LOWER: Lomitapide Observational Worldwide Evaluation Registry

NCT02135705 | Recruiting

• 1 other identifier: AEGR-733-025
• Study Type: observational
• Target: 300
• Locations: 8 countries
• Sites: 75

Brief Summary

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.

Conditions

Homozygous Familial Hypercholesterolemia

Outcome Measures

Primary Outcomes (1)

• Hepatic Abnormalities

  to evaluate the occurrence of hepatic abnormalities

  patients will be followed for 10 years

Secondary Outcomes (8)

• Gastrointestinal (GI) Events

  patients will be followed for 10 years

• Tumors

  patients will be followed for 10 years

• Events associated with coagulopathy

  patients will be followed for 10 years

• Major Adverse Cardiovascular Events (MACE) events

  patients will be followed for 10 years

• Death, including cause of death

  patients will be followed for 10 years

Study Arms (1)

Lomitapide

Lomitapide as prescribed by Physician.

Drug: Lomitapide

Interventions

Lomitapide DRUG

As prescribed by Physician.

Also known as: Juxtapid, Lojuxta

Lomitapide

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

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

You may qualify if:

• 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.

You may not qualify if:

• 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..

Sponsors & Collaborators

• Amryt Pharma: lead

Study Sites (75)

Brookwood Baptist Medical Center

Birmingham, Alabama, 35209, United States

RECRUITING

Springhill Physician Practices

Mobile, Alabama, 36608, United States

TERMINATED

Northern Arizona Healthcare

Cottonwood, Arizona, 86326, United States

RECRUITING

One Medical

Gilbert, Arizona, 85296, United States

WITHDRAWN

Scottsdale Family Health

Phoenix, Arizona, 85018, United States

RECRUITING

Pima Heart

Tucson, Arizona, 85718, United States

TERMINATED

Arkansas Heart Center

Fort Smith, Arkansas, 72901, United States

TERMINATED

Cedars-Sinai Medical Center

Los Angeles, California, 90048, United States

TERMINATED

University of Colorado Health

Aurora, Colorado, 80045, United States

TERMINATED

Alfieri Cardiology

Newark, Delaware, 19713, United States

TERMINATED

Preventative Cardiology, Inc.

Boca Raton, Florida, 33434, United States

RECRUITING

Florida Lipid Institute

Winter Park, Florida, 32792-2223, United States

TERMINATED

Piedmont Heart Institute

Atlanta, Georgia, 30309, United States

RECRUITING

Atlanta Heart Specialists

Cumming, Georgia, 30041, United States

RECRUITING

Northside Hospital, Inc.

Cumming, Georgia, 30041, United States

RECRUITING

Comprehensive Cardiovascular Care

Gurnee, Illinois, 60031, United States

TERMINATED

Community Health Network

Indianapolis, Indiana, 46250, United States

TERMINATED

St. Vincent Medical Group, Inc.

Indianapolis, Indiana, 46290, United States

RECRUITING

Henry County Medical Center

New Castle, Kentucky, 40050, United States

TERMINATED

Cardiovascular Institute of the South

Houma, Louisiana, 70360, United States

RECRUITING

Get Well Immediate Care

Towson, Maryland, 21204, United States

TERMINATED

University of Michigan Health System

Ann Arbor, Michigan, 48109, United States

WITHDRAWN

Complete Family Care Cholesterol Treatment Center

Sterling Heights, Michigan, 48314, United States

TERMINATED

Minneapolis Heart Institute Foundation

Minneapolis, Minnesota, 55407, United States

RECRUITING

HealthEast Care System

Saint Paul, Minnesota, 55102, United States

TERMINATED

Stern Cardiovascular Foundation

Southaven, Mississippi, 38671, United States

RECRUITING

Methodist Physicians Clinic

Omaha, Nebraska, 68114, United States

RECRUITING

Hackensack Meridian Health

Edison, New Jersey, 08817, United States

WITHDRAWN

Avinash C. Gupta, MD, PC

Lakewood, New Jersey, 08701, United States

WITHDRAWN

201 Route 17 North

Rutherford, New Jersey, 07070, United States

RECRUITING

Valley Medical Group

Wyckoff, New Jersey, 07481, United States

TERMINATED

Murray Hill Medical Group

New York, New York, 10016, United States

RECRUITING

NY Heart Center

Syracuse, New York, 13210, United States

TERMINATED

Pediatric Cardiology Associates

Syracuse, New York, 13210, United States

RECRUITING

Cardiology Specialists of the Carolinas PA

Charlotte, North Carolina, 28204, United States

RECRUITING

Cardiology Specialists Of Carolina

Charlotte, North Carolina, 28211, United States

RECRUITING

The Heart Care Group, PC

Allentown, Pennsylvania, 18103, United States

TERMINATED

St. Luke's University Health Network - Pennsylvania

Bethlehem, Pennsylvania, 18015, United States

TERMINATED

Lehigh Valley Hospital And Health Network

East Stroudsburg, Pennsylvania, 18301, United States

TERMINATED

Cardiology Consultants of Philadelphia

Philadelphia, Pennsylvania, 19107, United States

TERMINATED

Thomas Jefferson University Health System

Philadelphia, Pennsylvania, 19148, United States

RECRUITING

Palmetto Health

Columbia, South Carolina, 29203, United States

TERMINATED

317 Saint Francis Dr.

Greenville, South Carolina, 29601, United States

RECRUITING

Cardiovascular Specialists of Texas

Austin, Texas, 78758, United States

TERMINATED

Baylor Scott & White Health

Dallas, Texas, 75226, United States

TERMINATED

The University of Vermont Health Network

Burlington, Vermont, 05401-1473, United States

RECRUITING

West Virginia University Medicine

Morgantown, West Virginia, 26506, United States

RECRUITING

Ascension

Milwaukee, Wisconsin, 53211, United States

TERMINATED

Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States

TERMINATED

Aspirus Research Institute

Wausau, Wisconsin, 54401, United States

TERMINATED

Clinica de Salud de Chaco

Reconquista, Chaco CPA, H3500BJS, Argentina

RECRUITING

Nova Scotia Health Authority

Halifax, Nova Scotia, B3H2Y9, Canada

RECRUITING

Robarts Research Institute

London, Ontario, N6A5B7, Canada

RECRUITING

Ecogene-21

Chicoutimi, Quebec, G7H7K9, Canada

RECRUITING

McGill University

Montreal, Quebec, H4A3J1, Canada

RECRUITING

Centre Hospitalier Régional Universitaire de Lille

Lille, Cedex, 59037, France

RECRUITING

Centre Hospitalier Universitaire Lyon

Bron, Lyon, 69677, France

RECRUITING

Centre Hospitalier Universitaire Strasbourg

Strasbourg, 67000, France

RECRUITING

University General Hospital of Ioannina

Ioannina, 45500, Greece

RECRUITING

Metropolitan Hospital

Piraeus, 18547, Greece

RECRUITING

Policlinico S. Orsola-Malpighi

Bologna, BO, 40138, Italy

RECRUITING

Azienda Ospedaliera G. Brotzu

Cagliari, CA, 09134, Italy

RECRUITING

ASST Grande Ospedale Metropolitano Niguarda

Milan, MI, 20162, Italy

RECRUITING

Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone

Palermo, PA, 90127, Italy

RECRUITING

Azienda Ospedaliera Universita di Padova

Padua, PD, 35128, Italy

RECRUITING

Università degli Studi di Roma La Sapienza

Roma, RM, 00161, Italy

RECRUITING

Università degli Studi di Roma La Sapienza

Roma, Rome, 00161, Italy

TERMINATED

Azienda Ospedaliero-Universitaria San Luigi Gonzaga

Orbassano, TO, 10043, Italy

RECRUITING

Azienda Ospedaliero Universi consorziale policlinico di Bari

Bari, 70120, Italy

RECRUITING

Azienda Ospedaliera Specialistica Dei Colli

Napoli, 80131, Italy

RECRUITING

Azienda Ospedaliera Universitaria Federico II

Napoli, 80131, Italy

RECRUITING

CNR Regione Toscana - Fondazione Toscana Gabriele Monasterio

Pisa, 56124, Italy

RECRUITING

Radboud Universiteit Medisch Centrum

Nijmegen, Gelderland, 6525 GA, Netherlands

RECRUITING

Erasmus Medisch Centrum

Rotterdam, South Holland, 3015 GD, Netherlands

RECRUITING

Imperial College Healthcare - NHS Trust

London, W12 0NN, United Kingdom

RECRUITING

Related Publications (2)

• Larrey D, D'Erasmo L, O'Brien S, Arca M; Italian Working Group on Lomitapide. Long-term hepatic safety of lomitapide in homozygous familial hypercholesterolaemia. Liver Int. 2023 Feb;43(2):413-423. doi: 10.1111/liv.15497. Epub 2022 Dec 30.

  PMID: 36520008 DERIVED

• Giammanco A, Cefalu AB, Noto D, Averna MR. Therapeutic Options for Homozygous Familial Hypercholesterolemia: The Role of Lomitapide. Curr Med Chem. 2020;27(23):3773-3783. doi: 10.2174/0929867326666190121120735.

  PMID: 30663562 DERIVED

MeSH Terms

Conditions

Homozygous Familial Hypercholesterolemia

Interventions

BMS201038

Condition Hierarchy (Ancestors)

Hyperlipoproteinemia Type II; Lipid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hyperlipoproteinemias; Hyperlipidemias; Dyslipidemias; Lipid Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Janet Boylan

  Amryt Pharmaceuticals

  STUDY DIRECTOR

Central Study Contacts

Janet Boylan

CONTACT

1 855 303 2347; Janet.Boylan@AmrytPharma.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 10 Years
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 10, 2014
• First Posted: May 12, 2014
• Study Start: March 18, 2014
• Primary Completion (Estimated): March 1, 2028
• Study Completion (Estimated): September 1, 2028
• Last Updated: March 12, 2024

Record last verified: 2024-03

Locations

AR (1); NL (2); FR (3); IT (12); GR (2); CA (4); US (50); GB (1)