Efficacy and safety of ezetimibe in young children with familial hypercholesterolemia

ISRCTN	ISRCTN39762831
DOI	https://doi.org/10.1186/ISRCTN39762831
Protocol serial number	N/A
Sponsor	Academic Medical Center (AMC) (The Netherlands)
Funders	Merck Sharp and Dohme BV (MSD), Schering-Plough

Submission date: 07/06/2006
Registration date: 07/06/2006
Last edited: 07/06/2006

Recruitment status: No longer recruiting
Overall study status: Completed
Condition category: Nutritional, Metabolic, Endocrine

Prospectively registered

Protocol

Statistical analysis plan

Results

Individual participant data

Record updated in last year

Plain English summary of protocol

Not provided at time of registration

Contact information

Dr M.D. Trip
Scientific

Academic Medical Center (AMC)
Department of Cardiology
F4- 109
P.O. Box 22660
Amsterdam
1100 DD
Netherlands

Phone	+31 (0)20 5665882 or 020-5669111
Email	m.d.trip@amc.uva.nl

Study information

Primary study design	Interventional
Study design	Randomized, placebo-controlled trial
Secondary study design	Randomised controlled trial
Scientific title
Study acronym	EZKIMO
Study objectives	Ezetimibe monotherapy lowers low density lipoprotein-cholesterol (LDL-C) levels, plant sterol levels and inflammatory markers in young children with familial hypercholesterolemia (FH).
Ethics approval(s)	Not provided at time of registration
Health condition(s) or problem(s) studied	Familial hypercholesterolemia (FH)
Intervention	Ezetimibe 10 mg/day versus placebo treatment for 4 months
Intervention type	Drug
Phase	Not Specified
Drug / device / biological / vaccine name(s)	Ezetimibe
Primary outcome measure(s)	Primary endpoint will be the efficacy towards LDL-C levels and the safety of 10 mg ezetimibe.
Key secondary outcome measure(s)	Secondary endpoint will be the effect of 10 mg ezetimibe on inflammatory markers and plant sterols in plasma.
Completion date	01/08/2007

Eligibility

Participant type(s)	Patient
Age group	Child
Lower age limit	8 Years
Upper age limit	14 Years
Sex	All
Target sample size at registration	70
Key inclusion criteria	1. Male or female 2. Aged 8-14 years 3. Heterozygous familial hypercholesterolemia defined as: a. Molecular diagnosis of FH AND LDL-C above 95th percentile for age and sex (LDL-C >3.88 mmol/l) despite a lipid-lowering diet for at least 3 months b. LDL-cholesterol above 95th percentile for age and sex (LDL-C >3.88 mmol/l) despite a lipid-lowering diet for at least 3 months c. One parent with either a clinical or molecular diagnosis of FH
Key exclusion criteria	1. Homozygous familial hypercholesterolemia 2. Diseases that cause a secondary increase in LDL-C, such as diabetes mellitus, anorexia nervosa and renal, hepatic or thyroid disease 3. Length below the 3rd percentile for age and sex 4. Weight-compared-to-length above the 97th percentile for age and sex 5. Serious illness in the previous three months 6. Major surgery in the previous three months 7. Partial ileal bypass or any gastrointestinal disease that might interfere with drug absorption 8. Plasma triglycerides above 4.0 mmol/l 9. Hypertension (systolic >160 mmHg or diastolic >100 mmHg) 10. Psychological disorders that might interfere with adherence to the protocol 11. Pregnancy at baseline 12. History of allergy or sensitivity to ezetimibe 13. Liver function tests, aspartate aminotransferase or alanine aminotransferase (ASAT or ALAT), must be <1.5 times the upper limit of normal (ULN) using the central laboratory reference range 14. Creatinine clearance levels must be <1.5 times the ULN using the central laboratory reference range
Date of first enrolment	01/08/2006
Date of final enrolment	01/08/2007

Locations

Countries of recruitment

Netherlands

Study participating centre

Academic Medical Center (AMC)

Amsterdam
1100 DD
Netherlands

Results and Publications

Individual participant data (IPD) Intention to share	No
IPD sharing plan summary	Not provided at time of registration
IPD sharing plan