Safety and Effectiveness of Evinacumab for the Treatment of Homozygous Familial Hypercholesterolemia

Study Overview
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Brief Summary
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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.

Official Title
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Safety and Effectiveness of Evinacumab for the Treatment of Homozygous Familial Hypercholesterolemia in a Real Life Setting in Canada

Conditions
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Homozygous Familial Hypercholesterolemia

Intervention / Treatment
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* Drug: Evinacumab


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: HoFH patients having been previously or being currently treated with evinacumab or clinical diagnosis of HoFH requiring additional lipid lowering therapy. Exclusion Criteria: Any new condition or worsening of an existing condition, which in the opinion of the investigator would make the patient unsuitable for enrollment, or could interfere with the patient participating in or completing the study; Pregnant or breastfeeding women; Women of childbearing potential who are unwilling to practice highly effective contraception prior to the initial dose/start of the first treatment, during the study, and for at least 24 weeks after the last dose of study drug.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Interventional Model Description: Open-label
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Evinacumab-treated patients<br> | Drug: Evinacumab<br>* Evinacumab 15 mg/kg administered intravenously every 4 weeks<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | 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 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Lipoprotein (a) |  | Yearly up to 2 years | 
| Complete blood count |  | Every 12 weeks up to 2 years | 
| Total bilirubin |  | Every 12 weeks up to 2 years | 
| Creatine phosphokinase |  | Every 12 weeks up to 2 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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ANGPTL3, Real-life study, Monoclonal antibody