LOWER: Lomitapide Observational Worldwide Evaluation Registry

NCT ID: NCT02135705

Last Updated: 2024-03-12

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

300 participants

Study Classification

OBSERVATIONAL

Study Start Date

2014-03-18

Study Completion Date

2028-09-01

Brief Summary

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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.

Detailed Description

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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.

Conditions

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Homozygous Familial Hypercholesterolemia

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Lomitapide

Lomitapide as prescribed by Physician.

Lomitapide

Intervention Type DRUG

As prescribed by Physician.

Interventions

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Lomitapide

As prescribed by Physician.

Intervention Type DRUG

Other Intervention Names

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Juxtapid Lojuxta

Eligibility Criteria

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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..
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Amryt Pharma

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Janet Boylan

Role: STUDY_DIRECTOR

Amryt Pharmaceuticals

Locations

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Brookwood Baptist Medical Center

Birmingham, Alabama, United States

Site Status RECRUITING

Springhill Physician Practices

Mobile, Alabama, United States

Site Status TERMINATED

Northern Arizona Healthcare

Cottonwood, Arizona, United States

Site Status RECRUITING

One Medical

Gilbert, Arizona, United States

Site Status WITHDRAWN

Scottsdale Family Health

Phoenix, Arizona, United States

Site Status RECRUITING

Pima Heart

Tucson, Arizona, United States

Site Status TERMINATED

Arkansas Heart Center

Fort Smith, Arkansas, United States

Site Status TERMINATED

Cedars-Sinai Medical Center

Los Angeles, California, United States

Site Status TERMINATED

University of Colorado Health

Aurora, Colorado, United States

Site Status TERMINATED

Alfieri Cardiology

Newark, Delaware, United States

Site Status TERMINATED

Preventative Cardiology, Inc.

Boca Raton, Florida, United States

Site Status RECRUITING

Florida Lipid Institute

Winter Park, Florida, United States

Site Status TERMINATED

Piedmont Heart Institute

Atlanta, Georgia, United States

Site Status RECRUITING

Atlanta Heart Specialists

Cumming, Georgia, United States

Site Status RECRUITING

Northside Hospital, Inc.

Cumming, Georgia, United States

Site Status RECRUITING

Comprehensive Cardiovascular Care

Gurnee, Illinois, United States

Site Status TERMINATED

Community Health Network

Indianapolis, Indiana, United States

Site Status TERMINATED

St. Vincent Medical Group, Inc.

Indianapolis, Indiana, United States

Site Status RECRUITING

Henry County Medical Center

New Castle, Kentucky, United States

Site Status TERMINATED

Cardiovascular Institute of the South

Houma, Louisiana, United States

Site Status RECRUITING

Get Well Immediate Care

Towson, Maryland, United States

Site Status TERMINATED

University of Michigan Health System

Ann Arbor, Michigan, United States

Site Status WITHDRAWN

Complete Family Care Cholesterol Treatment Center

Sterling Heights, Michigan, United States

Site Status TERMINATED

Minneapolis Heart Institute Foundation

Minneapolis, Minnesota, United States

Site Status RECRUITING

HealthEast Care System

Saint Paul, Minnesota, United States

Site Status TERMINATED

Stern Cardiovascular Foundation

Southaven, Mississippi, United States

Site Status RECRUITING

Methodist Physicians Clinic

Omaha, Nebraska, United States

Site Status RECRUITING

Hackensack Meridian Health

Edison, New Jersey, United States

Site Status WITHDRAWN

Avinash C. Gupta, MD, PC

Lakewood, New Jersey, United States

Site Status WITHDRAWN

201 Route 17 North

Rutherford, New Jersey, United States

Site Status RECRUITING

Valley Medical Group

Wyckoff, New Jersey, United States

Site Status TERMINATED

Murray Hill Medical Group

New York, New York, United States

Site Status RECRUITING

NY Heart Center

Syracuse, New York, United States

Site Status TERMINATED

Pediatric Cardiology Associates

Syracuse, New York, United States

Site Status RECRUITING

Cardiology Specialists of the Carolinas PA

Charlotte, North Carolina, United States

Site Status RECRUITING

Cardiology Specialists Of Carolina

Charlotte, North Carolina, United States

Site Status RECRUITING

The Heart Care Group, PC

Allentown, Pennsylvania, United States

Site Status TERMINATED

St. Luke's University Health Network - Pennsylvania

Bethlehem, Pennsylvania, United States

Site Status TERMINATED

Lehigh Valley Hospital And Health Network

East Stroudsburg, Pennsylvania, United States

Site Status TERMINATED

Cardiology Consultants of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status TERMINATED

Thomas Jefferson University Health System

Philadelphia, Pennsylvania, United States

Site Status RECRUITING

Palmetto Health

Columbia, South Carolina, United States

Site Status TERMINATED

317 Saint Francis Dr.

Greenville, South Carolina, United States

Site Status RECRUITING

Cardiovascular Specialists of Texas

Austin, Texas, United States

Site Status TERMINATED

Baylor Scott & White Health

Dallas, Texas, United States

Site Status TERMINATED

The University of Vermont Health Network

Burlington, Vermont, United States

Site Status RECRUITING

West Virginia University Medicine

Morgantown, West Virginia, United States

Site Status RECRUITING

Ascension

Milwaukee, Wisconsin, United States

Site Status TERMINATED

Medical College of Wisconsin

Milwaukee, Wisconsin, United States

Site Status TERMINATED

Aspirus Research Institute

Wausau, Wisconsin, United States

Site Status TERMINATED

Clinica de Salud de Chaco

Reconquista, Chaco CPA, Argentina

Site Status RECRUITING

Nova Scotia Health Authority

Halifax, Nova Scotia, Canada

Site Status RECRUITING

Robarts Research Institute

London, Ontario, Canada

Site Status RECRUITING

Ecogene-21

Chicoutimi, Quebec, Canada

Site Status RECRUITING

McGill University

Montreal, Quebec, Canada

Site Status RECRUITING

Centre Hospitalier Régional Universitaire de Lille

Lille, Cedex, France

Site Status RECRUITING

Centre Hospitalier Universitaire Lyon

Bron, Lyon, France

Site Status RECRUITING

Centre Hospitalier Universitaire Strasbourg

Strasbourg, , France

Site Status RECRUITING

University General Hospital of Ioannina

Ioannina, , Greece

Site Status RECRUITING

Metropolitan Hospital

Piraeus, , Greece

Site Status RECRUITING

Policlinico S. Orsola-Malpighi

Bologna, BO, Italy

Site Status RECRUITING

Azienda Ospedaliera G. Brotzu

Cagliari, CA, Italy

Site Status RECRUITING

ASST Grande Ospedale Metropolitano Niguarda

Milan, MI, Italy

Site Status RECRUITING

Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone

Palermo, PA, Italy

Site Status RECRUITING

Azienda Ospedaliera Universita di Padova

Padua, PD, Italy

Site Status RECRUITING

Università degli Studi di Roma La Sapienza

Roma, RM, Italy

Site Status RECRUITING

Università degli Studi di Roma La Sapienza

Roma, Rome, Italy

Site Status TERMINATED

Azienda Ospedaliero-Universitaria San Luigi Gonzaga

Orbassano, TO, Italy

Site Status RECRUITING

Azienda Ospedaliero Universi consorziale policlinico di Bari

Bari, , Italy

Site Status RECRUITING

Azienda Ospedaliera Specialistica Dei Colli

Napoli, , Italy

Site Status RECRUITING

Azienda Ospedaliera Universitaria Federico II

Napoli, , Italy

Site Status RECRUITING

CNR Regione Toscana - Fondazione Toscana Gabriele Monasterio

Pisa, , Italy

Site Status RECRUITING

Radboud Universiteit Medisch Centrum

Nijmegen, Gelderland, Netherlands

Site Status RECRUITING

Erasmus Medisch Centrum

Rotterdam, South Holland, Netherlands

Site Status RECRUITING

Imperial College Healthcare - NHS Trust

London, , United Kingdom

Site Status RECRUITING

Countries

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United States Argentina Canada France Greece Italy Netherlands United Kingdom

Central Contacts

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Janet Boylan

Role: CONTACT

1 855 303 2347

Facility Contacts

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Barry McLean, MD

Role: primary

205-949-1907

Kenneth Bescak, MD

Role: primary

928-634-1331

Joseph Lillo, DO

Role: primary

480-941-4845

Seth Baum, MD

Role: primary

561-488-5535

Janice McAlister, NP

Role: primary

404-605-2800

Narendra Singh, MD

Role: primary

404-384-7227

David Song, MD

Role: primary

678-679-1065

James Trippi, MD

Role: primary

317-924-5444

Peter Fail, MD

Role: primary

985-876-0300

Thomas Knickelbine, MD

Role: primary

612-863-4071

Dharmesh Patel, MD

Role: primary

901-271-5468

John Henry, MD

Role: primary

Role: backup

(402) 384-2705

Michael Schwarz, MD

Role: primary

732-713-2872

James Underberg, MD

Role: primary

212-726-7431

Christopher Prendergast, MD

Role: primary

315-214-7700

John Pasquini, MD

Role: primary

704-343-9800

Daniel Wise, MD

Role: primary

704-369-8080

David Whellan, MD

Role: primary

215-503-4282

Jeffrey Siegel, MD

Role: primary

864-232-8296

David Schneider, MD

Role: primary

802-847-3734

Anthony Morise, MD

Role: primary

304-598-4651

Dario Gomez, MD

Role: primary

(54)(9362) 453-6261

Thomas Ransom, MD

Role: primary

(902) 473-3712

Robert Hegele, MD

Role: primary

(519) 931-5271

Daniel Gaudet, MD

Role: primary

(418) 541-1077

Jacques Genest, MD

Role: primary

(514) 934-1934

Cecile Yelnik, MD

Role: primary

33 (0) 3 20 44 59 62

Philippe Moulin, MD

Role: primary

33 04 27 85 66 66

Alain Pradignac, MD

Role: primary

+33 3 88 12 80 00

Georgis Liamis, MD

Role: primary

(+30) 265 1099 111

Genovefa Kolovou, MD

Role: primary

00306936886561

Sergio D Addato, MD

Role: primary

+39 051 2142224

Paolo Medde, MD

Role: primary

+39 070 539329

Antonia Alberti, MD

Role: primary

+39 02 64445803

Maurizio Averna, MD

Role: primary

+39 0916552993

Sabina Zambon, MD

Role: primary

+39 049 8212171

Marcello Arca, MD

Role: primary

+39 06 499 74692

Franca Napoli, MD

Role: primary

(+39) 011 90261

Patrizia Suppresa, MD

Role: primary

(+39) 0805592773

Paolo Calabro, MD

Role: primary

+39 0823232395

Gabriella Iannuzzo, MD

Role: primary

+39 3385498597

Francesco Sbrana, MD

Role: primary

+39 0503152705

Joost Rutten, MD

Role: primary

(+31) 24 361 1111

Jeanine Roeters van Lennep, MD

Role: primary

0031 652313132

Jaimini Cegla, MD

Role: primary

(+44) 077 7555 7295

References

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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.

Reference Type DERIVED
PMID: 36520008 (View on PubMed)

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.

Reference Type DERIVED
PMID: 30663562 (View on PubMed)

Other Identifiers

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AEGR-733-025

Identifier Type: -

Identifier Source: org_study_id

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