An 8-Week Dose-Finding Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents With Heterozygous Familial Hypercholesterolemia

• Status: Completed
• Trial end date: 2019-02-22
• Target enrollment: 0
• Participant gender: All

Summary

Primary Objective:

To evaluate the effect of alirocumab on low-density lipoprotein cholesterol (LDL-C) levels after 8 weeks of treatment in heterozygous familial hypercholesterolemia (heFH) participants aged of 8 to 17 years, with LDL-C >=130 milligrams per deciliter (mg/dL) (3.37 millimoles per litre [mmol/L]) 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 for at least 4 weeks prior to the screening period.

Secondary Objective:

• To evaluate the safety and tolerability of alirocumab.

• To evaluate the pharmacokinetics profile of alirocumab.

• To evaluate the effects of alirocumab on other lipid parameters.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Sanofi
• Collaborator: Regeneron Pharmaceuticals
• Treatments: Antibodies, Monoclonal; Cholestyramine Resin; Ezetimibe; Fenofibrate; Hydroxymethylglutaryl-CoA Reductase Inhibitors; Niacin; Niacinamide; Nicotinic Acids

Criteria

Inclusion criteria :

• Children and adolescent male and female participants aged of 8 to 17 years at the time of signed informed consent. For Russia only: Male and female participants aged >=12 and <=17 years at the time of signed informed consent.

• Participants with diagnosis of heterozygous familial hypercholesterolemia (heFH) through genotyping or clinical criteria.

• Participants 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.

• Participants with calculated LDL-C greater than or equal to 130 mg/dL (>=3.37 mmol/L) at the screening visit.

• Participants with body weight greater than or equal to 25 kg.

• Participants aged of 8 to 9 years to be at Tanner stage 1 and participants 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 participant assent.

Exclusion criteria:

• Participant with secondary hyperlipidemia.

• Diagnosis of homozygous familial hypercholesterolemia.

• Participant who had 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).

• Severe renal impairment (i.e., 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 participant's potential participation in a clinical trial.