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

NCT ID NCT07037771

Recruiting now Disease control Sponsor: Arrowhead Pharmaceuticals Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a new medicine called zodasiran in people aged 12 and older who have a rare genetic condition that causes extremely high cholesterol (homozygous familial hypercholesterolemia). The goal is to see if the drug can safely lower bad cholesterol levels over 12 months. About 60 participants will receive either zodasiran or a placebo, and those who complete the main study can choose to continue in an open-label extension where everyone gets the active drug.

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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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Conditions

The condition(s) this trial relates to.

homozygous familial hypercholesterolemia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA

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

    RECRUITING

    Christchurch, 8011, New Zealand

  • Research Site 13

    RECRUITING

    Camperdown, New South Wales, 2050, Australia

  • Research Site 14

    RECRUITING

    Pittsburgh, Pennsylvania, 15213, United States

  • Research Site 15

    RECRUITING

    Tel Litwinsky, 5265601, Israel

  • Research Site 16

    RECRUITING

    Okayama, 700-8558, Japan

  • Research Site 17

    RECRUITING

    Tbilisi, 0159, Georgia

  • Research Site 18

    RECRUITING

    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

    RECRUITING

    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 36

    RECRUITING

    Afyonkarahisar, 03200, Turkey (Türkiye)

  • Research Site 37

    RECRUITING

    Vienna, 1130, Austria

  • Research Site 38

    RECRUITING

    Melikgazi/Kayseri, 38030, Turkey (Türkiye)

  • Research Site 39

    RECRUITING

    Linz, 4021, Austria

  • Research Site 4

    RECRUITING

    Québec, G1V 4W2, Canada

  • Research Site 40

    RECRUITING

    Innsbruck, 6020, Austria

  • Research Site 41

    RECRUITING

    Bornova/Izmir, 35100, Turkey (Türkiye)

  • Research Site 42

    RECRUITING

    Vienna, 1090, Austria

  • Research Site 43

    RECRUITING

    Barcelona, 8907, Spain

  • 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