Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

Cure My Disease

Don't miss your cure!

 Log in / Sign up

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

NCT ID NCT07037771

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

70 people

The number who actually took part.

Started

Jun 2025

Expected to finish

Aug 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

12 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Age ≥12 years, non pregnant, non lactating, do not plan to become pregnant during the study * Body weight ≥35 kg at Screening as patients could theoretically be \<35 kg as the study continues. * HoFH based on a supportive genetic test or a clinical diagnosis (total cholesterol \>500 mg/dL\[13 mmol/L\] OR treated LDL-C concentration of ≥300 mg/dL \[≥8 mmol/L\] either accompanied by TGs \<300 mg/dL \[3.4 mmol/L\] AND both parents with documented total cholesterol \>250 mg/dL \[6.5 mmol/L\] OR cutaneous or tendinous xanthoma before 10 years of age) * LDL-C ≥70 mg/dL (1.8 mmol/L). For adolescents 12 to \<18 years of age, screening LDL-C ≥116 mg/dL (3 mmol/L). * Hemoglobin A1c (HbA1c) ≤9.5% * Total bilirubin \<2xULN, unless in previously confirmed cases of Gilbert's syndrome * Alanine aminotransferase or aspartate aminotransferase \<3×ULN * On standard of care, maximally tolerated lipid-lowering therapy to include a maximally tolerated statin, ezetimibe, and a PCSK9 inhibitor Exclusion Criteria: * Use of a hepatocyte-targeted siRNA within 365 days before Day 1 (except inclisiran, which is permitted; administration of inclisiran and study drug must be separated by at least 4 weeks) * Use of an antisense oligonucleotide molecule within 3 months before Day 1 (except inclisiran, which is permitted; administration of inclisiran and study drug must be separated by at least 4 weeks) * Use of evinacumab within 3 months before Day 1. Evinacumab use is prohibited during the study. * Non-response to evinacumab, defined as LDL-C reduction \<15% from baseline after 2 doses * Use of any other investigational agent or device within 30 days or 5 half-lives (whichever is longer) before Day 1 * Use of systemic corticosteroids (unless used as replacement therapy for pituitary/adrenal disease with a stable regimen) * Estimated glomerular filtration rate \<30 mL/min NOTE: Additional Inclusion/exclusion criteria may apply per protocol

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Homozygous familial hypercholesterolemia are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

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

More trials for these conditions

Other studies related to the condition(s) this trial covers.