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

Sponsor
Sanofi
Study ID
NCT03510715
Phase
PHASE3
Status
Completed

Conditions

Eligibility Criteria

Sex
ALL
Age
8 Years - 17 Years
Healthy Volunteers
Not accepted

Interventions

  • Alirocumab SAR236553 (REGN727) — DRUG
    Pharmaceutical form: solution for injection in pre-filled syringe, Route of administration: subcutaneous (SC)
  • Atorvastatin — DRUG
    Pharmaceutical form: tablet, Route of administration: oral
  • Simvastatin — DRUG
    Pharmaceutical form: tablet, Route of administration: oral
  • Fluvastatin — DRUG
    Pharmaceutical form: capsule, Route of administration: oral
  • Pravastatin — DRUG
    Pharmaceutical form: tablet, Route of administration: oral
  • Lovastatin — DRUG
    Pharmaceutical form: tablet, Route of administration: oral
  • Rosuvastatin — DRUG
    Pharmaceutical form: tablet, Route of administration: oral
  • Ezetimibe — DRUG
    Pharmaceutical form: tablet, Route of administration: oral
  • Cholestyramine — DRUG
    Pharmaceutical form: oral suspension, Route of administration: oral
  • Nicotinic acid — DRUG
    Pharmaceutical form: tablet, Route of administration: oral
  • Fenofibrate — DRUG
    Pharmaceutical form: tablet, Route of administration: oral
  • Omega-3 fatty acids — DRUG
    Pharmaceutical form: capsule, Route of administration: oral

Study Details

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.

Key Dates

Start date
Aug 31, 2018
Status verified
Oct 2020
Primary completion
Feb 17, 2020
Completion
Feb 17, 2020

Study Design

Enrollment
18 participants (actual)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Alirocumab
    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.

Primary Outcome Measure

Percent Change From Baseline in Low-Density Lipoprotein Cholesterol (LDL-C) at Week 12: Intent-to-Treat (ITT) Analysis [ Time Frame: Baseline to Week 12 ]

Related Studies