Evinacumab for High Cholesterol

Overseen By Diane Brisson, PhD CCRP

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Daniel Gaudet

Must be taking: Lipid-modifying therapies

Disqualifiers: Pregnancy, Breastfeeding, Others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 4

Duration: Up to 24 months

10 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

Eligibility Criteria

Adults with homozygous familial hypercholesterolemia (HoFH) who are already on or need additional lipid-lowering therapy can join this trial. Pregnant or breastfeeding women, and women able to have children but not using effective birth control, cannot participate.

Inclusion Criteria

I have HoFH and need more treatment to lower my lipids.

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

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.

Pregnant or breastfeeding women

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive evinacumab in an open-label treatment period, added on top of their background lipid-modifying therapy

Up to 24 months

Every 4 weeks (on-site, foldable sites)

End of Study (EoS)

End of study visit scheduled 4 weeks after the last dose

4 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

52 weeks

Treatment Details

Interventions

• Evinacumab

Trial Overview The study is testing the long-term safety and effectiveness of Evinacumab, an antibody treatment for HoFH. It's added to existing treatments like statins in a real-life setting over up to 24 months, with follow-ups.

Participant Groups

Group I: Evinacumab-treated patients Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

Daniel Gaudet

Lead Sponsor

Trials

1

Recruited

10+

Ultragenyx Pharmaceutical Inc

Industry Sponsor

Trials

94

Recruited

104,000+

Dr. Emil D. Kakkis

Ultragenyx Pharmaceutical Inc

Chief Executive Officer since 2010

MD/PhD in Biological Chemistry from UCLA

Dr. Eric Crombez

Ultragenyx Pharmaceutical Inc

Chief Medical Officer since 2023

MD from Wayne State University School of Medicine