Homozygous familial hypercholesterolemia MedDRA version: 20.1 Level: LLT Classification code 10020604 Term: Hypercholesterolemia System Organ Class: 100000004861
Conditions
Sponsors
Regeneron Pharmaceuticals Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Key inclusion criteria: 1. Male or female =12 years of age at the time of the screening visit 2. Diagnosis of functional HoFH 3. If undergoing LDL apheresis, must have initiated LDL apheresis at least 3 months prior to screening and must have been on a stable weekly or every other week schedule and/or stable settings for at least 8 weeks 4. Willing to consistently maintain his/her usual low fat or heart-healthy diet for the duration of the study Note: Other protocol defined inclusion/exclusion criteria may apply. Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 47 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Key exclusion criteria: 1. LDL-C level 9%) diabetes 7. History of a MI, unstable angina leading to hospitalization, coronary artery bypass graft surgery, percutaneous coronary intervention, uncontrolled cardiac arrhythmia, carotid surgery or stenting, stroke, transient ischemic attack, valve replacement surgery, carotid revascularization, endovascular procedure or surgical intervention for peripheral vascular disease within 3 months prior to the screening visit 8. Pregnant or breastfeeding women 9. Sexually active women of child bearing potential (WOCBP), who are unwilling to practice a highly effective birth control method prior to the initial dose, during the study, and for 24 weeks after the last dose of study drug 10. Sexually active men who are unwilling to use forms of medically acceptable birth control during the study drug treatment period and for 24 weeks after the last dose of study drug Note: Other protocol defined inclusion/exclusion criteria may apply.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of the study is to demonstrate the reduction of LDL-C by evinacumab 15 mg/kg IV in comparison to placebo after 24 weeks in patients with HoFH.; Secondary Objective: The secondary objectives of the study are: • To evaluate the effect of evinacumab 15 mg/kg IV on other lipid parameters (ie, Apo B, non-HDL-C, total cholesterol [TC]) in patients with HoFH • To evaluate the effect of evinacumab on LDL-C goal attainment • To assess the effect of evinacumab on eligibility for apheresis (using German and US apheresis criteria) • To evaluate the safety and tolerability of evinacumab 15 mg/kg in patients with HoFH • To determine concentrations of evinacumab in patients with HoFH • To evaluate the potential development of anti-evinacumab antibodies ;Primary end point(s): The primary endpoint is the percent change in calculated LDL-C from baseline to week 24. The primary endpoint is defined as: 100x (calculated LDL-C value at week 24 - calculated LDL C value at baseline)/calculated LDL-C value at baseline.;Timepoint(s) of evaluation of this end point: week 24 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): The key secondary endpoints are: • The percent change in Apo B from baseline to week 24 (ITT estimand) • The percent change in non-HDL-C from baseline to week 24 (ITT estimand) • The percent change in TC from baseline to week 24 (ITT estimand) • The proportion of patients with =30% reduction in calculated LDL-C at week 24 (ITT estimand) • The proportion of patients with =50% reduction in calculated LDL-C at week 24 (ITT estimand) • The proportion of patients with LDL-C <100 mg/dL [2.59 mmol/L] at week 24 (ITT estimand) • The change in calculated LDL-C from baseline to week 24 (ITT estimand) • The proportion of patients who meet EU apheresis eligibility criteria (see German Apheresis Working Group) at week 24 (ITT estimand) • The proportion of patients who meet US apheresis eligibility criteria (see US [National Lipid Association] Lipid Apheresis Criteria) at week 24 (ITT estimand) Refer to protocol for a full list. ;Timepoint(s) of evaluation of this end point: week 24 | — |
Countries
Australia, Austria, Canada, Czech Republic, France, Germany, Greece, Italy, Netherlands, South Africa, Turkey, Ukraine, United States
Contacts
Public ContactClinical Trial Information
Regeneron Pharmaceuticals, Inc.
Outcome results
None listed