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

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
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 Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Lomitapide
Lomitapide as prescribed by Physician.
Drug: Lomitapide
As prescribed by Physician.
Other Names:
  • Juxtapid
  • Lojuxta

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
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

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Amryt Pharma

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • 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)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
March 18, 2014

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
April 27, 2026

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
April 27, 2026

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
April 10, 2014

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 8, 2014

First Posted (Estimated)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
May 12, 2014

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
May 12, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 7, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
May 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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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