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

• Status: Completed
• Trial end date: 2020-02-17
• Target enrollment: 0
• Participant gender: All

Summary

Primary Objective:

To evaluate the efficacy of alirocumab (75 or 150 milligrams [mg] depending on body weight [BW]), administered every 2 weeks (Q2W), on low-density lipoprotein cholesterol (LDL-C) levels at Week 12 of treatment in children and adolescents with homozygous familial hypercholesterolemia (hoFH) of 8 to 17 years of age on top of background treatments.

Secondary Objectives:

• To evaluate the efficacy of alirocumab after 24 and 48 weeks of treatment on LDL-C levels.

• To evaluate the effects of alirocumab on other lipid parameters (eg, apolipoprotein B [Apo B], non-high density lipoprotein cholesterol [non-HDL-C], total cholesterol [Total-C], high density lipoprotein cholesterol [HDL-C], lipoprotein a [Lp (a)], triglycerides [TG], apolipoprotein A-1 [Apo A-1] levels) after 12, 24, and 48 weeks of treatment.

• To evaluate the safety and tolerability of alirocumab up to 48 weeks of treatment.

• 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 :

• Participants genetically diagnosed with hoFH.

• Participants treated with optimal dose of statin +/- other lipid modifying therapies (LMTs), or non-statin LMTs if statin-intolerant at stable dose(s) for at least 4 weeks.

• A signed informed consent indicating parental permission with or without participants assent.

• For participants on apheresis, currently undergoing stable LDL apheresis therapy prior to the screening visit (Week -2) and had initiated apheresis treatment for at least 6 months.

Exclusion criteria:

• Participants with LDL-C <130 milligram per deciliter [mg/dL] (3.37 millimoles per liter [mmol/L]) obtained during the screening period after the participant had been on stable apheresis procedure or LMT (i.e., stable optimal dose of statin ± other stable LMTs, or stable non statin LMTs in statin-intolerant participants) treatment for at least 4 weeks.

• Participants with BW <25 kg.

• Participants aged 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 uncontrolled Type 1 or 2 diabetes mellitus.

• Participants with known uncontrolled thyroid disease.

• Participants with uncontrolled hypertension.

• Participants who will receive statin de novo during the run-in period.

• Fasting triglycerides greater than (>) 350 mg/dL (3.95 mmol/L) at the screening visit.

• Severe renal impairment (i.e., estimated glomerular filtration rate <30 milliliter per minute/1.73 meter square) at the screening visit.

• Alanine aminotransferase or aspartate aminotransferase >2 * upper limit of normal (ULN) at the screening visit.

• Creatine phosphokinase >3 * ULN at the screening visit.

The above information was not intended to contain all considerations relevant to a participants potential participation in a clinical trial.