A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of REGN727/SAR236553 in Patients with Heterozygous Familial Hypercholesterolemia Not Adequately Controlled with Their Lipid-Modifying Therapy
- Conditions
- familial hyperchomesterolemiainherited hyperlipidemia100274241001331710003216
- Registration Number
- NL-OMON39622
- Lead Sponsor
- Regeneron Pharmaceuticals, Inc.
- Brief Summary
Trial is onging in other countries
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 100
1.Patients with heFH* who are not adequately controlled** with a maximally-tolerated daily dose*** of statin with or without other LMT, at a stable dose prior to the screening visit (week -2).;*Diagnosis of heFH must be made either by genotyping or by clinical criteria. For those patients not genotyped, the clinical diagnosis may be based on either the Simon Broome criteria for definite FH (Appendix 1) or the WHO/Dutch Lipid Network criteria with a score of >8 points (Appendix 2).;***Not adequately controlled* is defined as LDL-C *70 mg/dL (1.81 mmol/L) at the screening visit (week -2) in patients with a history of documented CVD (Appendix 3), or LDL-C *100 mg/dL (2.59 mmol/L) at the screening visit (week -2) in patients without a history of documented CVD.;****Maximally-tolerated dose* is defined as (any of the following are acceptable):;*Rosuvastatin 20 mg or 40 mg daily;*Atorvastatin 40 mg or 80 mg daily;*Simvastatin 80 mg daily (if already on this dose for >1 year * see exclusion criterion #7);Note: Patients who are not able to be on any of the above statin doses should be treated with the dose of daily atorvastatin, rosuvastatin, or simvastatin which is considered appropriate for the patient, according to the investigator's judgment. Some examples of acceptable reasons for a patient taking a lower statin dose include, but are not limited to: adverse effects on higher doses, advanced age, low body mass index, regional practices, local prescribing information, concomitant medications, co-morbid conditions such as impaired glucose tolerance/impaired fasting glucose. The reason(s) will be documented in the case report form (CRF).;2.Provide signed informed consent
1.Patient without diagnosis of heFH made either by genotyping or by clinical criteria;2.LDL-C <70 mg/dL (<1.81 mmol/L) at the screening visit (week-2) in patients with history of documented cardiovascular disease;3.LDL-C <100 mg/dL (<2.59 mmol/L) at the screening visit (week *2) in patients without history of documented cardiovascular disease;4.Not on a stable dose of LMT (including statin) for at least 4 weeks and/or fenofibrate for at least 6 weeks, as applicable, prior to the screening visit (week -2) and from screening to randomization;5.Currently taking another statin than simvastatin, atorvastatin, or rosuvastatin;6.Simvastatin, atorvastatin, or rosuvastatin is not taken daily or not taken at a registered dose;7.Daily doses above atorvastatin 80 mg, rosuvastatin 40 mg, or simvastatin 40 mg (except for patients on simvastatin 80 mg for more than 1 year, who are eligible);8.Use of fibrates, other than fenofibrate within 6 weeks of the screening visit (week-2) or between screening and randomization visits;9.Use of nutraceutical products or over-the-counter therapies that may affect lipids which have not been at a stable dose/amount for at least 4 weeks prior to the screening visit (week -2) or between screening and randomization visits;10.Use of red yeast rice products within 4 weeks of the screening visit (week-2), or between screening and randomization visits;11.Patient who has received plasmapheresis treatment within 2 months prior to the screening visit (week -2), or has plans to receive it during the study;12.Recent (within 3 months prior to the screening visit [week -2] or between screening and randomization visits) MI, unstable angina leading to hospitalization, percutaneous coronary intervention (PCI), coronary artery bypass graft surgery (CABG), uncontrolled cardiac arrhythmia, stroke, transient ischemic attack (TIA), carotid revascularization, endovascular procedure or surgical intervention for peripheral vascular disease
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>The percent change in calculated LDL-C from baseline to week 24, which is<br /><br>defined as : 100x (calculated LDL-C value at week 24 - calculated LDL-C value<br /><br>at baseline) / calculated LDL-C value at baseline.<br /><br>The baseline calculated LDL-C value will be the last LDL-C level obtained<br /><br>before the first double-blind IMP injection.</p><br>
- Secondary Outcome Measures
Name Time Method