An Efficacy and Safety Study of Alirocumab in Children and Adolescents With Heterozygous Familial Hypercholesterolemia



Status:Recruiting
Conditions:High Cholesterol
Therapuetic Areas:Cardiology / Vascular Diseases
Healthy:No
Age Range:8 - 17
Updated:3/27/2019
Start Date:May 31, 2018
End Date:March 2022
Contact:Trial Transparency email recommended (Toll free number for US & Canada)
Email:Contact-Us@sanofi.com
Phone:800-633-1610

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A Randomized, Double-Blind, Placebo-Controlled Study Followed by an Open Label Treatment Period to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents With Heterozygous Familial Hypercholesterolemia

Primary Objective:

To evaluate the efficacy of alirocumab administered every 2 weeks (Q2W) and every 4 weeks
(Q4W) versus placebo after 24 weeks of double-blind (DB) treatment on low-density lipoprotein
cholesterol (LDL-C) levels in patients with heterozygous familial hypercholesterolemia (heFH)
8 to 17 years of age on optimal stable daily dose of statin therapy ± other lipid modifying
therapies (LMTs) or a stable dose of non-statin LMTs in case of intolerance to statins.

Secondary Objective:

- To evaluate the efficacy of alirocumab versus placebo on low-density lipoprotein
cholesterol (LDL-C) levels.

- To evaluate the effects of alirocumab versus placebo on other lipid parameters.

- To evaluate the safety and tolerability of alirocumab in comparison with placebo.

- To evaluate the efficacy, safety, and tolerability of alirocumab after open label
treatment.

- To evaluate the development of anti-alirocumab antibodies.

The study duration is approximately up to 110 weeks (run-in period [if needed]: up to 4 weeks
[+2 days], screening period: up to 2 weeks [+5 days], double-blind treatment period: 24
weeks, open label treatment period: 80 weeks).

Inclusion criteria :

- Children and adolescent male and female patients aged 8 to 17 years at the time of
signed informed consent.

- Patients with diagnosis of heterozygous familial hypercholesterolemia (heFH) through
genotyping or clinical criteria.

- Patients treated with optimal dose of statin +/- other LMT(s) or non-statin LMT(s) if
statin intolerant at stable dose for at least 4 weeks prior to screening lipid
sampling.

- Patients with calculated LDL-C greater than or equal to 130 mg/dL (≥3.37 mmol/L) at
the screening visit except for patients who have previously participated in the
DFI14223 study.

- A signed informed consent indicating parental permission with or without patient
assent.

Exclusion criteria:

- Patient with body weight less than 25 kg.

- Patients aged of 8 to 9 years not at Tanner stage 1 and patients aged of 10 to 17
years not at least at Tanner stage 2 in their development.

- Patients 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.

- Patients with uncontrolled type 1 or type 2 diabetes mellitus.

- Patients with known uncontrolled thyroid disease.

- Patients with uncontrolled hypertension.

- Fasting triglycerides >350 mg/dL (3.95 mmol/L).

- Severe renal impairment (ie, estimated glomerular filtration rate [eGFR] <30
mL/min/1.73 m^2.

- Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >2 x upper limit of
normal (ULN).

- Creatinine phosphokinase (CPK) >3 x ULN.

The above information is not intended to contain all considerations relevant to a patient's
potential participation in a clinical trial.
We found this trial at
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