Efficacy and safety of ezetimibe in young children with familial hypercholesterolemia
| ISRCTN | ISRCTN39762831 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN39762831 |
| Secondary identifying numbers | N/A |
- 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
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 |
|---|---|
| m.d.trip@amc.uva.nl |
Study information
| Study design | Randomized, placebo-controlled trial |
|---|---|
| Primary study design | Interventional |
| Secondary study design | Randomised controlled trial |
| Study setting(s) | Not specified |
| Study type | Treatment |
| 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 |
| Pharmaceutical study type(s) | |
| Phase | Not Specified |
| Drug / device / biological / vaccine name(s) | Ezetimibe |
| Primary outcome measure | Primary endpoint will be the efficacy towards LDL-C levels and the safety of 10 mg ezetimibe. |
| Secondary outcome measures | Secondary endpoint will be the effect of 10 mg ezetimibe on inflammatory markers and plant sterols in plasma. |
| Overall study start date | 01/08/2006 |
| Completion date | 01/08/2007 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Child |
| Lower age limit | 8 Years |
| Upper age limit | 14 Years |
| Sex | Both |
| Target number of participants | 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
1100 DD
Netherlands
Sponsor information
Academic Medical Center (AMC) (The Netherlands)
University/education
University/education
P.O. Box 22660
Amsterdam
1100 DD
Netherlands
"ROR" |
https://ror.org/03t4gr691 |
|---|
Funders
Funder type
Industry
Merck Sharp and Dohme BV (MSD)
No information available
Schering-Plough
Private sector organisation / For-profit companies (industry)
Private sector organisation / For-profit companies (industry)
- Location
- United States of America
Results and Publications
| Intention to publish date | |
|---|---|
| Individual participant data (IPD) Intention to share | No |
| IPD sharing plan summary | Not provided at time of registration |
| Publication and dissemination plan | Not provided at time of registration |
| IPD sharing plan |
