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| Name | Class |
|---|---|
| Ultragenyx Pharmaceutical Inc | INDUSTRY |
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This is an open-label study designed to evaluate the long-term safety and efficacy of evinacumab, a fully human ANGPTL3 antibody, in patients with homozygous familial hypercholesterolemia (HoFH), in a real-life setting in Canada.
Eligible patients for this study are male and female adult patients with HoFH. Evinacumab will be added on top of the patient's background lipid-modifying therapy (LMT), including statins, ezetimibe, PCSK9 inhibitors, lomitapide or other lipid lowering therapies. This study will be conducted using an hybrid (on-site, foldable sites) approach. Patients will enter the current study, in an open-label treatment period, following their screening. This study will continue until reimbursement of evinacumab in Canada or for a maximum of 24 months. The end of study (EoS) visit will be scheduled 4 weeks after the last dose has been injected and will be followed by a 52-week follow-up.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Evinacumab-treated patients | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Evinacumab | Drug | Evinacumab 15 mg/kg administered intravenously every 4 weeks |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change in lipid profile | Change in total cholesterol, LDL-Cholesterol, HDL-Cholesterol, triglyceride, non-HDL-Cholesterol and apolipoprotein B plasma concentration | Every 4 weeks up to 2 years |
| Change in aspartate transaminase (AST) plasma concentration | Every 4 weeks up to 2 years | |
| change in alanine aminotransferase (ALT) plasma concentration | Every 4 weeks up to 2 years |
| Measure | Description | Time Frame |
|---|---|---|
| Lipoprotein (a) | Yearly up to 2 years | |
| Complete blood count | Every 12 weeks up to 2 years | |
| Total bilirubin |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Ecogene-21 | Saguenay | Quebec | G7H 7K9 | Canada |
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| 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 |
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| ID | Term |
|---|---|
| C000621590 | evinacumab |
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Open-label
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| Every 12 weeks up to 2 years |
| Creatine phosphokinase | Every 12 weeks up to 2 years |
| 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 |