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

Homozygous Familial Hypercholesterolemia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05611528
• Other study ID #: ECO HoFH-2022-01
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: February 21, 2023
• Est. completion date: February 2026

Study information

• Verified date: August 2023
• Source: Ecogene 21
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 10
• Est. completion date: February 2026
• Est. primary completion date: December 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Homozygous Familial Hypercholesterolemia
• Hypercholesterolemia
• Hyperlipoproteinemia Type II

Intervention

Drug:
• Evinacumab
Evinacumab 15 mg/kg administered intravenously every 4 weeks

Locations

Country	Name	City	State
Canada	Ecogene-21	Saguenay	Quebec

Sponsors (2)

Lead Sponsor	Collaborator
Daniel Gaudet	Ultragenyx Pharmaceutical Inc

Country where clinical trial is conducted

Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	Change in aspartate transaminase (AST) plasma concentration		Every 4 weeks up to 2 years
Primary	change in alanine aminotransferase (ALT) plasma concentration		Every 4 weeks up to 2 years
Secondary	Lipoprotein (a)		Yearly up to 2 years
Secondary	Complete blood count		Every 12 weeks up to 2 years
Secondary	Total bilirubin		Every 12 weeks up to 2 years
Secondary	Creatine phosphokinase		Every 12 weeks up to 2 years