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

NCT ID NCT07037771

Ongoing Disease control Sponsor: Arrowhead Pharmaceuticals Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Aug 13, 2026 · Updated 2 times

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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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

Locations

Research Site 1

Cincinnati, Ohio, 45227, United States
Research Site 10

Jerusalem, 9112001, Israel
Research Site 11

Parktown, Johannesburg, 2193, South Africa
Research Site 12

Christchurch, 8011, New Zealand
Research Site 13

Camperdown, New South Wales, 2050, Australia
Research Site 14

Pittsburgh, Pennsylvania, 15213, United States
Research Site 15

Tel Litwinsky, 5265601, Israel
Research Site 16

Okayama, 700-8558, Japan
Research Site 17

Tbilisi, 0159, Georgia
Research Site 18

Tbilisi, 160, Georgia
Research Site 19

Tbilisi, 186, Georgia
Research Site 2

New York, New York, 10029, United States
Research Site 20

Fukushima, 960-1295, Japan
Research Site 21

Heidelberg, Victoria, 3084, Australia
Research Site 22

Saitama, 350-0495, Japan
Research Site 23

Paris, 75013, France
Research Site 24

Cerqueira César, São Paulo, CEP-05403-000, Brazil
Research Site 25

Tokyo, 113-8510, Japan
Research Site 26

Kanazawa, Ishikawa-ken, 920-8641, Japan
Research Site 27

Suita, Osaka, 564-8565, Japan
Research Site 28

Córdoba, 14004, Spain
Research Site 29

Madrid, 28041, Spain
Research Site 3

Nedlands, Western Australia, 6009, Australia
Research Site 30

Hradec Králové, General E3 500 0, Czechia
Research Site 31

Riyadh, 12713, Saudi Arabia
Research Site 32

Gothenburg, 413 46, Sweden
Research Site 33

La Louvière, 7100, Belgium
Research Site 34

Leuven, 3000, Belgium
Research Site 35

Berlin, 13353, Germany
Research Site 36

Afyonkarahisar, 03200, Turkey (Türkiye)
Research Site 37

Vienna, 1130, Austria
Research Site 38

Melikgazi/Kayseri, 38030, Turkey (Türkiye)
Research Site 39

Linz, 4021, Austria
Research Site 4

Québec, G1V 4W2, Canada
Research Site 40

Innsbruck, 6020, Austria
Research Site 41

Bornova/Izmir, 35100, Turkey (Türkiye)
Research Site 42

Vienna, 1090, Austria
Research Site 43

Barcelona, 8907, Spain
Research Site 5

Vancouver, British Columbia, V6Z 2H2, Canada
Research Site 6

Chicoutimi, Quebec, G7H 7K9, Canada
Research Site 7

Park Ridge, Illinois, 60068, United States
Research Site 8

Cape Town, 7925, South Africa
Research Site 9

Saint Leonards, New South Wales, 2065, Australia

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