Clinical trial NCT02135705: LOWER: Lomitapide Observational Worldwide Evaluation Registry
Official title: LOWER: Lomitapide Observational Worldwide Evaluation Registry
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Homozygous Familial Hypercholesterolemia
Lead sponsor: Amryt Pharma
Sponsor class: INDUSTRY
First posted: 2014-05-12
Enrollment: 300
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients treated with lomitapide who agree to participate in the study.

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.

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.

Eligibility criteria: 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..

Primary outcomes:
measure: Hepatic Abnormalities
timeFrame: patients will be followed for 10 years
Secondary outcomes:
measure: Gastrointestinal (GI) Events
timeFrame: patients will be followed for 10 years
measure: Tumors
timeFrame: patients will be followed for 10 years
measure: Events associated with coagulopathy
timeFrame: patients will be followed for 10 years
measure: Major Adverse Cardiovascular Events (MACE) events
timeFrame: patients will be followed for 10 years
measure: Death, including cause of death
timeFrame: patients will be followed for 10 years
measure: Pregnancy
timeFrame: patients will be followed for 10 years
measure: Serum lipid levels
timeFrame: patients will be followed for 10 years
measure: Prescriber behavior
timeFrame: patients will be followed for 10 years
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Brookwood Baptist Medical Center
state: Alabama
city: Mobile
country: United States
facility: Springhill Physician Practices
state: Alabama
city: Cottonwood
country: United States
facility: Northern Arizona Healthcare
state: Arizona
city: Gilbert
country: United States
facility: One Medical
state: Arizona
city: Phoenix
country: United States
facility: Scottsdale Family Health
state: Arizona
city: Tucson
country: United States
facility: Pima Heart
state: Arizona
city: Fort Smith
country: United States
facility: Arkansas Heart Center
state: Arkansas
city: Los Angeles
country: United States
facility: Cedars-Sinai Medical Center
state: California
city: Aurora
country: United States
facility: University of Colorado Health
state: Colorado
city: Newark
country: United States
facility: Alfieri Cardiology
state: Delaware
city: Boca Raton
country: United States
facility: Preventative Cardiology, Inc.
state: Florida
city: Winter Park
country: United States
facility: Florida Lipid Institute
state: Florida
city: Atlanta
country: United States
facility: Piedmont Heart Institute
state: Georgia
city: Cumming
country: United States
facility: Atlanta Heart Specialists
state: Georgia
city: Cumming
country: United States
facility: Northside Hospital, Inc.
state: Georgia
city: Gurnee
country: United States
facility: Comprehensive Cardiovascular Care
state: Illinois
city: Indianapolis
country: United States
facility: Community Health Network
state: Indiana
city: Indianapolis
country: United States
facility: St. Vincent Medical Group, Inc.
state: Indiana
city: New Castle
country: United States
facility: Henry County Medical Center
state: Kentucky
city: Houma
country: United States
facility: Cardiovascular Institute of the South
state: Louisiana
city: Towson
country: United States
facility: Get Well Immediate Care
state: Maryland
city: Ann Arbor
country: United States
facility: University of Michigan Health System
state: Michigan
city: Sterling Heights
country: United States
facility: Complete Family Care Cholesterol Treatment Center
state: Michigan
city: Minneapolis
country: United States
facility: Minneapolis Heart Institute Foundation
state: Minnesota
city: Saint Paul
country: United States
facility: HealthEast Care System
state: Minnesota
city: Southaven
country: United States
facility: Stern Cardiovascular Foundation
state: Mississippi
city: Omaha
country: United States
facility: Methodist Physicians Clinic
state: Nebraska
city: Edison
country: United States
facility: Hackensack Meridian Health
state: New Jersey
city: Lakewood
country: United States
facility: Avinash C. Gupta, MD, PC
state: New Jersey
city: Rutherford
country: United States
facility: 201 Route 17 North
state: New Jersey
city: Wyckoff
country: United States
facility: Valley Medical Group
state: New Jersey
city: New York
country: United States
facility: Murray Hill Medical Group
state: New York
city: Syracuse
country: United States
facility: NY Heart Center
state: New York
city: Syracuse
country: United States
facility: Pediatric Cardiology Associates
state: New York
city: Charlotte
country: United States
facility: Cardiology Specialists of the Carolinas PA
state: North Carolina
city: Charlotte
country: United States
facility: Cardiology Specialists Of Carolina
state: North Carolina
city: Allentown
country: United States
facility: The Heart Care Group, PC
state: Pennsylvania
city: Bethlehem
country: United States
facility: St. Luke's University Health Network - Pennsylvania
state: Pennsylvania
city: East Stroudsburg
country: United States
facility: Lehigh Valley Hospital And Health Network
state: Pennsylvania
city: Philadelphia
country: United States
facility: Cardiology Consultants of Philadelphia
state: Pennsylvania
city: Philadelphia
country: United States
facility: Thomas Jefferson University Health System
state: Pennsylvania
city: Columbia
country: United States
facility: Palmetto Health
state: South Carolina
city: Greenville
country: United States
facility: 317 Saint Francis Dr.
state: South Carolina
city: Austin
country: United States
facility: Cardiovascular Specialists of Texas
state: Texas
city: Dallas
country: United States
facility: Baylor Scott & White Health
state: Texas
city: Burlington
country: United States
facility: The University of Vermont Health Network
state: Vermont
city: Morgantown
country: United States
facility: West Virginia University Medicine
state: West Virginia
city: Milwaukee
country: United States
facility: Ascension
state: Wisconsin
city: Milwaukee
country: United States
facility: Medical College of Wisconsin
state: Wisconsin
city: Wausau
country: United States
facility: Aspirus Research Institute
state: Wisconsin
city: Reconquista
country: Argentina
facility: Clinica de Salud de Chaco
state: Chaco CPA
city: Halifax
country: Canada
facility: Nova Scotia Health Authority
state: Nova Scotia
city: London
country: Canada
facility: Robarts Research Institute
state: Ontario
city: Chicoutimi
country: Canada
facility: Ecogene-21
state: Quebec
city: Montréal
country: Canada
facility: McGill University
state: Quebec
city: Lille
country: France
facility: Centre Hospitalier Régional Universitaire de Lille
state: Cedex
city: Bron
country: France
facility: Centre Hospitalier Universitaire Lyon
state: Lyon
city: Strasbourg
country: France
facility: Centre Hospitalier Universitaire Strasbourg
city: Ioánnina
country: Greece
facility: University General Hospital of Ioannina
city: Piraeus
country: Greece
facility: Metropolitan Hospital
city: Bologna
country: Italy
facility: Policlinico S. Orsola-Malpighi
state: BO
city: Cagliari
country: Italy
facility: Azienda Ospedaliera G. Brotzu
state: CA
city: Milano
country: Italy
facility: ASST Grande Ospedale Metropolitano Niguarda
state: MI
city: Palermo
country: Italy
facility: Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone
state: PA
city: Padova
country: Italy
facility: Azienda Ospedaliera Universita di Padova
state: PD
city: Roma
country: Italy
facility: Università degli Studi di Roma La Sapienza
state: RM
city: Roma
country: Italy
facility: Università degli Studi di Roma La Sapienza
state: Rome
city: Orbassano
country: Italy
facility: Azienda Ospedaliero-Universitaria San Luigi Gonzaga
state: TO
city: Bari
country: Italy
facility: Azienda Ospedaliero Universi consorziale policlinico di Bari
city: Napoli
country: Italy
facility: Azienda Ospedaliera Specialistica Dei Colli
city: Napoli
country: Italy
facility: Azienda Ospedaliera Universitaria Federico II
city: Pisa
country: Italy
facility: CNR Regione Toscana - Fondazione Toscana Gabriele Monasterio
city: Nijmegen
country: Netherlands
facility: Radboud Universiteit Medisch Centrum
state: Gelderland
city: Rotterdam
country: Netherlands
facility: Erasmus Medisch Centrum
state: Zuid Holland
city: London
country: United Kingdom
facility: Imperial College Healthcare - NHS Trust
Study officials:
affiliation: Amryt Pharmaceuticals
name: Janet Boylan
role: STUDY_DIRECTOR
References:
citation: 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.