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

Sanofi logo


Status and phase

Phase 3




Drug: Pravastatin
Drug: Atorvastatin
Drug: Cholestyramine
Drug: Lovastatin
Drug: Ezetimibe
Drug: Simvastatin
Drug: Rosuvastatin
Drug: Omega-3 fatty acids
Drug: Fenofibrate
Drug: Nicotinic acid
Drug: Alirocumab SAR236553 (REGN727)
Drug: Fluvastatin

Study type


Funder types



2017-002297-39 (EudraCT Number)
U1111-1200-2046 (Other Identifier)

Details and patient eligibility


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.

Full description

The study duration was up to 62 weeks, which included (if needed) a run-in period of up to 4 weeks, a screening period of up to 2 weeks, a treatment period of up to 48 weeks, and a follow-up of 8 weeks.


18 patients




8 to 17 years old


No Healthy Volunteers

Inclusion and exclusion 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.

Trial design

18 participants in 1 patient group

Experimental group
Participants with BW less than (<) 50 kilograms (kg) received subcutaneous (SC) injection of alirocumab 75 mg Q2W for 48 weeks. Alirocumab dose was up-titrated to 150 mg Q2W from Week 12 in case of increase in BW with BW greater than or equal to [>=] 50 kg. Participants with BW >=50 kg received SC injection of alirocumab 150 mg Q2W for 48 weeks.
Drug: Fluvastatin
Drug: Omega-3 fatty acids
Drug: Rosuvastatin
Drug: Alirocumab SAR236553 (REGN727)
Drug: Nicotinic acid
Drug: Fenofibrate
Drug: Ezetimibe
Drug: Simvastatin
Drug: Cholestyramine
Drug: Lovastatin
Drug: Atorvastatin
Drug: Pravastatin

Trial documents

Trial contacts and locations



Data sourced from

Clinical trials

Find clinical trialsTrials by location


© Copyright 2024 Veeva Systems