The aim of this study is to evaluate the efficacy, safety, compliance and tolerability of 15 years of pravastatin therapy initiated during childhood in young adult FH patients.
ID
Source
Brief title
Condition
- Metabolic and nutritional disorders congenital
- Lipid metabolism disorders
- Arteriosclerosis, stenosis, vascular insufficiency and necrosis
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
- Difference in carotid IMT and aspect of the carotid arteries (echo, MRI)
between FH patients who started with statin therapy in childhood, and their
non-affected siblings after 15 years of follow-up
- The difference in cardiovascular endpoints between FH patients who started
with statin therapy in childhood, and their non-affected siblings after 15
years of follow-up
Secondary outcome
For patients with FH:
- Association between age at start statins and the incidence of cardiovascular
endpoints
- Association between age at start statins and the intima-media thickness and
aspect of the carotid arteries (ultrasound, MRI)
- Incidence of side effects
- Degree of compliance
Background summary
Familial hypercholesterolemia (FH), an hereditary disorder characterized by
elevated levels of low-density lipoprotein cholesterol (LDL-C) from birth
onwards, which strongly predisposes to premature atherosclerosis and
cardiovascular events. This is reflected by an increased carotid intima-media
thickness - a surrogate marker for atherosclerotic vascular disease - in
children with FH.
Statins have been shown to reduce the incidence of CVD in adults and were
expected to be beneficial for children as well. In a previous study, it was
shown that two years of treatment with statins reduced atherosclerosis
progression in children with FH. However, the long-term efficacy and safety of
statin therapy initiated during childhood are unknown.
Study objective
The aim of this study is to evaluate the efficacy, safety, compliance and
tolerability of 15 years of pravastatin therapy initiated during childhood in
young adult FH patients.
Study design
Patients with FH and their unaffected siblings will be requested to visit our
center once. During the visit we will obtain medical history, perform a
physical examination, measure the intima-media thickness of the carotid artery
by ultrasound, perform a MRI of the carotid arteries, and draw blood.
Furthermore, a questionnaire on medication use and family medical history will
be filled out. Participants will be asked for permission to contact their
physicians for information about medication use.
Intervention
none
Study burden and risks
Patients will pay one visit to the AMC of approximately 4 to 5 hours for blood
sampling (50ml), medical history, physical examination, intima-media thickness
measurement of the carotid vessel by ultrasound, and a MRI of the carotid
arteries. We consider this of minimal burden for the participants. Patients who
are not treated according to current best practice will benefit from
participation since their treatment regimens will be optimized according to
current guidelines.
Meibergdreef 9
Amsterdam 1105AZ
NL
Meibergdreef 9
Amsterdam 1105AZ
NL
Listed location countries
Age
Inclusion criteria
Patients with FH who previously participated in a randomized double-blinded placebo-controlled study (JAMA 2004; 292(3):331-7) evaluating the 2-years efficacy and safety of pravastatin, and their 95 unaffected siblings.
Exclusion criteria
none
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| CCMO | NL53903.018.15 |