Double-blind, Randomized, Multicenter, Placebo-controlled, Parallel Group Study to Characterize the Efficacy, Safety, and Tolerability of 24 Weeks of Evolocumab for Low Density Lipoprotein-cholesterol (LDL-C) Reduction, as Add-On to Diet and Lipid-Lowering Therapy, in Pediatric Subjects 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH)
- Conditions
- Hypercholesterolemia in children - HeFH10027424
- Registration Number
- NL-OMON47734
- Lead Sponsor
- Amgen
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 25
- Male or female, aged 10 to 17 years of age at time of randomization
- Diagnosis of heterozygous familial hypercholesterolemia by local applicable
diagnostic criteria for HeFH or by genetic testing.
- Subject must be on an approved statin with stable dose for >= 4 weeks before
LDL*C screening and, in the opinion of the investigator, not requiring
up*titration
- Fasting LDL-C at screening >= 130 mg/dL (3.4 mmol/L)
- Subject has fasting triglycerides <= 400 mg/dL (4.5 mmol/L)
- Type 1 diabetes, newly diagnosed/ poorly controlled type 2 diabetes
(HbA1c>8,5%), or newly diagnosed impaired glucose tolerance.
- Untreated or inadequately treated hyper/hypo-thyroidism
- Moderate to severe renal dysfunction (eGFR < 30 ml/min/1.73m2 at screening)
- Persistent active liver disease or hepatic dysfunction (AST or ALT >2 times
ULN as determined by central lab analysis at screening)
- CK > 3 times the ULN at screening, confirmed by a repeat measurement at least
1 week apart.
- Known active infection or major hematologic, renal, metabolic,
gastrointestinal or endocrine dysfunction in the judgment of the investigator
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Hypotheses: The primary hypothesis is that SC evolocumab will be well tolerated<br /><br>and will result in greater reduction of LDL-C, defined as percent change from<br /><br>baseline to week 24, compared with placebo, when added to standard of care in<br /><br>pediatric subjects 10 to 17 years of age with HeFH.<br /><br>Primary Endpoint: Percent change from baseline to week 24 in LDL-C</p><br>
- Secondary Outcome Measures
Name Time Method <p>Secondary Endpoints:<br /><br>• Mean percent change from baseline to weeks 22 and 24 in LDL-C<br /><br>• change from baseline to week 24 in LDL-C<br /><br>• percent change from baseline to week 24 in the following:<br /><br>* non-HDL-C<br /><br>* ApoB<br /><br>* total cholesterol/HDL-C ratio<br /><br>* ApoB/ApoA1 ratio</p><br>