Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Heterozygous Familial Hypercholesterolemia

Completed · Phase 3 · Has a placebo group

Conditions studied: Familial Hypercholesterolemia - Heterozygous

In brief

This was a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in adolescents with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDL-C).

Key facts

Study ID

NCT04652726

Run by

Novartis Pharmaceuticals

People needed

141

Starts

2021-01-27

Expected to finish

2024-11-27

Last updated by the study team

2026-01-13

Who can join

Age: 12 and older, up to 17. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Heterozygous Familial Hypercholesterolemia (HeFH) diagnosed either by genetic testing or on phenotypic criteria
• Fasting LDL-C >130 mg/dL (3.4 mmol/L) at screening
• Fasting triglycerides <400 mg/dL (4.5 mmol/L) at screening
• On maximally tolerated dose of statin (investigator's discretion) with or without other lipid-lowering therapy; stable for ≥ 30 days before screening

You may not qualify if…

• Homozygous familial hypercholesterolemia (HoFH)
• Active liver disease
• Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome
• Previous treatment with monoclonal antibodies directed towards PCSK9 (within 90 days of screening)
• Recent and/or planned use of other investigational medicinal products or devices

Where it is running

• Icahn School of Medicine at Mount Sinai — New York, New York, United States
• Wake Forest U of Health Sciences — Winston-Salem, North Carolina, United States
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
• Childrens Hospital Pittsburgh of UPMC — Pittsburgh, Pennsylvania, United States
• Primary Children's Medical Center — Salt Lake City, Utah, United States
• Novartis Investigative Site — Formosa, Formosa Province, Argentina
• Unidade de pesquisa clinica - Hospital Universitario Walter Cantidio — Fortaleza, Ceará, Brazil
• Nucleo de Pesquisa Clinica do Rio Grande do Sul — Porto Alegre, Rio Grande do Sul, Brazil
• Setor de Lípides, Aterosclerose e Biologia — São Paulo, São Paulo, Brazil
• Heart Institute (InCOr) HCMFUSP — São Paulo, São Paulo, Brazil
• Novartis Investigative Site — Québec, Quebec, Canada
• Novartis Investigative Site — Prague, Czechia
• Novartis Investigative Site — Prague, Czechia
• Novartis Investigative Site — Besançon, France
• Novartis Investigative Site — Bron, France
• Novartis Investigative Site — Toulouse, France
• Universitaetsmedizin Mannheim — Mannheim, Baden-Wurttemberg, Germany
• KKIM UK Frankfurt/Main — Frankfurt, Germany
• Universitaetsklinikum Freiburg — Freiburg im Breisgau, Germany
• University General Hospital of Ioannina — Ioannina, GR, Greece
• Hippokrateion General Hospital of Athens Greece — Athens, Greece
• Metropolitan Hospital — Athens, Greece
• Novartis Investigative Site — Pécs, Hungary
• Lipid Research — Jerusalem, Israel
• Tucson Medical Center — Tucson, Arizona, United States

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.