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

Completed · Phase 3 · Has a placebo group

Conditions studied: Familial Hypercholesterolemia - Homozygous

In brief

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

Key facts

Study ID

NCT04659863

Run by

Novartis Pharmaceuticals

People needed

13

Starts

2021-02-16

Expected to finish

2024-11-18

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…

• Homozygous Familial Hypercholesterolemia (HoFH) diagnosed by genetic confirmation
• Fasting LDL-C >130 mg/dL (3.4 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
• Male or female participants >=12 to <18 years of age at screening

You may not qualify if…

• Documented evidence of a null (negative) mutation in both LDLR alleles
• Heterozygous familial hypercholesterolemia (HeFH)
• Active liver disease
• Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome
• Previous treatment with monoclonal antibodies directed towards PCSK9 (within 90 days of screening)
• Treatment with mipomersen or lomitapide (within 5 months of screening)
• Recent and/or planned use of other investigational medicinal products or devices

Where it is running

• Excel Medical Clinical Trials LLC — Boca Raton, Florida, United States
• Novartis Investigative Site — Québec, Quebec, Canada
• Novartis Investigative Site — Bron, France
• University General Hospital of Ioannina — Ioannina, GR, Greece
• Metropolitan Hospital — Athens, Greece
• American University of Beirut Medical Center — Beirut, Lebanon
• Hotel Dieu de France Hospital — El Achrafiyé, Lebanon
• Novartis Investigative Site — Kuala Lumpur, Malaysia
• Novartis Investigative Site — Amsterdam, Netherlands
• Novartis Investigative Site — Istanbul, TUR, Turkey (Türkiye)
• Novartis Investigative Site — Izmir, Turkey (Türkiye)

Full record on ClinicalTrials.gov

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