familiaire hypercholesterolemie Familial hypercholesterolemia high cholesterol
Conditions
Interventions
Cohort 1: Alirocumab SC Q2W
Cohort 2: Alirocumab SC Q2W
Cohort 3: Alirocumab SC Q4W
Cohort 4: Alirocumab SC Q4W
Alirocumab
Children
Dose-finding
Familial hypercholesterolemia
Sponsors
Sanofi-aventis
Eligibility
Age
2 Years to 17 Years
Inclusion criteria
Inclusion criteria: - Children and adolescent male and female patients aged of 8 to 17 years at the time of signed informed consent. - Patients with a diagnosis of heterozygous familial hypercholesterolemia (he FH) through genotyping or clinical criteria. - Patients treated with an optimal dose of statin with or without other LMT(s) or non-statin LMT(s) if statin intolerant at stable dose for at least 4 weeks prior to screening visit (Week -2). - Patients with calculated LDL-C greater than or equal to 130 mg/dL (*3.37 mmol/L) at the screening visit (Week -2). - Patients with body weight greater than or equal to 25 kg. - Patients aged of 8 to 9 years to be at Tanner stage1 and patients aged of 10 to 17 years to be at least at Tanner stage 2 in their development. - A signed informed consent indicating parental permission with or without patient assent, depending on capacity for understanding based on developmental maturity. In cases involving emancipated or mature minors with adequate decision-making capacity, or when otherwise permitted by law, a signed informed consent directly from patients.
Exclusion criteria
Exclusion criteria: - Patient with secondary hyperlipidemia. - Diagnosis of homozygous familial hypercholesterolemia. - Patient who has received lipid apheresis treatment within 2 months prior to the screening period, or has plans to receive it during the study. - Known history of type 1 or type 2 diabetes mellitus. - Known history of thyroid disease. - Known history of hypertension. - Fasting triglycerides >350 mg/dL (3.95 mmol/L) at the screening visit (Week -2). - Severe renal impairment (ie, eGFR 2 x ULN (1 repeat lab is allowed). - CPK >3 x ULN (1 repeat lab is allowed).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Percent change in calculated LDL-C | — |
Secondary
| Measure | Time frame |
|---|---|
| - Absolute change in calculated LDL-C - Percentage of participants achieving a calculated LDL-C level lower than 130 mg/dL (3.37 mmol/L) - Percentage of participants achieving a calculated LDL-C level lower than 110 mg/dL (2.84 mmol/L) - Percent change in Apolipoprotein B (Apo B) - Percent change in non-high density lipoprotein cholesterol (non-HDL-C) - Percent change in Total-C - Percent change in Lipoprotein (a) (Lp[a]) - Percent change in triglycerides (TG) - Percent change in HDL-C - Percent change in Apo A-1 - Absolute change in Apo B - Absolute change in non-HDL-C - Absolute change in Total-C - Absolute change in Lp(a) - Absolute change in TG - Absolute change in HDL-C - Absolute change in Apo A-1 - Absolute change in ratio Apo B/Apo A-1 | — |
Countries
The Netherlands
Outcome results
None listed