An Efficacy and Safety Study of Alirocumab in Children and Adolescents With Heterozygous Familial Hypercholesterolemia
Condition: Hypercholesterolaemia
MeSH terms: Hyperlipoproteinemia Type II; Hypercholesterolemia
Intervention: Alirocumab SAR236553 (REGN727); Rosuvastatin; Atorvastatin; Simvastatin; Pravastatin; Lovastatin; Fluvastatin; Ezetimibe; Cholestyramine; Nicotinic acid; Fenofibrate; Omega-3 fatty acids; Placebo
Intervention description: Pharmaceutical form:solution Route of administration: subcutaneous injection; Pharmaceutical form:tablet Route of administration: oral; Pharmaceutical form:Tablet Route of administration: Oral; Pharmaceutical form:Tablet Route of administration: Oral; Pharmaceutical form:Tablet Route of administration: Oral; Pharmaceutical form:Tablet Route of administration: Oral; Pharmaceutical form:Capsule Route of administration: Oral; Pharmaceutical form:Tablet Route of administration: Oral; Pharmaceutical form:oral suspension Route of administration: oral; Pharmaceutical form:Tablet Route of administration: Oral; Pharmaceutical form:Tablet Route of administration: Oral; Pharmaceutical form:capsule Route of administration: oral; Pharmaceutical form:solution Route of administration: subcutaneous injection
Intervention MeSH terms: Nicotinic Acids; Niacin; Niacinamide; Atorvastatin; Rosuvastatin Calcium; Simvastatin; Ezetimibe; Pravastatin; Alirocumab; Lovastatin; Cholestyramine Resin; Fenofibrate
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Enrollment: 153.0
Sex: All
Minimum age: 8 Years
Maximum age: 17 Years
Healthy volunteers: No

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 participants 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 Objectives:

        -  To evaluate the efficacy of alirocumab versus placebo on 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.
    

Eligibility criteria:

        Inclusion criteria:

          -  Children and adolescent male and female participants 8 to 17 years of age at the time
             of signed informed consent.

          -  Participants with diagnosis of 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 except for participants who have previously participated in the
             DFI14223 (NCT02890992) study.

          -  A signed informed consent indicating parental permission with or without participant
             assent.

        Exclusion criteria:

          -  Participant with body weight < 25 kg.

          -  Participants aged of 8 to 9 years not at Tanner stage 1 and participants aged of 10 to
             17 years not at least at Tanner stage 2 in their development.

          -  Participants 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.

          -  Participants with uncontrolled type 1 or type 2 diabetes mellitus.

          -  Participants with known uncontrolled thyroid disease.

          -  Participants with uncontrolled hypertension.

          -  Fasting triglycerides greater than (>) 350 mg/dL (3.95 mmol/L).

          -  Severe renal impairment (ie, estimated glomerular filtration rate <30 mL/min/1.73
             m^2).

          -  Alanine aminotransferase or aspartate aminotransferase >2*upper limit of normal (ULN).

          -  Creatinine phosphokinase (CPK) >3*ULN.

        The above information was not intended to contain all considerations relevant to a
        participant's potential participation in a clinical trial.