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

NCT ID: NCT05611528

Last Updated: 2025-03-25

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 10 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-02-21
• Study Completion Date: 2025-03-18

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.

Conditions

Homozygous Familial Hypercholesterolemia

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Evinacumab-treated patients

Group Type: EXPERIMENTAL

Evinacumab

Intervention Type: DRUG

Evinacumab 15 mg/kg administered intravenously every 4 weeks

Interventions

Evinacumab

Evinacumab 15 mg/kg administered intravenously every 4 weeks

Intervention Type: DRUG

Eligibility Criteria

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.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Ultragenyx Pharmaceutical Inc

INDUSTRY

Sponsor Role: collaborator

Daniel Gaudet

OTHER

Sponsor Role: lead

Responsible Party

Daniel Gaudet

Director

Responsibility Role: SPONSOR_INVESTIGATOR

Locations

Ecogene-21

Saguenay, Quebec, Canada

Countries

Canada

Other Identifiers

ECO HoFH-2022-01

Identifier Type: -

Identifier Source: org_study_id