NCT05611528

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
10

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Feb 2023

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

October 31, 2022

Completed
10 days until next milestone

First Posted

Study publicly available on registry

November 10, 2022

Completed
3 months until next milestone

Study Start

First participant enrolled

February 21, 2023

Completed
1.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 27, 2024

Completed
3 months until next milestone

Study Completion

Last participant's last visit for all outcomes

March 18, 2025

Completed
Last Updated

March 25, 2025

Status Verified

March 1, 2025

Enrollment Period

1.8 years

First QC Date

October 31, 2022

Last Update Submit

March 21, 2025

Conditions

Homozygous Familial Hypercholesterolemia

Keywords

ANGPTL3Real-life studyMonoclonal antibody

Outcome Measures

Primary Outcomes (3)

  • 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 Outcomes (4)

  • 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

Study Arms (1)

Evinacumab-treated patients

EXPERIMENTAL
Drug: Evinacumab

Interventions

EvinacumabDRUG

Evinacumab 15 mg/kg administered intravenously every 4 weeks

Evinacumab-treated patients

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • HoFH patients having been previously or being currently treated with evinacumab or clinical diagnosis of HoFH requiring additional lipid lowering therapy.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Ecogene-21

Saguenay, Quebec, G7H 7K9, Canada

Location

MeSH Terms

Conditions

Homozygous Familial Hypercholesterolemia

Interventions

evinacumab

Condition Hierarchy (Ancestors)

Hyperlipoproteinemia Type IILipid Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesHyperlipoproteinemiasHyperlipidemiasDyslipidemiasLipid Metabolism DisordersMetabolic DiseasesNutritional and Metabolic Diseases

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Model Details: Open-label
Sponsor Type
OTHER
Responsible Party
SPONSOR INVESTIGATOR
PI Title
Director

Study Record Dates

First Submitted

October 31, 2022

First Posted

November 10, 2022

Study Start

February 21, 2023

Primary Completion

December 27, 2024

Study Completion

March 18, 2025

Last Updated

March 25, 2025

Record last verified: 2025-03

Locations

CA(1)