NCT00867165

Pediatric Study to Evaluate the Efficacy and Safety of Ezetimibe Monotherapy in Children With Primary Hypercholesterolemia (P05522)

Completed Phase 3 Results posted Last updated 21 May 2024

What this trial tests

Phase 3 trial testing ezetimibe in Primary Hypercholesterolemia in 138 participants. Completed in 13 April 2012.

Timeline

21 May 2009

Primary endpoint
13 April 2012

13 April 2012

Quick facts

Lead sponsor	Organon and Co
Phase	Phase 3
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	double
Primary purpose	treatment
Enrollment	138
Start date	21 May 2009
Primary completion	13 April 2012
Estimated completion	13 April 2012

Drugs / interventions tested

• ezetimibe (EZETIMIBE) — full drug profile →
• Placebo

Conditions studied

• Primary Hypercholesterolemia — all drugs for Primary Hypercholesterolemia →

Sponsor

Organon and Co — full company profile →

Who can join

Adults 6 to 10, any sex, with Primary Hypercholesterolemia. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Percentage Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) at Week 12 Primary · Baseline and Week 12

Serum LDL-C levels calculated at baseline and after 12 weeks of study drug administration. LDL-C were calculated by the method of Friedewald equation, LDL-C = Total Cholesterol (TC) - (High-density lipoprotein cholesterol \[HDL-C\] + triglyceride \[TG\]/5).

Group	Value	95% CI
Ezetimibe	-27.70	-30.80 – -24.59
Placebo	-0.95	-4.94 – 3.04

Percentage Change From Baseline in Total Cholesterol (TC) at Week 12 Secondary · Baseline and Week 12

Serum TC levels measured using enzymatic methods at baseline and after 12 weeks of study drug administration.

Group	Value	95% CI
Ezetimibe	-20.74	-23.29 – -18.18
Placebo	0.19	-3.07 – 3.44

Percentage Change From Baseline in Apolipoprotein B (Apo B) at Week 12 Secondary · Baseline and Week 12

Serum Apo B measured at baseline and after 12 weeks of study drug administration.

Group	Value	95% CI
Ezetimibe	-21.66	-24.94 – -18.38
Placebo	-1.42	-5.55 – 2.70

Percentage Change From Baseline High-density Lipoprotein Cholesterol (HDL-C) at Week 12 Secondary · Baseline and Week 12

Serum HDL-C levels measured by photometry after precipitation at baseline and after 12 weeks of study drug administration.

Group	Value	95% CI
Ezetimibe	2.11	-2.27 – 6.48
Placebo	1.41	-4.14 – 6.96

Percentage Change From Baseline in Non-HDL-C at Week 12 Secondary · Baseline and Week 12

Serum Non-HDL-C calculated at baseline and after 12 weeks of study drug administration. Non-HDL-C values were calculated as follows: Non-HDL-C (mg/dL) = TC - HDL-C.

Group	Value	95% CI
Ezetimibe	-25.47	-28.45 – -22.49
Placebo	0.28	-3.52 – 4.08

Percentage Change From Baseline in Triglycerides (TG) at Week 12 Secondary · Baseline and Week 12

Serum TG levels measured using enzymatic methods at baseline and after 12 weeks of study drug.

Group	Value	95% CI
Ezetimibe	-6.23	-12.73 – 0.75
Placebo	8.44	-2.03 – 20.03

Percent Change From Baseline in LDL-C at Week 2 Secondary · Baseline and Week 2

Serum LDL-C levels calculated at baseline and after 2 weeks of study drug administration. LDL-C were calculated by the method of Friedewald equation, LDL-C = Total Cholesterol (TC) - (High-density lipoprotein cholesterol \[HDL-C\] + triglyceride \[TG\]/5).

Group	Value	95% CI
Ezetimibe	-24.75	-27.80 – -21.69
Placebo	0.23	-3.74 – 4.20

Percent Change From Baseline in LDL-C at Week 4 Secondary · Baseline and Week 4

Serum LDL-C levels calculated at baseline and after 4 weeks of study drug administration. LDL-C were calculated by the method of Friedewald equation, LDL-C = Total Cholesterol (TC) - (High-density lipoprotein cholesterol \[HDL-C\] + triglyceride \[TG\]/5).

Group	Value	95% CI
Ezetimibe	-26.93	-29.81 – -24.05
Placebo	-2.99	-6.66 – 0.68

Percent Change From Baseline in LDL-C at Week 8 Secondary · Baseline and Week 8

Serum LDL-C levels calculated at baseline and after 8 weeks of study drug administration. LDL-C were calculated by the method of Friedewald equation, LDL-C = Total Cholesterol (TC) - (High-density lipoprotein cholesterol \[HDL-C\] + triglyceride \[TG\]/5).

Group	Value	95% CI
Ezetimibe	-27.22	-30.30 – -24.15
Placebo	0.49	-3.46 – 4.43

Percentage Change From Baseline in TC at Week 2 Secondary · Baseline and Week 2

Serum TC levels measured using enzymatic methods at baseline and after 2 weeks of study drug administration.

Group	Value	95% CI
Ezetimibe	-18.69	-21.21 – -16.18
Placebo	1.40	-1.83 – 4.63

Percentage Change From Baseline in TC at Week 4 Secondary · Baseline and Week 4

Serum TC levels measured using enzymatic methods at baseline and after 4 weeks of study drug administration.

Group	Value	95% CI
Ezetimibe	-20.57	-23.05 – -18.08
Placebo	-0.29	-3.45 – 2.86

Percentage Change From Baseline in TC at Week 8 Secondary · Baseline and Week 8

Serum TC levels measured using enzymatic methods at baseline and after 8 weeks of study drug administration.

Group	Value	95% CI
Ezetimibe	-20.84	-23.40 – -18.27
Placebo	0.54	-2.73 – 3.81

Adverse events — posted to ClinicalTrials.gov

Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Ezetimibe

Serious: 2/92 (2%)

Deaths: —

Placebo

Serious: 0/45 (0%)

Deaths: —

Serious adverse events (2 terms)

Reaction	System	Ezetimibe	Placebo
Epilepsy congenital	Congenital, familial and genetic disorders	—	—
Appendicitis	Infections and infestations	—	—

Other adverse events (8 terms — click to expand)

Reaction	System	Ezetimibe	Placebo
Nasopharyngitis	Infections and infestations	—	—
Upper respiratory tract infection	Infections and infestations	—	—
Headache	Nervous system disorders	—	—
Abdominal pain	Gastrointestinal disorders	—	—
Influenza	Infections and infestations	—	—
Diarrhoea	Gastrointestinal disorders	—	—
Nausea	Gastrointestinal disorders	—	—
Vomiting	Gastrointestinal disorders	—	—

Most-reported serious reactions: Epilepsy congenital, Appendicitis.

Data from ClinicalTrials.gov NCT00867165 adverse events section.

Sponsor's own description

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.

Publications & conference data

1 peer-reviewed publication reference this trial (live from Europe PMC):

1. Efficacy and safety of ezetimibe monotherapy in children with heterozygous familial or nonfamilial hypercholesterolemia.
   Kusters DM, Caceres M, Coll M, Cuffie C, et al · · 2015 · cited 47× · PMID 25841542 · DOI 10.1016/j.jpeds.2015.02.043