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

NCT03510884

CompletedPhase 3

Sponsor: Sanofi

Conditions: Hypercholesterolaemia

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

Countries: United States, Argentina, Austria, Brazil, Bulgaria, Canada, Czechia, Denmark

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 overview

Sex: ALL

Age: 8 Years to 17 Years

Healthy volunteers: No

Study type: INTERVENTIONAL

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.
Locations (43)
  • Boca Raton, Florida, United States
  • St Louis, Missouri, United States
  • Charlotte, North Carolina, United States
  • Cincinnati, Ohio, United States
  • Nashville, Tennessee, United States
  • Buenos Aires, Argentina
  • Vienna, Austria
  • Porto Alegre, Rio Grande do Sul, Brazil
  • São Paulo, Brazil
  • Plovdiv, Bulgaria
  • Québec, Canada
  • Brno, Czechia
  • Praha 5 - Motol, Czechia
  • Copenhagen, Denmark
  • HUS, Finland
  • Bron, France
  • Nantes, France
  • Budapest, Hungary
  • Milan, Italy
  • Palermo, Italy
  • Roma, Italy
  • Beirut, Lebanon
  • Room Hospital Street, Achrafie, Lebanon
  • Guadalajara, Jalisco, Mexico
  • Oaxaca City, Mexico
  • Amsterdam, Netherlands
  • Oslo, Norway
  • Gdansk, Pomeranian Voivodeship, Poland
  • Lodz, Poland
  • Kazan', Russia
  • + 13 more on CT.gov