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 Yearsof Age With Heterozygous Familial Hypercholesterolemia (HeFH)
- Conditions
- Heterozygous familial hypercholesterolaemiaMedDRA version: 18.0Level: LLTClassification code 10057079Term: Heterozygous familial hypercholesterolemiaSystem Organ Class: 100000004850Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]
- Registration Number
- EUCTR2014-002277-11-IT
- Lead Sponsor
- Amgen Inc
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 158
-Subject has provided informed consent or subject assent prior to initiation of any study-specific activities/procedures.
and/or
- Subject’s legally acceptable representative has provided informed consent when the subject is legally too young to provide informed consent and the subject has provided written subject assent based on local regulations and/or guidelines prior to any study-specific activities/procedures being initiated.
- Male or female, = 10 to = 17 years of age at time of randomization (includes the year after the subject completes the 17th year after birth but not the day of completing the 18th year after birth).
-Diagnosis of heterozygous familial hypercholesterolemia by local applicable diagnostic criteria for HeFH (ie, criteria outlined by the Simon Broome Register Group [Scientific Steering Committee, 1991] or the Dutch Lipid Clinic Network [World Health Organization, 1999]) or by genetic testing.
- 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.
Are the trial subjects under 18? yes
Number of subjects for this age range: 150
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
- Type 1 diabetes or newly diagnosed (within 3 months of randomization) type 2 diabetes, or poorly controlled type 2 diabetes (HbA1c > 8.5%) or newly diagnosed impaired glucose tolerance (within 3 months of randomization).
- Untreated or inadequately treated hyperthyroidism or hypothyroidism as defined by thyroid stimulating hormone (TSH) < lower limit of normal (LLN) or > 1.5 times the upper limit of normal (ULN), respectively, and free thyroxine (T4) levels that are outside normal range at screening.
- Moderate to severe renal dysfunction, defined as an estimated glomerular filtration rate (eGFR) < 30 ml/min/1.73m2 at screening, confirmed by a repeat measurement at least 1 week apart.
- Persistent active liver disease or hepatic dysfunction, defined as aspartate aminotransferase (AST) or alanine aminotransferase (ALT) > 2 times the ULN as determined by central laboratory analysis at screening, confirmed by a repeat measurement at least 1 week apart.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To evaluate the effect of 24 weeks of subcutaneous (SC) evolocumab compared with placebo, when added to standard of care, on percent change from baseline in low-density lipoprotein cholesterol (LDL-C) in pediatric subjects 10 to 17 years of age with heterozygous familial hypercholesterolemia (HeFH).;Secondary Objective: • to evaluate the safety of SC evolocumab compared with placebo, when added to standard of care, in pediatric subjects 10 to 17 years of age with HeFH<br>• to assess the effects of SC evolocumab compared with placebo, when added to standard of care, on mean percent change from baseline to weeks 22 and 24 and change from baseline to week 24 in LDL-C, and on percent change from baseline to week 24 in other lipid parameters in<br>pediatric subjects 10 to 17 years of age with HeFH<br>• To characterize PK exposure;Primary end point(s): Percent change from baseline to week 24 in LDL-C;Timepoint(s) of evaluation of this end point: Baseline and week 24
- Secondary Outcome Measures
Name Time Method Secondary end point(s): -Mean percent change from baseline to weeks 22 and 24 in LDL-C<br>-Change from baseline to week 24 in LDL-C<br>-Percent change from baseline to week 24 in the following:<br>- non-HDL-C<br>- ApoB<br>- total cholesterol/HDL-C ratio<br>- ApoB/ApoA1 ratio;Timepoint(s) of evaluation of this end point: Baseline, week 22 and week 24