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

Completed · Phase 3 · Has a placebo group

Conditions studied: Hypercholesterolaemia

In brief

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.

Key facts

Study ID

NCT03510884

Run by

Sanofi

People needed

153

Starts

2018-05-31

Expected to finish

2022-08-05

Last updated by the study team

2023-05-06

Who can join

Age: 8 and older, up to 17. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• Excel Medical Clinical Trials, LLC-Site Number:8400001 — Boca Raton, Florida, United States
• Washington University School of Medicine-Site Number:8400006 — St Louis, Missouri, United States
• Presbyterian Novant Heart & Wellness-Site Number:8400002 — Charlotte, North Carolina, United States
• Cincinnati Children's Hospital Medical Center-Site Number:8400005 — Cincinnati, Ohio, United States
• Vanderbilt University-Site Number:8400003 — Nashville, Tennessee, United States
• Investigational Site Number :0320001 — Buenos Aires, Argentina
• Investigational Site Number :0400001 — Vienna, Austria
• Investigational Site Number :0760004 — Porto Alegre, Rio Grande do Sul, Brazil
• Investigational Site Number :0760001 — São Paulo, Brazil
• Investigational Site Number :1000002 — Plovdiv, Bulgaria
• Investigational Site Number :1240001 — Québec, Canada
• Investigational Site Number :2030002 — Brno, Czechia
• Investigational Site Number :2030001 — Praha 5 - Motol, Czechia
• Investigational Site Number :2080001 — Copenhagen, Denmark
• Investigational Site Number :2460001 — HUS, Finland
• Investigational Site Number :2500001 — Bron, France
• Investigational Site Number :2500002 — Nantes, France
• Investigational Site Number :3480001 — Budapest, Hungary
• Investigational Site Number :3800003 — Milan, Italy
• Investigational Site Number :3800001 — Palermo, Italy
• Investigational Site Number :3800002 — Roma, Italy
• Investigational Site Number :4220001 — Beirut, Lebanon
• Investigational Site Number :4220003 — Room Hospital Street, Achrafie, Lebanon
• Investigational Site Number :4840008 — Guadalajara, Jalisco, Mexico
• Investigational Site Number :4840007 — Oaxaca City, Mexico

Full record on ClinicalTrials.gov

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