New hope for kids with rare high cholesterol: drug trial launches

NCT ID NCT06597006

First seen Nov 01, 2025 · Last updated May 08, 2026 · Updated 27 times

Summary

This study tests a drug called inclisiran in children aged 2 to under 12 who have a severe inherited form of high cholesterol (homozygous familial hypercholesterolemia). The goal is to see if the drug safely lowers their LDL (bad) cholesterol compared to a placebo over one year, followed by an open-label phase. Only 9 children will take part, and they must already be on standard cholesterol-lowering treatments.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

  • Contact

    Phone: •••-•••-••••

Locations

  • Childrens National Hospital

    RECRUITING

    Washington D.C., District of Columbia, 20010, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • Novartis Investigative Site

    RECRUITING

    Vienna, 1090, Austria

  • Novartis Investigative Site

    RECRUITING

    Beijing, Beijing Municipality, 100013, China

  • Novartis Investigative Site

    RECRUITING

    Frankfurt am Main, Hesse, 60590, Germany

  • Novartis Investigative Site

    RECRUITING

    Ioannina, 455 00, Greece

  • Novartis Investigative Site

    RECRUITING

    Thessaloniki, 546 42, Greece

  • Novartis Investigative Site

    RECRUITING

    Kota Bharu, Kelantan, 16150, Malaysia

  • Novartis Investigative Site

    RECRUITING

    Amsterdam, North Holland, 1105 AZ, Netherlands

  • Novartis Investigative Site

    RECRUITING

    Bloemfontein, Free State, 9301, South Africa

  • Novartis Investigative Site

    RECRUITING

    Taichung, 407219, Taiwan

  • Novartis Investigative Site

    RECRUITING

    Taipei, 111045, Taiwan

  • Novartis Investigative Site

    RECRUITING

    Adana, Saricam, 01330, Turkey (Türkiye)

  • Novartis Investigative Site

    RECRUITING

    Ankara, Yenimahalle, 06500, Turkey (Türkiye)

  • Novartis Investigative Site

    RECRUITING

    Izmir, 35100, Turkey (Türkiye)

  • Novartis Investigative Site

    RECRUITING

    Southampton, SO16 6YD, United Kingdom

  • UC San Francisco Medical Center

    RECRUITING

    San Francisco, California, 94143, United States

  • UC San Francisco Medical Center

    RECRUITING

    San Francisco, California, 94143, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

    Contact

  • Washington Univ School Of Medicine

    RECRUITING

    St Louis, Missouri, 63110, United States

    Contact

    Contact Email: •••••@•••••

Conditions

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