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OpenTrials
Completed

NCT Number: NCT05611528

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

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.

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Key information

Conditions

Homozygous Familial Hypercholesterolemia Congenital, Hereditary, and Neonatal Diseases and Abnormalities Dyslipidemias Genetic Diseases, Inborn Hyperlipidemias Hyperlipoproteinemia Type II Hyperlipoproteinemias Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Metabolic Diseases Metabolism, Inborn Errors Nutritional and Metabolic Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Ecogene-21

Saguenay, Quebec, G7H 7K9, Canada

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Evinacumab

Drug

Evinacumab 15 mg/kg administered intravenously every 4 weeks

Primary outcomes

  1. Change in lipid profile

    Time frame: Every 4 weeks up to 2 years

    Change in total cholesterol, LDL-Cholesterol, HDL-Cholesterol, triglyceride, non-HDL-Cholesterol and apolipoprotein B plasma concentration

  2. Change in aspartate transaminase (AST) plasma concentration

    Time frame: Every 4 weeks up to 2 years

  3. change in alanine aminotransferase (ALT) plasma concentration

    Time frame: Every 4 weeks up to 2 years

Secondary outcomes

  1. Lipoprotein (a)

    Time frame: Yearly up to 2 years

  2. Complete blood count

    Time frame: Every 12 weeks up to 2 years

  3. Total bilirubin

    Time frame: Every 12 weeks up to 2 years

  4. Creatine phosphokinase

    Time frame: Every 12 weeks up to 2 years

Sponsors and collaborators

Lead sponsor

Daniel Gaudet

Other

Collaborators

  • Ultragenyx Pharmaceutical Inc

Registry information

Official study title

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

Important dates

Study start
2023
Primary completion
2024
Study completion
2025
First posted
Nov 10, 2022
Registry last updated
Mar 25, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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