Safety and Effectiveness of Evinacumab for the Treatment of Homozygous Familial Hypercholesterolemia
- Registration Number
- NCT05611528
- Lead Sponsor
- Daniel Gaudet
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 10
- HoFH patients having been previously or being currently treated with evinacumab or clinical diagnosis of HoFH requiring additional lipid lowering therapy.
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Evinacumab-treated patients Evinacumab -
- Primary Outcome Measures
Name Time Method Change in lipid profile 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 Every 4 weeks up to 2 years Change in aspartate transaminase (AST) plasma concentration Every 4 weeks up to 2 years
- Secondary Outcome Measures
Name Time Method 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
Trial Locations
- Locations (1)
Ecogene-21
🇨🇦Saguenay, Quebec, Canada