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

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

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 Overview

• Status: Completed
• Conditions: Homozygous Familial Hypercholesterolemia
• Intervention / Treatment: Drug: Evinacumab

• Study Type: Interventional
• Enrollment (Actual): 10
• Phase: Phase 3

Contacts and Locations

Study Locations

• Canada
  • Quebec
    • Saguenay, Quebec, Canada, G7H 7K9
      • Ecogene-21

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Evinacumab-treated patients	Drug: Evinacumab; Evinacumab 15 mg/kg administered intravenously every 4 weeks

What is the study measuring?

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	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

Collaborators and Investigators

• Sponsor: Daniel Gaudet
• Collaborators: Ultragenyx Pharmaceutical Inc

Study record dates

Study Major Dates

• Study Start (Actual): February 21, 2023
• Primary Completion (Actual): December 27, 2024
• Study Completion (Actual): March 18, 2025

Study Registration Dates

• First Submitted: October 31, 2022
• First Submitted That Met QC Criteria: November 3, 2022
• First Posted (Actual): November 10, 2022

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: March 21, 2025
• Last Verified: March 1, 2025

More Information

Terms related to this study

• Keywords: Monoclonal antibody; ANGPTL3; Real-life study
• Additional Relevant MeSH Terms: Metabolism, Inborn Errors; Genetic Diseases, Inborn; Metabolic Diseases; Hyperlipidemias; Dyslipidemias; Lipid Metabolism Disorders; Lipid Metabolism, Inborn Errors; Hyperlipoproteinemias; Homozygous Familial Hypercholesterolemia; Hypercholesterolemia; Hyperlipoproteinemia Type II
• Other Study ID Numbers: ECO HoFH-2022-01

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes