An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia

Regeneron Pharmaceuticals5 sites in 2 countries116 target enrollmentMarch 13, 2018

ConditionsHomozygous Familial Hypercholesterolemia

Interventionsevinacumab

Drugsevinacumab

Overview

Phase: Phase 3
Intervention: evinacumab
Conditions: Homozygous Familial Hypercholesterolemia
Sponsor: Regeneron Pharmaceuticals
Enrollment: 116
Locations: 5
Primary Endpoint: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Up to Week 216
Status: Completed
Last Updated: last year

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

The primary objectives of the study are:

To evaluate the long-term safety and tolerability of evinacumab in patients with Homozygous Familial Hypercholesterolemia (HoFH)
To evaluate the long-term safety and tolerability of evinacumab in adolescent patients with HoFH

The secondary objectives of the study are:

To evaluate the effect of evinacumab on lipid parameters in patients with HoFH
To evaluate the effect of evinacumab on lipid parameters in adolescent patients with HoFH
To evaluate the potential development of anti-evinacumab antibodies

Registry

clinicaltrials.gov

Start Date

March 13, 2018

End Date

April 13, 2023

Last Updated

last year

Study Type

Interventional

Study Design

Single Group

Sex

All

Investigators

Sponsor

Regeneron Pharmaceuticals

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Completion of the parent study in which they participated
•Able to understand and complete study-related questionnaires

Exclusion Criteria

•Significant protocol deviation in the parent study based on the investigator's judgment, such as non-compliance by the patient
•Concomitant medications that have not been stable prior to the baseline visit
•Adverse event leading to permanent discontinuation from parent study
•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
•Member of the clinical site study team and/or his/her immediate family
•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
•Men who are sexually active with women of childbearing potential and are unwilling to use the following forms of medically acceptable birth control during the study drug treatment period and for 24 weeks after the last injection of study drug: vasectomy with medical assessment of surgical success OR consistent use of a condom.
•Note: Other protocol defined Inclusion/Exclusion criteria apply

Arms & Interventions

evinacumab

Intervention: evinacumab

Outcomes

Primary Outcomes

Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Up to Week 216

Time Frame: Up to 216 weeks

The safety analysis set (SAF) included all participants who were enrolled and received at least 1 dose or part of a dose of open-label study treatment.

Secondary Outcomes

Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) Over Time(Up to 120 weeks)
Absolute Change in Non-HDL-C Over Time(Up to 120 weeks)
Absolute Change in LDL-C Over Time(Up to 120 weeks)
Percent Change in Triglycerides (TGs) Over Time(Up to 120 weeks)
Percent Change in Apolipoprotein B (Apo B) Over Time(Up to 120 weeks)
Percent Change in Total Cholesterol (TC) Over Time(Up to 120 weeks)
Absolute Change in Apo B Over Time(Up to 120 weeks)
Percent Change in Non-High-Density Lipoprotein Cholesterol (HDL-C) Over Time(Up to 120 weeks)
Absolute Change in TC Over Time(Up to 120 weeks)
Absolute Change in TGs Over Time(Up to 120 weeks)