STUDY TO EVALUATE THE EFFICACY AND SAFETY OF EVINACUMAB IN PATIENTS WITH HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA
- Conditions
- Homozygous familial hypercholesterolemia (HoFH)
- Registration Number
- JPRN-jRCT2080224038
- Lead Sponsor
- Regeneron Pharmaceuticals, Inc.(ICCC: PAREXEL International)
- Brief Summary
In patients with homozygous familial hypercholesterolemia receiving maximum doses of lipid-lowering therapy, the reduction from baseline in the LDL cholesterol level in the evinacumab group, as compared with the small increase in the placebo group, resulted in a between-group difference of 49.0 percentage points at 24 weeks.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- completed
- Sex
- All
- Target Recruitment
- 65
1. Diagnosis of functional HoFH
2. 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
1.LDL-C level <70 mg/dL (1.81 mmol/L) at the screening visit
2.Background medical Lipid Modifying Therapy (LMT) (if applicable) that has not been stable before the screening visit
3.Lipid-apheresis schedule /apheresis settings (if applicable) that have not been stable for at least 8 weeks before the screening visit
4.Use of nutraceuticals or over-the-counter therapies known to affect lipids, at a dose/amount that has not been stable for at least 4 weeks prior to the screening visit
5.Presence of any clinically significant uncontrolled endocrine disease known to influence serum lipids or lipoproteins
6.Newly diagnosed (within 3 months prior to randomization visit) diabetes mellitus or poorly controlled (HbA1c >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.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method efficacy<br>LDL-C <br>Percent change in calculated LDL-C from baseline to week 24
- Secondary Outcome Measures
Name Time Method efficacy<br>Apolipoprotein B (Apo B) <br>non-High-density lipoprotein cholesterol (non-HDL-C) <br>Total cholesterol (TC) <br>Triglyceride (TG) <br>lipoprotein a [Lp(a)] <br>apolipoprotein CIII (Apo CIII) <br><br>Percent change in Apolipoprotein B (Apo B) from baseline to week 24<br>Percent change in non-High-density lipoprotein cholesterol (HDL-C) from baseline to week 24<br>Percent change in Total cholesterol (TC) from baseline to week 24 <br>Percent change in Triglyceride (TG) from baseline to week 24 <br>Percent change in lipoprotein a [Lp(a)] from baseline to week 24 <br>Percent change in apolipoprotein CIII (Apo CIII) from baseline to week 24