera Specialistica Dei Colli
    • BO
      • Bologna, BO, Italy, 40138
        • Policlinico S. Orsola-Malpighi
    • Bari
      • Bari, Bari, Italy, 70120
        • Azienda Ospedaliero Universi consorziale policlinico di Bari
    • CA
      • Cagliari, CA, Italy, 09134
        • Azienda Ospedaliera G. Brotzu
    • MI
      • Milan, MI, Italy, 20162
        • Asst Grande Ospedale Metropolitano Niguarda
    • PA
      • Palermo, PA, Italy, 90127
        • Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone
    • PD
      • Padova, PD, Italy, 35128
        • Azienda Ospedaliera Universita di Padova
    • Pisa
      • Pisa, Pisa, Italy, 56124
        • CNR Regione Toscana - Fondazione Toscana Gabriele Monasterio
    • RM
      • Roma, RM, Italy, 00161
        • Università degli Studi di Roma La Sapienza
    • Rome
      • Roma, Rome, Italy, 00161
        • Università degli Studi di Roma La Sapienza
    • TO
      • Orbassano, TO, Italy, 10043
        • Azienda Ospedaliero-Universitaria San Luigi Gonzaga
  • Netherlands
    • Gelderland
      • Nijmegen, Gelderland, Netherlands, 6525 GA
        • Radboud Universiteit Medisch Centrum
    • South Holland
      • Rotterdam, South Holland, Netherlands, 3015 GD
        • Erasmus Medisch Centrum
  • United Kingdom
    • London
      • London, London, United Kingdom, W12 0NN
        • Imperial College Healthcare - NHS Trust
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35209
        • Brookwood Baptist Medical Center
      • Mobile, Alabama, United States, 36608
        • Springhill Physician Practices
    • Arizona
      • Cottonwood, Arizona, United States, 86326
        • Northern Arizona Healthcare
      • Phoenix, Arizona, United States, 85018
        • Scottsdale Family Health
      • Tucson, Arizona, United States, 85718
        • Pima Heart
    • Arkansas
      • Fort Smith, Arkansas, United States, 72901
        • Arkansas Heart Center
    • California
      • Los Angeles, California, United States, 90048
        • Cedars-Sinai Medical Center
    • Colorado
      • Aurora, Colorado, United States, 80045
        • University of Colorado Health
    • Delaware
      • Newark, Delaware, United States, 19713
        • Alfieri Cardiology
    • Florida
      • Boca Raton, Florida, United States, 33434
        • Preventative Cardiology, Inc.
      • Winter Park, Florida, United States, 32792-2223
        • Florida Lipid Institute
    • Georgia
      • Atlanta, Georgia, United States, 30309
        • Piedmont Heart Institute
      • Cumming, Georgia, United States, 30041
        • Atlanta Heart Specialists
      • Cumming, Georgia, United States, 30041
        • Northside Hospital, Inc.
    • Illinois
      • Gurnee, Illinois, United States, 60031
        • Comprehensive Cardiovascular Care
    • Indiana
      • Indianapolis, Indiana, United States, 46290
        • St. Vincent Medical Group, Inc.
      • Indianapolis, Indiana, United States, 46250
        • Community Health Network
    • Kentucky
      • New Castle, Kentucky, United States, 40050
        • Henry County Medical Center
    • Louisiana
      • Houma, Louisiana, United States, 70360
        • Cardiovascular Institute of the South
    • Maryland
      • Towson, Maryland, United States, 21204
        • Get Well Immediate Care
    • Michigan
      • Sterling Heights, Michigan, United States, 48314
        • Complete Family Care Cholesterol Treatment Center
    • Minnesota
      • Minneapolis, Minnesota, United States, 55407
        • Minneapolis Heart Institute Foundation
      • Saint Paul, Minnesota, United States, 55102
        • HealthEast Care System
    • Mississippi
      • Southaven, Mississippi, United States, 38671
        • Stern Cardiovascular Foundation
    • Nebraska
      • Omaha, Nebraska, United States, 68114
        • Methodist Physicians Clinic
    • New Jersey
      • Rutherford, New Jersey, United States, 07070
        • 201 Route 17 North
      • Wyckoff, New Jersey, United States, 07481
        • Valley Medical Group
    • New York
      • New York, New York, United States, 10016
        • Murray Hill Medical Group
      • Syracuse, New York, United States, 13210
        • NY Heart Center
      • Syracuse, New York, United States, 13210
        • Pediatric Cardiology Associates
    • North Carolina
      • Charlotte, North Carolina, United States, 28204
        • Cardiology Specialists of the Carolinas PA
      • Charlotte, North Carolina, United States, 28211
        • Cardiology Specialists Of Carolina
    • Pennsylvania
      • Allentown, Pennsylvania, United States, 18103
        • The Heart Care Group, PC
      • Bethlehem, Pennsylvania, United States, 18015
        • St. Luke's University Health Network - Pennsylvania
      • East Stroudsburg, Pennsylvania, United States, 18301
        • Lehigh Valley Hospital And Health Network
      • Philadelphia, Pennsylvania, United States, 19107
        • Cardiology Consultants of Philadelphia
      • Philadelphia, Pennsylvania, United States, 19148
        • Thomas Jefferson University Health System
    • South Carolina
      • Columbia, South Carolina, United States, 29203
        • Palmetto Health
      • Greenville, South Carolina, United States, 29601
        • 317 Saint Francis Dr.
    • Texas
      • Austin, Texas, United States, 78758
        • Cardiovascular Specialists of Texas
      • Dallas, Texas, United States, 75226
        • Baylor Scott & White Health
    • Vermont
      • Burlington, Vermont, United States, 05401-1473
        • The University of Vermont Health Network
    • West Virginia
      • Morgantown, West Virginia, United States, 26506
        • West Virginia University Medicine
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Medical College of Wisconsin
      • Milwaukee, Wisconsin, United States, 53211
        • Ascension
      • Wausau, Wisconsin, United States, 54401
        • Aspirus Research Institute

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Patients treated with lomitapide who agree to participate in the study.

Description

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..

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Lomitapide
Lomitapide as prescribed by Physician.
Drug: Lomitapide
As prescribed by Physician.
Other Names:
  • Juxtapid
  • Lojuxta

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Hepatic Abnormalities
Time Frame: patients will be followed for 10 years
to evaluate the occurrence of hepatic abnormalities
patients will be followed for 10 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Gastrointestinal (GI) Events
Time Frame: patients will be followed for 10 years
to evaluate the occurrence of GI events
patients will be followed for 10 years
Tumors
Time Frame: patients will be followed for 10 years
to evaluate any occurrence of tumors (small bowel, hepatic, colorectal or pancreatic)
patients will be followed for 10 years
Events associated with coagulopathy
Time Frame: patients will be followed for 10 years
to evaluate the occurrence of events associated with coagulopathy (abnormal bleeding, cerebral haemorrhage or GI bleeding)
patients will be followed for 10 years
Major Adverse Cardiovascular Events (MACE) events
Time Frame: patients will be followed for 10 years
to evaluate the occurrence of MACE events
patients will be followed for 10 years
Death, including cause of death
Time Frame: patients will be followed for 10 years
to evaluate the occurrence and cause of death
patients will be followed for 10 years
Pregnancy
Time Frame: patients will be followed for 10 years
to evaluate the occurrence and outcomes of pregnancy in females of reproductive potential treated with lomitapide. Patients who become pregnant will be offered enrolment into a separate Pregnancy Exposure Registry (PER).
patients will be followed for 10 years
Serum lipid levels
Time Frame: patients will be followed for 10 years
to evaluate the long-term effectiveness of lomitapide in maintaining control of serum lipid levels in a clinical practice setting.
patients will be followed for 10 years
Prescriber behavior
Time Frame: patients will be followed for 10 years
to evaluate whether prescribers of lomitapide enrolled at registry sites are following the screening and monitoring recommendations as specified in the PI and the prescriber educational materials aimed at risk minimization.
patients will be followed for 10 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Amryt Pharma

Investigators

  • Study Director: Janet Boylan, Amryt Pharmaceuticals

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

March 18, 2014

Primary Completion (Actual)

April 27, 2026

Study Completion (Actual)

April 27, 2026

Study Registration Dates

First Submitted

April 10, 2014

First Submitted That Met QC Criteria

May 8, 2014

First Posted (Estimated)

May 12, 2014

Study Record Updates

Last Update Posted (Actual)

May 12, 2026

Last Update Submitted That Met QC Criteria

May 7, 2026

Last Verified

May 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AEGR-733-025

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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