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New shot could slash 'Bad' cholesterol in kids with genetic heart risk

NCT ID NCT06597019

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jun 27, 2026

Summary

This study tests a drug called inclisiran in children aged 6 to 12 who have a genetic condition that causes very high cholesterol. The drug is given as a shot under the skin and aims to lower 'bad' LDL cholesterol. The trial compares inclisiran to a placebo for one year, then everyone gets inclisiran for a second year. About 60 children will take part to see if the drug is safe and works well.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Inclisiran (a drug that lowers cholesterol by blocking a protein in the liver)
What this could lead to
If it works, this could provide a new, convenient treatment option (given as a shot every few months) to help children with inherited high cholesterol lower their LDL levels.
What could go wrong
This is a relatively small, early-stage trial in children, so results may not apply to all. The drug's long-term safety and effectiveness in this age group are still unknown.

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

About 60 people

The number the study aims to enrol. It can still change while the study runs.

Started

Dec 2024

Expected to finish

Apr 2029

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

6 to 11 years

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: * Male or female participants, 6 to \<12 years of age at screening * HeFH diagnosed either by genetic testing or on phenotypic criteria * Fasting LDL-C \>130 mg/dL (3.4 mmol/L) at screening * For participants 8 to \<12 years, on an optimal dose of statin (investigator's discretion) unless statin intolerant, with or without other lipid-lowering therapy (e.g. ezetimibe). For participants \<8 years, the use of background lipid-lowering treatment is based on investigator's discretion. * Participants on lipid-lowering therapies (such as statin and/or e.g. ezetimibe) must be on a stable dose for ≥30 days before screening with no planned medication or dose changes during study participation. Exclusion Criteria: * Previous treatment (within 90 days of screening) with monoclonal antibodies directed towards PCSK9 * Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome * Homozygous familial hypercholesterolemia (HoFH) * Body weight \<16 kg at the screening and/or randomization (Day 1) visit * Active liver disease defined as any known current infectious, neoplastic, or metabolic pathology of the liver or unexplained alanine aminotransferase (ALT), aspartate aminotransferase (AST) elevation \>3x ULN, or total bilirubin elevation \>2x ULN (except patients with Gilbert's syndrome) * Pregnant or nursing females * Recent and/or planned use of other investigational medicinal products or devices

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Conditions

The condition(s) this trial relates to.

aortic valve stenosis cardiovascular disorder Dyslipidemias familial hypercholesterolemia heart failure Hypercholesterolemia hyperlipidemia Hyperlipoproteinemia Type II

As listed by the trial registrant

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

Familial hypercholesterolemia - heterozygous

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    66 sites in 23 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Children's National Hospital

    NOT_YET_RECRUITING

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

  • Childrens National Hospital

    RECRUITING

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

  • Excel Medical Clinical Trials LLC

    RECRUITING

    Boca Raton, Florida, 33434, United States

  • Icahn School of Med at Mt Sinai

    RECRUITING

    New York, New York, 10029, United States

  • Novartis Investigative Site

    RECRUITING

    Buenos Aires, C1245AAM, Argentina

  • Novartis Investigative Site

    RECRUITING

    CABA, C1181ACH, Argentina

  • Novartis Investigative Site

    RECRUITING

    Salzburg, 5020, Austria

  • Novartis Investigative Site

    RECRUITING

    Vienna, 1090, Austria

  • Novartis Investigative Site

    RECRUITING

    Brussels, 1200, Belgium

  • Novartis Investigative Site

    RECRUITING

    Leuven, 3000, Belgium

  • Novartis Investigative Site

    RECRUITING

    Fortaleza, Ceará, 60430-275, Brazil

  • Novartis Investigative Site

    RECRUITING

    Rio de Janeiro, Rio de Janeiro, 20211-340, Brazil

  • Novartis Investigative Site

    RECRUITING

    Porto Alegre, Rio Grande do Sul, 90020-020, Brazil

  • Novartis Investigative Site

    RECRUITING

    São Paulo, São Paulo, 05403 000, Brazil

  • Novartis Investigative Site

    RECRUITING

    Beijing, Beijing Municipality, 100013, China

  • Novartis Investigative Site

    RECRUITING

    Shanghai, 200127, China

  • Novartis Investigative Site

    RECRUITING

    Prague, 128 08, Czechia

  • Novartis Investigative Site

    RECRUITING

    Prague, 150 06, Czechia

  • Novartis Investigative Site

    RECRUITING

    Marseille, 13885, France

  • Novartis Investigative Site

    RECRUITING

    Nantes, 44093, France

  • Novartis Investigative Site

    RECRUITING

    Paris, 75012, France

  • Novartis Investigative Site

    RECRUITING

    Freiburg im Breisgau, Baden-Wurttemberg, 79106, Germany

  • Novartis Investigative Site

    RECRUITING

    Frankfurt am Main, Hesse, 60590, Germany

  • Novartis Investigative Site

    RECRUITING

    Hanover, 30173, Germany

  • Novartis Investigative Site

    RECRUITING

    Athens, 115 27, Greece

  • Novartis Investigative Site

    RECRUITING

    Ioannina, 455 00, Greece

  • Novartis Investigative Site

    RECRUITING

    Hong Kong, 999077, Hong Kong

  • Novartis Investigative Site

    RECRUITING

    Budapest, 1026, Hungary

  • Novartis Investigative Site

    RECRUITING

    Jerusalem, 9112001, Israel

  • Novartis Investigative Site

    RECRUITING

    Ramat Gan, 5265601, Israel

  • Novartis Investigative Site

    RECRUITING

    Milan, MI, 20162, Italy

  • Novartis Investigative Site

    RECRUITING

    Roma, RM, 00165, Italy

  • Novartis Investigative Site

    RECRUITING

    Torino, TO, 10126, Italy

  • Novartis Investigative Site

    RECRUITING

    Verona, VR, 37126, Italy

  • Novartis Investigative Site

    RECRUITING

    Kuala Lumpur, Kuala Lumpur, 50586, Malaysia

  • Novartis Investigative Site

    RECRUITING

    Amsterdam, North Holland, 1105 AZ, Netherlands

  • Novartis Investigative Site

    RECRUITING

    Bialystok, 15-274, Poland

  • Novartis Investigative Site

    RECRUITING

    Gdansk, 80 952, Poland

  • Novartis Investigative Site

    RECRUITING

    Lodz, Łódź Voivodeship, 93-338, Poland

  • Novartis Investigative Site

    RECRUITING

    Coimbra, 3000-602, Portugal

  • Novartis Investigative Site

    RECRUITING

    Lisbon, 1649-035, Portugal

  • Novartis Investigative Site

    RECRUITING

    Porto, 4050-651, Portugal

  • Novartis Investigative Site

    RECRUITING

    Porto, 4200 319, Portugal

  • Novartis Investigative Site

    RECRUITING

    Bloemfontein, Free State, 9301, South Africa

  • Novartis Investigative Site

    RECRUITING

    Elche, Alicante, 03203, Spain

  • Novartis Investigative Site

    RECRUITING

    Cadiz, Andalusia, 11009, Spain

  • Novartis Investigative Site

    RECRUITING

    Badalona, Barcelona, 08916, Spain

  • Novartis Investigative Site

    RECRUITING

    Esplugues, Barcelona, 08950, Spain

  • Novartis Investigative Site

    RECRUITING

    Pamplona, Navarre, 31008, Spain

  • Novartis Investigative Site

    RECRUITING

    Barcelona, 08041, Spain

  • Novartis Investigative Site

    RECRUITING

    Málaga, 29011, Spain

  • Novartis Investigative Site

    RECRUITING

    Seville, 41013, Spain

  • Novartis Investigative Site

    RECRUITING

    Taipei, 111045, Taiwan

  • Novartis Investigative Site

    RECRUITING

    Taipei, 11217, 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

    West Midlands, Birmingham, B4 6NH, United Kingdom

  • Novartis Investigative Site

    RECRUITING

    London, NW3 2QG, United Kingdom

  • Primary Childrens Medical Center

    NOT_YET_RECRUITING

    Salt Lake City, Utah, 84113, United States

  • Primary Childrens Medical Center

    RECRUITING

    Salt Lake City, Utah, 84113, United States

  • UC San Francisco Medical Center

    RECRUITING

    San Francisco, California, 94143-0348, United States

  • UC San Francisco Medical Center

    RECRUITING

    San Francisco, California, 94143, United States

  • Virginia Commonwealth University

    RECRUITING

    Richmond, Virginia, 23298, United States

  • West Virginia Childrens Hospital

    RECRUITING

    Morgantown, West Virginia, 26506, United States

  • West Virginia Childrens Hospital

    RECRUITING

    Morgantown, West Virginia, 26506, United States

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