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

Sponsor

Daniel Gaudet (Other)

Overall Status

Not yet recruiting

CT.gov ID

NCT05611528

Collaborator

Ultragenyx Pharmaceutical Inc (Industry)

Enrollment

Arm

Anticipated Duration (Months)

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Homozygous Familial Hypercholesterolemia	Drug: Evinacumab	Phase 4

Study Design

Study Type:

Interventional

Anticipated Enrollment :

10 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Intervention Model Description:

Open-labelOpen-label

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

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

Anticipated Study Start Date :

Dec 1, 2022

Anticipated Primary Completion Date :

Dec 1, 2025

Anticipated Study Completion Date :

Feb 1, 2026

Arms and Interventions

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

Arm

Intervention/Treatment

Experimental: Evinacumab-treated patients

Drug: Evinacumab

Evinacumab 15 mg/kg administered intravenously every 4 weeks

Outcome Measures

Primary Outcome Measures

Change in lipid profile [Every 4 weeks up to 2 years]
Change in total cholesterol, LDL-Cholesterol, HDL-Cholesterol, triglyceride, non-HDL-Cholesterol and apolipoprotein B plasma concentration
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

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]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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.

Contacts and Locations

Locations

No locations specified.

Sponsors and Collaborators

Daniel Gaudet
Ultragenyx Pharmaceutical Inc

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Daniel Gaudet, Director, Ecogene 21

ClinicalTrials.gov Identifier:

NCT05611528

Other Study ID Numbers:

ECO HoFH-2022-01

First Posted:

Nov 10, 2022

Last Update Posted:

Nov 10, 2022

Last Verified:

Nov 1, 2022

Individual Participant Data (IPD) Sharing Statement:

Undecided

Plan to Share IPD:

Undecided

Studies a U.S. FDA-regulated Drug Product:

Yes

Studies a U.S. FDA-regulated Device Product:

Product Manufactured in and Exported from the U.S.:

Yes

Keywords provided by Daniel Gaudet, Director, Ecogene 21

ANGPTL3

Real-life study

Monoclonal antibody

Additional relevant MeSH terms:

Hyperlipoproteinemia Type II

Hypercholesterolemia

Study Results

No Results Posted as of Nov 10, 2022