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Could a Twice-Yearly shot help kids with rare high cholesterol?

NCT ID NCT06597006

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jul 24, 2026 · Updated 1 time

Summary

This study tests a drug called inclisiran in children aged 2 to 12 who have a rare genetic condition that causes extremely high cholesterol. The goal is to see if the drug safely lowers their bad cholesterol compared to a placebo. Participants will receive either inclisiran or placebo for one year, then all will receive inclisiran for a second year.

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 9 people

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

Started

Feb 2025

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

2 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, 2 to \<12 years of age at screening * HoFH diagnosed by genetic confirmation \- Note: Participants with known null (negative) mutations in both LDLR alleles are not eligible (see also exclusion criteria) * Fasting LDL-C \>130 mg/dL (3.4 mmol/L) at screening * On an optimal dose of statin (investigator's discretion), unless statin intolerant, with or without other lipid-lowering therapy (e.g. ezetimibe) * Participants on lipid-lowering therapies (such as e.g. statins, ezetimibe) must be on a stable dose for ≥30 days before screening with no planned medication or dose changes during study participation * Participants on a documented regimen of LDL-apheresis for ≥ 3 months before screening will be allowed to continue the apheresis during the study, if needed. The apheresis schedule/settings/duration must be stable prior to screening, are not allowed to change during the double-blind period of the trial and must permit that an apheresis coincides with each study visit. Exclusion Criteria: * Documented evidence of a null (negative) mutation in both LDLR alleles * Previous treatment (within 90 days of screening) with monoclonal antibodies directed towards PCSK9 * History of poor response to therapy with any monoclonal antibody directed towards PCSK9 (e.g. \<15% reduction in LDL-C) * Treatment with mipomersen or lomitapide (within 5 months of screening) * Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome * Heterozygous familial hypercholesterolemia (HeFH) * Body weight (at the screening and/or randomization (Day 1) visit) \<16 kg for participants 6 to \<12 years (at screening) or \<11 kg for participants 2 to \<6 years (at screening) * 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 heart failure homozygous familial hypercholesterolemia Hypercholesterolemia hyperlipidemia Hyperlipoproteinemia Type II

As listed by the trial registrant

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

Familial hypercholesterolemia - homozygous

How to take part

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

  1. The places running it

    19 sites in 11 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

  • Childrens National Hospital

    RECRUITING

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

  • Novartis Investigative Site

    RECRUITING

    Vienna, 1090, Austria

  • Novartis Investigative Site

    RECRUITING

    Beijing, Beijing Municipality, 100013, China

  • Novartis Investigative Site

    RECRUITING

    Frankfurt am Main, Hesse, 60590, Germany

  • Novartis Investigative Site

    RECRUITING

    Ioannina, 455 00, Greece

  • Novartis Investigative Site

    RECRUITING

    Thessaloniki, 546 42, Greece

  • Novartis Investigative Site

    RECRUITING

    Kota Bharu, Kelantan, 16150, Malaysia

  • Novartis Investigative Site

    RECRUITING

    Amsterdam, North Holland, 1105 AZ, Netherlands

  • Novartis Investigative Site

    RECRUITING

    Bloemfontein, Free State, 9301, South Africa

  • Novartis Investigative Site

    RECRUITING

    Johannesburg, Gauteng, 2193, South Africa

  • Novartis Investigative Site

    RECRUITING

    Taichung, 407219, Taiwan

  • Novartis Investigative Site

    RECRUITING

    Taipei, 111045, 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

    Southampton, SO16 6YD, United Kingdom

  • 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

  • Washington Univ School Of Medicine

    RECRUITING

    St Louis, Missouri, 63110, United States

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