LOWER: Lomitapide Observational Worldwide Evaluation Registry

Homozygous Familial Hypercholesterolemia Clinical Trial

— LOWER  

Official title:

LOWER: Lomitapide Observational Worldwide Evaluation Registry

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT02135705
• Other study ID #: AEGR-733-025
• Status: Recruiting
• Start date: March 18, 2014
• Est. completion date: September 1, 2028

Study information

• Verified date: March 2024
• Source: Amryt Pharma
• Contact: Janet Boylan
• Phone: 1 855 303 2347
• Email: Janet.Boylan[@]AmrytPharma.com
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 300
• Est. completion date: September 1, 2028
• Est. primary completion date: March 1, 2028
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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..

Related Conditions & MeSH terms

• Homozygous Familial Hypercholesterolemia
• Hypercholesterolemia
• Hyperlipoproteinemia Type II

Intervention

Drug:
• Lomitapide
As prescribed by Physician.

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
Amryt Pharma

Countries where clinical trial is conducted

United States,  Argentina,  Canada,  France,  Greece,  Italy,  Netherlands,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Hepatic Abnormalities	to evaluate the occurrence of hepatic abnormalities	patients will be followed for 10 years
Secondary	Gastrointestinal (GI) Events	to evaluate the occurrence of GI events	patients will be followed for 10 years
Secondary	Tumors	to evaluate any occurrence of tumors (small bowel, hepatic, colorectal or pancreatic)	patients will be followed for 10 years
Secondary	Events associated with coagulopathy	to evaluate the occurrence of events associated with coagulopathy (abnormal bleeding, cerebral haemorrhage or GI bleeding)	patients will be followed for 10 years
Secondary	Major Adverse Cardiovascular Events (MACE) events	to evaluate the occurrence of MACE events	patients will be followed for 10 years
Secondary	Death, including cause of death	to evaluate the occurrence and cause of death	patients will be followed for 10 years
Secondary	Pregnancy	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
Secondary	Serum lipid levels	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
Secondary	Prescriber behavior	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