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

NCT03510715

CompletedPhase 3

Sponsor: Sanofi

Conditions: Hypercholesterolemia

Interventions: Alirocumab SAR236553 (REGN727), Atorvastatin, Simvastatin, Fluvastatin, Pravastatin

Countries: Brazil, Canada, Denmark, Mexico, Netherlands, Russia, Slovenia, Spain

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.

Eligibility overview

Sex: ALL

Age: 8 Years to 17 Years

Healthy volunteers: No

Study type: INTERVENTIONAL

Eligibility 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.
Locations (10)
  • São Paulo, Brazil
  • Québec, Canada
  • Viborg, Denmark
  • Oaxaca City, Mexico
  • Amsterdam, Netherlands
  • Kemerovo, Russia
  • Ljubljana, Slovenia
  • A Coruña, Spain
  • Taipei, Taiwan
  • Izmir, Turkey (Türkiye)