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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Lomitapide | Lomitapide as prescribed by Physician. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Lomitapide | Drug | As prescribed by Physician. |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Hepatic Abnormalities | to evaluate the occurrence of hepatic abnormalities | patients will be followed for 10 years |
| Measure | Description | Time Frame |
|---|---|---|
| Gastrointestinal (GI) Events | to evaluate the occurrence of GI events | patients will be followed for 10 years |
| Tumors | to evaluate any occurrence of tumors (small bowel, hepatic, colorectal or pancreatic) |
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Inclusion Criteria:
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:
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Patients treated with lomitapide who agree to participate in the study.
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| Name | Affiliation | Role |
|---|---|---|
| Janet Boylan | Amryt Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Brookwood Baptist Medical Center | Birmingham | Alabama | 35209 | United States | ||
| Springhill Physician Practices |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 36520008 | Derived | 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. | |
| 30663562 | 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. |
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| patients will be followed for 10 years |
| 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 |
| Major Adverse Cardiovascular Events (MACE) events | to evaluate the occurrence of MACE events | patients will be followed for 10 years |
| Death, including cause of death | to evaluate the occurrence and cause of death | patients will be followed for 10 years |
| 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 |
| 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 |
| 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 |
| Mobile |
| Alabama |
| 36608 |
| United States |
| Northern Arizona Healthcare | Cottonwood | Arizona | 86326 | United States |
| Scottsdale Family Health | Phoenix | Arizona | 85018 | United States |
| Pima Heart | Tucson | Arizona | 85718 | United States |
| Arkansas Heart Center | Fort Smith | Arkansas | 72901 | United States |
| Cedars-Sinai Medical Center | Los Angeles | California | 90048 | United States |
| University of Colorado Health | Aurora | Colorado | 80045 | United States |
| Alfieri Cardiology | Newark | Delaware | 19713 | United States |
| Preventative Cardiology, Inc. | Boca Raton | Florida | 33434 | United States |
| Florida Lipid Institute | Winter Park | Florida | 32792-2223 | United States |
| Piedmont Heart Institute | Atlanta | Georgia | 30309 | United States |
| Atlanta Heart Specialists | Cumming | Georgia | 30041 | United States |
| Northside Hospital, Inc. | Cumming | Georgia | 30041 | United States |
| Comprehensive Cardiovascular Care | Gurnee | Illinois | 60031 | United States |
| Community Health Network | Indianapolis | Indiana | 46250 | United States |
| St. Vincent Medical Group, Inc. | Indianapolis | Indiana | 46290 | United States |
| Henry County Medical Center | New Castle | Kentucky | 40050 | United States |
| Cardiovascular Institute of the South | Houma | Louisiana | 70360 | United States |
| Get Well Immediate Care | Towson | Maryland | 21204 | United States |
| Complete Family Care Cholesterol Treatment Center | Sterling Heights | Michigan | 48314 | United States |
| Minneapolis Heart Institute Foundation | Minneapolis | Minnesota | 55407 | United States |
| HealthEast Care System | Saint Paul | Minnesota | 55102 | United States |
| Stern Cardiovascular Foundation | Southaven | Mississippi | 38671 | United States |
| Methodist Physicians Clinic | Omaha | Nebraska | 68114 | United States |
| 201 Route 17 North | Rutherford | New Jersey | 07070 | United States |
| Valley Medical Group | Wyckoff | New Jersey | 07481 | United States |
| Murray Hill Medical Group | New York | New York | 10016 | United States |
| NY Heart Center | Syracuse | New York | 13210 | United States |
| Pediatric Cardiology Associates | Syracuse | New York | 13210 | United States |
| Cardiology Specialists of the Carolinas PA | Charlotte | North Carolina | 28204 | United States |
| Cardiology Specialists Of Carolina | Charlotte | North Carolina | 28211 | United States |
| The Heart Care Group, PC | Allentown | Pennsylvania | 18103 | United States |
| St. Luke's University Health Network - Pennsylvania | Bethlehem | Pennsylvania | 18015 | United States |
| Lehigh Valley Hospital And Health Network | East Stroudsburg | Pennsylvania | 18301 | United States |
| Cardiology Consultants of Philadelphia | Philadelphia | Pennsylvania | 19107 | United States |
| Thomas Jefferson University Health System | Philadelphia | Pennsylvania | 19148 | United States |
| Palmetto Health | Columbia | South Carolina | 29203 | United States |
| 317 Saint Francis Dr. | Greenville | South Carolina | 29601 | United States |
| Cardiovascular Specialists of Texas | Austin | Texas | 78758 | United States |
| Baylor Scott & White Health | Dallas | Texas | 75226 | United States |
| The University of Vermont Health Network | Burlington | Vermont | 05401-1473 | United States |
| West Virginia University Medicine | Morgantown | West Virginia | 26506 | United States |
| Ascension | Milwaukee | Wisconsin | 53211 | United States |
| Medical College of Wisconsin | Milwaukee | Wisconsin | 53226 | United States |
| Aspirus Research Institute | Wausau | Wisconsin | 54401 | United States |
| Clinica de Salud de Chaco | Reconquista | Chaco CPA | H3500BJS | Argentina |
| Nova Scotia Health Authority | Halifax | Nova Scotia | B3H2Y9 | Canada |
| Robarts Research Institute | London | Ontario | N6A5B7 | Canada |
| Ecogene-21 | Chicoutimi | Quebec | G7H7K9 | Canada |
| McGill University | Montreal | Quebec | H4A3J1 | Canada |
| Centre Hospitalier Régional Universitaire de Lille | Lille | Cedex | 59037 | France |
| Centre Hospitalier Universitaire Lyon | Bron | Lyon | 69677 | France |
| Centre Hospitalier Universitaire Strasbourg | Strasbourg | 67000 | France |
| Metropolitan Hospital | Piraeus | Piraeus | 18547 | Greece |
| University General Hospital of Ioannina | Ioannina | 45500 | Greece |
| Azienda Ospedaliero Universi consorziale policlinico di Bari | Bari | Bari | 70120 | Italy |
| Policlinico S. Orsola-Malpighi | Bologna | BO | 40138 | Italy |
| Azienda Ospedaliera G. Brotzu | Cagliari | CA | 09134 | Italy |
| ASST Grande Ospedale Metropolitano Niguarda | Milan | MI | 20162 | Italy |
| Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone | Palermo | PA | 90127 | Italy |
| Azienda Ospedaliera Universita di Padova | Padova | PD | 35128 | Italy |
| CNR Regione Toscana - Fondazione Toscana Gabriele Monasterio | Pisa | Pisa | 56124 | Italy |
| Università degli Studi di Roma La Sapienza | Roma | RM | 00161 | Italy |
| Università degli Studi di Roma La Sapienza | Roma | Rome | 00161 | Italy |
| Azienda Ospedaliero-Universitaria San Luigi Gonzaga | Orbassano | TO | 10043 | Italy |
| Azienda Ospedaliera Specialistica Dei Colli | Naples | 80131 | Italy |
| Azienda Ospedaliera Universitaria Federico II | Naples | 80131 | Italy |
| Radboud Universiteit Medisch Centrum | Nijmegen | Gelderland | 6525 GA | Netherlands |
| Erasmus Medisch Centrum | Rotterdam | South Holland | 3015 GD | Netherlands |
| Imperial College Healthcare - NHS Trust | London | London | W12 0NN | United Kingdom |
| ID | Term |
|---|---|
| D000090542 | Homozygous Familial Hypercholesterolemia |
| ID | Term |
|---|---|
| D006938 | Hyperlipoproteinemia Type II |
| D008052 | Lipid Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D006951 | Hyperlipoproteinemias |
| D006949 | Hyperlipidemias |
| D050171 | Dyslipidemias |
| D052439 | Lipid Metabolism Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
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| ID | Term |
|---|---|
| C473731 | BMS201038 |
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