Efficacy and safety of ezetimibe in young children with familial hypercholesterolemia

Completed

• Conditions: Familial hypercholesterolemia (FH); Nutritional, Metabolic, Endocrine; Familial hypercholesterolemia

• Registration Number: ISRCTN39762831
• Lead Sponsor: Academic Medical Center (AMC) (The Netherlands)

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2006-06-07
• Last Update Posted: 13 January 2015

Recruitment & Eligibility

• Status: Completed
• Sex: All
• Target Recruitment: 70

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

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

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Primary endpoint will be the efficacy towards LDL-C levels and the safety of 10 mg ezetimibe.

Secondary Outcome Measures

Name	Time	Method
Secondary endpoint will be the effect of 10 mg ezetimibe on inflammatory markers and plant sterols in plasma.