Safety and Effectiveness of Evinacumab for the Treatment of Homozygous Familial Hypercholesterolemia in a Real Life Setting in Canada
Trial Snapshot
- Phase
- Phase 3
- Status
- Completed
- Sponsor
- Enrollment
- 10
- Locations
- 1
- Primary Endpoint
- Change in lipid profile
Study Overview
Brief Summary
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.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 18 Years to — (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
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.
Arms & Interventions
Evinacumab-treated patients
Intervention: Evinacumab (Drug)
Outcomes
Primary Outcomes
Change in lipid profile
Time Frame: Every 4 weeks up to 2 years
Change in total cholesterol, LDL-Cholesterol, HDL-Cholesterol, triglyceride, non-HDL-Cholesterol and apolipoprotein B plasma concentration
change in alanine aminotransferase (ALT) plasma concentration
Time Frame: Every 4 weeks up to 2 years
Change in aspartate transaminase (AST) plasma concentration
Time Frame: Every 4 weeks up to 2 years
Secondary Outcomes
- Total bilirubin(Every 12 weeks up to 2 years)
- Lipoprotein (a)(Yearly up to 2 years)
- Complete blood count(Every 12 weeks up to 2 years)
- Creatine phosphokinase(Every 12 weeks up to 2 years)
Investigators
Daniel Gaudet
Director
Ecogene 21