Homozygous Familial Hypercholesterolemia
Conditions
Keywords
ANGPTL3, Real-life study, Monoclonal antibody
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.
Interventions
DRUGEvinacumab
Evinacumab 15 mg/kg administered intravenously every 4 weeks
Sponsors
Ultragenyx Pharmaceutical Inc
Daniel Gaudet
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Open-label
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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 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
| Measure | Time frame |
|---|---|
| Lipoprotein (a) | Yearly up to 2 years |
| Creatine phosphokinase | Every 12 weeks up to 2 years |
| Complete blood count | Every 12 weeks up to 2 years |
| Total bilirubin | Every 12 weeks up to 2 years |
Countries
Canada
Outcome results
None listed