New shot aims to slash sky-high cholesterol in rare genetic disease

NCT ID NCT07037771

First seen Oct 31, 2025 · Last updated May 13, 2026 · Updated 25 times

Summary

This study tests a new medicine called zodasiran in people aged 12 and older with homozygous familial hypercholesterolemia (HoFH), a rare genetic condition causing extremely high cholesterol. The goal is to see if the drug safely lowers bad cholesterol levels over 12 months. Participants receive either zodasiran or a placebo, and after the main study, everyone can choose to receive the active drug in an open-label extension.

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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: •••••@•••••

Locations

  • Research Site 1

    RECRUITING

    Cincinnati, Ohio, 45227, United States

  • Research Site 10

    RECRUITING

    Jerusalem, 9112001, Israel

  • Research Site 11

    RECRUITING

    Parktown, Johannesburg, 2193, South Africa

  • Research Site 12

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    Christchurch, 8011, New Zealand

  • Research Site 13

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    Camperdown, New South Wales, 2050, Australia

  • Research Site 14

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    Pittsburgh, Pennsylvania, 15213, United States

  • Research Site 15

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    Tel Litwinsky, 5265601, Israel

  • Research Site 16

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    Okayama, 700-8558, Japan

  • Research Site 17

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    Tbilisi, 0159, Georgia

  • Research Site 18

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    Tbilisi, 160, Georgia

  • Research Site 19

    RECRUITING

    Tbilisi, 186, Georgia

  • Research Site 2

    RECRUITING

    New York, New York, 10029, United States

  • Research Site 20

    RECRUITING

    Fukushima, 960-1295, Japan

  • Research Site 21

    RECRUITING

    Heidelberg, Victoria, 3084, Australia

  • Research Site 22

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    Saitama, 350-0495, Japan

  • Research Site 23

    RECRUITING

    Paris, 75013, France

  • Research Site 24

    RECRUITING

    Cerqueira César, São Paulo, CEP-05403-000, Brazil

  • Research Site 25

    RECRUITING

    Tokyo, 113-8510, Japan

  • Research Site 26

    RECRUITING

    Kanazawa, Ishikawa-ken, 920-8641, Japan

  • Research Site 27

    RECRUITING

    Suita, Osaka, 564-8565, Japan

  • Research Site 28

    RECRUITING

    Córdoba, 14004, Spain

  • Research Site 29

    RECRUITING

    Madrid, 28041, Spain

  • Research Site 3

    RECRUITING

    Nedlands, Western Australia, 6009, Australia

  • Research Site 30

    RECRUITING

    Hradec Králové, General E3 500 0, Czechia

  • Research Site 31

    RECRUITING

    Riyadh, 12713, Saudi Arabia

  • Research Site 32

    RECRUITING

    Gothenburg, 413 46, Sweden

  • Research Site 33

    RECRUITING

    La Louvière, 7100, Belgium

  • Research Site 34

    RECRUITING

    Leuven, 3000, Belgium

  • Research Site 35

    RECRUITING

    Berlin, 13353, Germany

  • Research Site 4

    RECRUITING

    Québec, G1V 4W2, Canada

  • Research Site 5

    RECRUITING

    Vancouver, British Columbia, V6Z 2H2, Canada

  • Research Site 6

    RECRUITING

    Chicoutimi, Quebec, G7H 7K9, Canada

  • Research Site 7

    RECRUITING

    Park Ridge, Illinois, 60068, United States

  • Research Site 8

    RECRUITING

    Cape Town, 7925, South Africa

  • Research Site 9

    RECRUITING

    Saint Leonards, New South Wales, 2065, Australia

Conditions

Explore the condition pages connected to this study.