Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia

Study Overview
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Brief Summary
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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

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

Conditions
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Homozygous Familial Hypercholesterolemia

Intervention / Treatment
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* Drug: evinacumab


Participation Criteria
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Eligibility Criteria
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Key Inclusion Criteria: Completion of the parent study in which they participated Able to understand and complete study-related questionnaires Key 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

Ages Eligible for Study
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Minimum Age: 12 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: evinacumab<br> | Drug: evinacumab<br>* Intravenous (IV) administration<br>* Other names: EVKEEZA®;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence and severity of treatment-emergent adverse events (TEAEs) |  | Up to 192 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percent change in Low-Density Lipoprotein Cholesterol (LDL-C) over time |  | Up to 192 weeks | 
| Absolute change in LDL-C over time |  | Up to 192 weeks | 
| Percent change in Apolipoprotein B (Apo B) over time |  | Up to 192 weeks | 
| Absolute change in Apo B over time |  | Up to 192 weeks | 
| Percent change in non-High-Density Lipoprotein Cholesterol (HDL-C) over time |  | Up to 192 weeks | 
| Absolute change in non-HDL-C over time |  | Up to 192 weeks | 
| Percent change in Total Cholesterol (TC) over time |  | Up to 192 weeks | 
| Absolute change in TC over time |  | Up to 192 weeks | 
| Percent change in Triglycerides (TGs) over time |  | Up to 192 weeks | 
| Absolute change in TGs over time |  | Up to 192 weeks | 
| Presence of anti-evinacumab antibodies |  | Up to 192 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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HoFH