An 8-Week Open-Label, Sequential, Repeated Dose-Finding Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents with Heterozygous Familial Hypercholesterolemia Followed by an Extension Phase

Phase 2

Completed

Conditions: familiaire hypercholesterolemie
Familial hypercholesterolemia
high cholesterol

Registration Number: NL-OMON47221

Lead Sponsor: Sanofi-aventis

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Completed

Sex: Not specified

Target Recruitment: 9

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

- 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 <30 mL/min/1.73 m2 at the screening visit [Week -2]).
- ALT or AST >2 x ULN (1 repeat lab is allowed).
- CPK >3 x ULN (1 repeat lab is allowed).

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Percent change in calculated LDL-C</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>- Absolute change in calculated LDL-C<br /><br>- Percentage of participants achieving a calculated LDL-C level lower than 130<br /><br>mg/dL (3.37 mmol/L)<br /><br>- Percentage of participants achieving a calculated LDL-C level lower than 110<br /><br>mg/dL (2.84 mmol/L)<br /><br>- Percent change in Apolipoprotein B (Apo B)<br /><br>- Percent change in non-high density lipoprotein cholesterol (non-HDL-C)<br /><br>- Percent change in Total-C<br /><br>- Percent change in Lipoprotein (a) (Lp[a])<br /><br>- Percent change in triglycerides (TG)<br /><br>- Percent change in HDL-C<br /><br>- Percent change in Apo A-1<br /><br>- Absolute change in Apo B<br /><br>- Absolute change in non-HDL-C<br /><br>- Absolute change in Total-C<br /><br>- Absolute change in Lp(a)<br /><br>- Absolute change in TG<br /><br>- Absolute change in HDL-C<br /><br>- Absolute change in Apo A-1<br /><br>- Absolute change in ratio Apo B/Apo A-1</p><br>