Clinical trial NCT00867165: Pediatric Study to Evaluate the Efficacy and Safety of Ezetimibe Monotherapy in Children With Primary Hypercholesterolemia (P05522)
Official title: A Randomized, Double-Blind, Parallel-Group, Placebo-Controlled Efficacy and Safety Study of Ezetimibe Monotherapy in Children (Ages 6 to 10 Years) With Primary Hypercholesterolemia (Heterozygous Familial and Nonfamilial) (Phase 3, Protocol No. P05522)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Primary Hypercholesterolemia
Keywords: heterozygous familial hypercholesterolemia
nonfamilial hypercholesterolemia
Lead sponsor: Organon and Co
Sponsor class: INDUSTRY
Collaborators: name: Merck Sharp & Dohme LLC
First posted: 2009-03-23
Enrollment: 138
Sex: ALL
Minimum age (years): 6
Maximum age (years): 10
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to determine the effect of ezetimibe 10 mg/day compared to placebo on the reduction of low-density lipoprotein cholesterol (LDL-C) from baseline to 12 weeks of treatment in children \>=6 to \<=10 years old with primary hypercholesterolemia. The study will also evaluate the effect of ezetimibe on total cholesterol (TC), apolipoprotein B (Apo B), high-density lipoprotein cholesterol (HDL-C), non-HDL-C, and triglycerides (TG). The safety of ezetimibe in this subject population will also be evaluated.


Eligibility criteria: Inclusion Criteria:

* Each subject may be of either sex and of any race/ethnicity, and must be \>=6 and \<=10 years of age, with a diagnosis of primary hypercholesterolemia (heterozygous familial or nonfamilial) despite being on a lipid-lowering diet for at least 3 months with a LDL-C of \>159mg/dL
* Each subject's parent/guardian must be willing to give written informed consent on his/her behalf.

Exclusion Criteria:

Each subject must not:

* Have known hypersensitivity or any contraindication to ezetimibe.
* Have use of any investigational drugs within 30 days of study entry.
* Be a member or a family member of the personnel of the investigational or sponsor staff directly involved with this trial.
* Be a female of child-bearing potential who is pregnant, intends to become pregnant, or is nursing
* Have known congenital cardiac disorder.
* Have documented or laboratory values consistent with homozygous familial hypercholesterolemia (HoFH).
* Be known to be human immunodeficiency virus (HIV) positive.

Primary outcomes:
measure: Percentage Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) at Week 12
timeFrame: Baseline and Week 12
Secondary outcomes:
measure: Percentage Change From Baseline in Total Cholesterol (TC) at Week 12
timeFrame: Baseline and Week 12
measure: Percentage Change From Baseline in Apolipoprotein B (Apo B) at Week 12
timeFrame: Baseline and Week 12
measure: Percentage Change From Baseline High-density Lipoprotein Cholesterol (HDL-C) at Week 12
timeFrame: Baseline and Week 12
measure: Percentage Change From Baseline in Non-HDL-C at Week 12
timeFrame: Baseline and Week 12
measure: Percentage Change From Baseline in Triglycerides (TG) at Week 12
timeFrame: Baseline and Week 12
measure: Percent Change From Baseline in LDL-C at Week 2
timeFrame: Baseline and Week 2
measure: Percent Change From Baseline in LDL-C at Week 4
timeFrame: Baseline and Week 4
measure: Percent Change From Baseline in LDL-C at Week 8
timeFrame: Baseline and Week 8
measure: Percentage Change From Baseline in TC at Week 2
timeFrame: Baseline and Week 2
measure: Percentage Change From Baseline in TC at Week 4
timeFrame: Baseline and Week 4
measure: Percentage Change From Baseline in TC at Week 8
timeFrame: Baseline and Week 8
measure: Percentage Change From Baseline HDL-C at Week 2
timeFrame: Baseline and Week 2
measure: Percentage Change From Baseline HDL-C at Week 4
timeFrame: Baseline and Week 4
measure: Percentage Change From Baseline HDL-C at Week 8
timeFrame: Baseline and Week 8
measure: Percentage Change From Baseline in Non-HDL-C at Week 2
timeFrame: Baseline and Week 2
measure: Percentage Change From Baseline in Non-HDL-C at Week 4
timeFrame: Baseline and Week 4
measure: Percentage Change From Baseline in Non-HDL-C at Week 8
timeFrame: Baseline and Week 8
measure: Percentage Change From Baseline in TG at Week 2
timeFrame: Baseline and Week 2
measure: Percentage Change From Baseline in TG at Week 4
timeFrame: Baseline and Week 4
measure: Percentage Change From Baseline in TG at Week 8
timeFrame: Baseline and Week 8
measure: Percentage Change From Baseline in Apolipoprotein A-I (Apo A-I) at Week 12
timeFrame: Baseline and Week 12
measure: Percentage Change From Baseline in TC:HDL-C Ratio at Week 2
timeFrame: Baseline and Week 2
measure: Percentage Change From Baseline in TC:HDL-C Ratio at Week 4
timeFrame: Baseline and Week 4
measure: Percentage Change From Baseline in TC:HDL-C Ratio at Week 8
timeFrame: Baseline and Week 8
measure: Percentage Change From Baseline in TC:HDL-C Ratio at Week 12
timeFrame: Baseline and Week 12
measure: Percentage Change From Baseline in LDL-C:HDL-C Ratio at Week 2
timeFrame: Baseline and Week 2
measure: Percentage Change From Baseline in LDL-C:HDL-C Ratio at Week 4
timeFrame: Baseline and Week 4
measure: Percentage Change From Baseline in LDL-C:HDL-C Ratio at Week 8
timeFrame: Baseline and Week 8
measure: Percentage Change From Baseline in LDL-C:HDL-C Ratio at Week 12
timeFrame: Baseline and Week 12
measure: Percentage Change From Baseline in Apo B:Apo A-I Ratio at Week 12
timeFrame: Baseline and Week 12
measure: Percentage Change From Baseline in High-sensitivity C-reactive Protein (Hs-CRP) at Week 4
timeFrame: Baseline and Week 4
measure: Percentage Change From Baseline in High-sensitivity C-reactive Protein (Hs-CRP) at Week 12
timeFrame: Baseline and Week 12
measure: Percent Change From Baseline in Sitosterol at Week 2
timeFrame: Baseline and Week 2
measure: Percentage Change From Baseline in Sitosterol at Week 4
timeFrame: Baseline and Week 4
measure: Percentage Change From Baseline in Sitosterol at Week 8
timeFrame: Baseline and Week 8
measure: Percentage Change From Baseline in Sitosterol at Week 12
timeFrame: Baseline and Week 12
measure: Percentage Change From Baseline in Campesterol at Week 2
timeFrame: Baseline and Week 2
measure: Percentage Change From Baseline in Campesterol at Week 4
timeFrame: Baseline and Week 4
measure: Percentage Change From Baseline in Campesterol at Week 8
timeFrame: Baseline and Week 8
measure: Percentage Change From Baseline in Campesterol at Week 12
timeFrame: Baseline and Week 12
measure: Percentage Change From Baseline in Cholestanol at Week 2
timeFrame: Baseline and Week 2
measure: Percentage Change From Baseline in Cholestanol at Week 4
timeFrame: Baseline and Week 4
measure: Percentage Change From Baseline in Cholestanol at Week 8
timeFrame: Baseline and Week 8
measure: Percentage Change From Baseline in Cholestanol at Week 12
timeFrame: Baseline and Week 12
measure: Percentage Change From Baseline in Lathosterol at Week 2
timeFrame: Baseline and Week 2
measure: Percentage Change From Baseline in Lathosterol at Week 4
timeFrame: Baseline and Week 4
measure: Percentage Change From Baseline in Lathosterol at Week 8
timeFrame: Baseline and Week 8
measure: Percentage Change From Baseline in Lathosterol at Week 12
timeFrame: Baseline and Week 12
Other outcomes:
Locations:
Study officials:
References:
citation: Kusters DM, Caceres M, Coll M, Cuffie C, Gagne C, Jacobson MS, Kwiterovich PO, Lee R, Lowe RS, Massaad R, McCrindle BW, Musliner TA, Triscari J, Kastelein JJ. Efficacy and safety of ezetimibe monotherapy in children with heterozygous familial or nonfamilial hypercholesterolemia. J Pediatr. 2015 Jun;166(6):1377-84.e1-3. doi: 10.1016/j.jpeds.2015.02.043. Epub 2015 Apr 1.