Trial reportArteriosclerosis, thrombosis, and vascular biology2022
Efficacy and Safety of Alirocumab in Children and Adolescents With Homozygous Familial Hypercholesterolemia: Phase 3, Multinational Open-Label Study.
Trial report in Arteriosclerosis, thrombosis, and vascular biology, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03510715 (An Open-Label Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents With Homozygous Familial Hypercholesterolemia), which is not on this map. Cited by 17 papers, 3 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
An Open-Label Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents With Homozygous Familial Hypercholesterolemia
Who cites it
17 citing papers in PubMed, 3 syntheses or guidelines pooled it, 39 citations in OpenAlex.
- Pooled it
- AlirocumabCurrent cardiology reviews · 2026Pooled it
- Efficacy and Safety of Evolocumab and Alirocumab as PCSK9 Inhibitors in Pediatric Patients with Familial Hypercholesterolemia: A Systematic Review and Meta-Analysis.Medicina (Kaunas, Lithuania) · 2024Pooled it
- Pediatric Familial Hypercholesterolemia: Targeting Intestinal Absorption and Other Therapeutic Strategies.Nutrients · 2025Review
- Clinical Management of Dyslipidemia in Infants and Toddlers.Current atherosclerosis reports · 2025Review
- Molecular Therapeutics in Development to Treat Hyperlipoproteinemia.Molecular diagnosis & therapy · 2025Review
- Dyslipidemia in Children and Adolescents: Current Insights and Updated Treatment Approaches.Pediatric gastroenterology, hepatology & nutrition · 2025Review
- Transitioning Adolescents and Young Adults with Lipid Disorders to Adult Health Care.Current atherosclerosis reports · 2024Review
- Alirocumab: Pediatric First Approval.Paediatric drugs · 2024Review
- Hypercholesterolemia: a literature review on management using tafolecimab: a novel member of PCSK9 monoclonal antibodies.Annals of medicine and surgery (2012) · 2024Review
- Comprehensive Assessment of PCSK9 Inhibitors for Lipid Management: Scientific Guidance Based on Drug Selection Recommendations for Chinese Medical Institutions.Drug design, development and therapy · 2024Article
- International Atherosclerosis Society guidance for implementing best practice in the care of familial hypercholesterolaemia.Nature reviews. Cardiology · 2023Review
- Dysregulation of Cholesterol Homeostasis in Ovarian Cancer.Current oncology (Toronto, Ont.) · 2023Review
- In vitro assessment of the pathogenicity of the LDLR c.2160delC variant in familial hypercholesterolemia.Lipids in health and disease · 2023Article
- Contemporary Homozygous Familial Hypercholesterolemia in the United States: Insights From the CASCADE FH Registry.Journal of the American Heart Association · 2023Article
- Review
- Atherogenic Dyslipidemias: Unmet Needs and the Therapeutic Potential of Emerging and Novel Approaches and Drugs.Pharmaceuticals (Basel, Switzerland) · 2023Review
Corrections and comments
- Commented on by
Authors and funding
10 authors at 9 institutions in 5 countries.
Funding
Abstract
backgroundDespite progress in treating homozygous familial hypercholesterolemia, most patients do not achieve low-density lipoprotein cholesterol (LDL-C) targets. This study examined efficacy and safety of the PCSK9 (proprotein convertase subtilisin/kexin type 9) inhibitor, alirocumab, in pediatric patients (aged 8-17 years) with inadequately controlled homozygous familial hypercholesterolemia.
methodsIn this open-label, single-arm, multinational, Phase 3 study, patients (n=18) received alirocumab 75 mg or 150 mg (bodyweight <50 kg/≥50 kg) every 2 weeks as an adjunct to background treatment. The primary endpoint was percent change in LDL-C from baseline to Week 12. Secondary endpoints included changes in LDL-C and other lipid parameters up to 48 weeks, safety/tolerability, and alirocumab pharmacokinetics.
resultsThe mean age of patients was 12.4 years; 16/18 (89%) had mutations in the low-density lipoprotein receptor gene
conclusionsThe study supports the efficacy and safety of alirocumab as a potential adjunct to treatment for some pediatric patients with homozygous familial hypercholesterolemia. REGISTRATION: URL: https://www. CLINICALTRIALS: gov; NCT03510715.
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What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.