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

A randomized double-blind placebo controlled trial to evaluate the safety and efficacy of the cholesterol absorption inhibitor, ezetimibe in a dosage of 10 mg, on plasma lipids, plant sterols and inflammatory markers in children with familial hypercholesterolemia.

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

NL-OMON

Registry ID

NL-OMON25426

Enrollment

Registered

2006-04-04

Start date

2006-08-01

Completion date

Unknown

Last updated

2024-02-28

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Familial hypercholesterolemia

Interventions

Ezetimibe 10 mg/day or placebo treatment during 4 months.

Eligibility

Inclusion criteria

Inclusion criteria: 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.

Exclusion criteria

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 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.

Design outcomes

Primary

Measure	Time frame
Primary endpoint will be the efficacy towards LDL-C levels and the safety of 10 mg ezetimibe.	—

Secondary

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

Contacts

Public ContactM.D. Trip

Academic Medical Center (AMC), Department of Cardiology, F4- 109, P.O. Box 22660

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

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP)