Efficacy and safety of ezetimibe in young children with familial hypercholesterolemia.

1. Males or females;

2. Aged 8-14 years;

3. Heterozygous Familial hypercholesterolemia defined as:

a. Molecular diagnosis of FH AND 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; OR

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; AND

c. One parent with either a clinical or molecular diagnosis of FH.

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 mm Hg or diastolic > 100 mm Hg);

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, ASAT or ALAT, must be < 1.5 times the upper limit of normal (ULN) using the central laboratory reference range;

14. CK levels must be <1.5 times the ULN using the central laboratory reference range.