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

• Status: Active, not recruiting
• Trial end date: 2022-07-01
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Sanofi
• Collaborator: Regeneron Pharmaceuticals
• Treatments: Antibodies, Monoclonal; Atorvastatin; Atorvastatin Calcium; Cholestyramine Resin; Ezetimibe; Fenofibrate; Fluvastatin; Lovastatin; Niacin; Niacinamide; Nicotinic Acids; Pravastatin; Rosuvastatin Calcium; Simvastatin

Criteria

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.