Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children With Homozygous Familial Hypercholesterolemia

Recruiting now · Phase 3 · Has a placebo group

Conditions studied: Familial Hypercholesterolemia - Homozygous

In brief

This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in children (aged 2 to \<12 years) with homozygous familial hypercholesterolemia (HoFH) and elevated low density lipoprotein cholesterol (LDLC).

Key facts

Study ID

NCT06597006

Run by

Novartis Pharmaceuticals

People needed

9

Starts

2025-02-28

Expected to finish

2029-04-15

Last updated by the study team

2026-07-24

Who can join

Age: 2 and older, up to 11. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Male or female participants, 2 to <12 years of age at screening
• HoFH diagnosed by genetic confirmation
• Note: Participants with known null (negative) mutations in both LDLR alleles are not eligible (see also exclusion criteria)
• Fasting LDL-C >130 mg/dL (3.4 mmol/L) at screening
• On an optimal dose of statin (investigator's discretion), unless statin intolerant, with or without other lipid-lowering therapy (e.g. ezetimibe)
• Participants on lipid-lowering therapies (such as e.g. statins, ezetimibe) must be on a stable dose for ≥30 days before screening with no planned medication or dose changes during study participation
• Participants on a documented regimen of LDL-apheresis for ≥ 3 months before screening will be allowed to continue the apheresis during the study, if needed. The apheresis schedule/settings/duration must be stable prior to screening, are not allowed to change during the double-blind period of the trial and must permit that an apheresis coincides with each study visit.

You may not qualify if…

• Documented evidence of a null (negative) mutation in both LDLR alleles
• Previous treatment (within 90 days of screening) with monoclonal antibodies directed towards PCSK9
• History of poor response to therapy with any monoclonal antibody directed towards PCSK9 (e.g. <15% reduction in LDL-C)
• Treatment with mipomersen or lomitapide (within 5 months of screening)
• Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome
• Heterozygous familial hypercholesterolemia (HeFH)
• Body weight (at the screening and/or randomization (Day 1) visit) <16 kg for participants 6 to <12 years (at screening) or <11 kg for participants 2 to <6 years (at screening)
• Active liver disease defined as any known current infectious, neoplastic, or metabolic pathology of the liver or unexplained alanine aminotransferase (ALT), aspartate aminotransferase (AST) elevation >3x ULN, or total bilirubin elevation >2x ULN (except patients with Gilbert's syndrome)
• Pregnant or nursing females
• Recent and/or planned use of other investigational medicinal products or devices

Where it is running

• UC San Francisco Medical Center — San Francisco, California, United States (enrolling)
• Childrens National Hospital — Washington D.C., District of Columbia, United States (enrolling)
• Washington Univ School Of Medicine — St Louis, Missouri, United States (enrolling)
• Primary Childrens Medical Center — Salt Lake City, Utah, United States (enrolling)
• Novartis Investigative Site — Vienna, Austria (enrolling)
• Novartis Investigative Site — Beijing, Beijing Municipality, China (enrolling)
• Novartis Investigative Site — Frankfurt am Main, Hesse, Germany (enrolling)
• Novartis Investigative Site — Ioannina, Greece (enrolling)
• Novartis Investigative Site — Thessaloniki, Greece (enrolling)
• Novartis Investigative Site — Kota Bharu, Kelantan, Malaysia (enrolling)
• Novartis Investigative Site — Amsterdam, North Holland, Netherlands (enrolling)
• Novartis Investigative Site — Bloemfontein, Free State, South Africa (enrolling)
• Novartis Investigative Site — Johannesburg, Gauteng, South Africa (enrolling)
• Novartis Investigative Site — Taichung, Taiwan (enrolling)
• Novartis Investigative Site — Taipei, Taiwan (enrolling)
• Novartis Investigative Site — Adana, Saricam, Turkey (Türkiye) (enrolling)
• Novartis Investigative Site — Ankara, Yenimahalle, Turkey (Türkiye) (enrolling)
• Novartis Investigative Site — Izmir, Turkey (Türkiye) (enrolling)
• Novartis Investigative Site — Southampton, United Kingdom (enrolling)

Full record on ClinicalTrials.gov

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