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Study of the Safety and Efficacy of Varying Doses and Dose Regimens of Evinacumab in Patients With Persistent Hypercholesterolemia Despite Maximally Tolerated Lipid Modifying Therapy

Phase 2
Completed
Conditions
Therapeutic Category Code: 218 (Anti-hyperlipidemic)
Registration Number
JPRN-jRCT2080224063
Lead Sponsor
Regeneron Pharmaceuticals, Inc. (Clinical Trial In-Country Representative:PAREXEL International)
Brief Summary

In this phase 2 trial, use of evinacumab reduced levels of LDL cholesterol and atherogenic lipoproteins in patients with refractory hypercholesterolemia. The response to treatment, or reduction in the LDL cholesterol level, with subcutaneous and intravenous evinacumab was observed as early as the first postbaseline lipid assessment (week 2) and was maintained through week 16.

Detailed Description

Not available

Recruitment & Eligibility

Status
completed
Sex
All
Target Recruitment
272
Inclusion Criteria

1.Men and women, ages 18 through 80 at the screening visit
2.Diagnosis of primary hypercholesterolemia, either HeFH or non-HeFH with clinical ASCVD
3.A history of clinical ASCVD, for those patients who are non-HeFH.
4.Receiving a stable maximally tolerated statin (+- ezetimibe) for at least 4 weeks at screening
5.For those patients with HeFH who are not receiving a statin at screening, documentation of inability to tolerate at least 2 statins.
6.Receiving alirocumab 150 mg SC Q2W, OR evolocumab 140 mg SC Q2W or 420 mg SC Q4W for at least 8 weeks prior to the screening visit
7.For those patients with a history of clinical ASCVD, serum LDL-C >= 70 mg/dL at screening (1 repeat lab is allowed)
8.For those patients without a history of clinical ASCVD, serum LDL-C >=100 mg/dL at screening (1 repeat lab is allowed)
9.Provide signed informed consent

Exclusion Criteria

1.Known history of homozygous FH (clinically, or by previous genotyping)
2.Presence of any clinically significant uncontrolled endocrine disease known to influence serum lipids or lipoproteins
3.Newly diagnosed diabetes (within 3 months prior to screening)
4.Use of thyroid medications (except for replacement therapy which has been stable for at least 12 weeks before screening)
5.Laboratory findings during screening period (not including randomization labs):
a.Triglycerides > 400 mg/dL (> 4.52 mmol/L) for patients without a known history of diabetes mellitus; OR Triglycerides > 300 mg/dL (> 3.39 mmol/L) for patients with a known history of diabetes mellitus
b.Positive test for Hepatitis B surface antigen and/or Hepatitis C antibody (associated with a positive HCV ribonucleic acid [RNA] polymerase chain reaction)
c.Positive serum beta-human chorionic gonadotropin or urine pregnancy test in women of childbearing potential
d.Estimated glomerular filtration rate < 30 mL/min/1.73 m^2
e.TSH > 1.5 x ULN
f.Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 2 x ULN
6.Systolic blood pressure > 160 mmHg or diastolic blood pressure > 100 mmHg at screening visit or time of randomization
7.History of heart failure (New York Heart Association [NYHA] Class III-IV) within 12 months before screening
8.History of MI, unstable angina leading to hospitalization, CABG surgery, PCI, uncontrolled cardiac arrhythmia, carotid surgery or stenting, stroke, TIA, carotid revascularization, endovascular procedure or surgical intervention for peripheral vascular disease within 3 months prior screening
9.History of cancer within the past 5 years (except for adequately treated basal cell skin cancer, squamous cell skin cancer, or in situ cervical cancer)
10.Having received LDL apheresis within 2 months before screening
11.Pregnant or breast-feeding women
12.Women of childbearing potential who are unwilling to practice a highly effective birth control method
13.Men who are sexually active with women of childbearing potential (WOCBP) and are unwilling to consistently use condoms during the study drug treatment period regardless of vasectomy status.

Study & Design

Study Type
Interventional
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
efficacy<br>The primary efficacy endpoint is the percent change in calculated LDL-C from baseline to week 16 in the intent-to-treat (ITT) population, using all LDL-C values regardless of adherence to treatment and subsequent therapies.
Secondary Outcome Measures
NameTimeMethod
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