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New study tests Long-Term safety of Cholesterol-Lowering injection for kids

NCT ID NCT05682378

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Sep 18, 2026 · Updated 1 time

Summary

This study looks at the long-term safety of inclisiran, a cholesterol-lowering injection, in children with a genetic condition that causes very high cholesterol. About 195 kids who completed earlier inclisiran studies will receive the drug for an extended period. Researchers will track side effects and how well the drug keeps cholesterol levels down.

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 cholesterol-lowering injection)
What this could lead to
If successful, this could show that inclisiran is safe and effective for long-term use in children with inherited high cholesterol, helping control their condition.
What could go wrong
This is an extension study with no placebo group, so results may be less reliable. It only includes kids who already benefited, which may not represent all patients. Long-term side effects 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 195 people

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

Started

Feb 2023

Expected to finish

Mar 2032

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 100 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.

Key inclusion: * Male and female participants with a diagnosis of HeFH or HoFH who completed the ORION-16, ORION-13, ORION-20 or ORION-19 studies * Per investigator's clinical judgment, participant derived benefit from treatment with inclisiran in the ORION-16, ORION-13, ORION-20 or ORION-19 studies Key exclusion: * Participants who in the feeder ORION-16, ORION-13, ORION-20, or ORION-19 studies either screen failed or permanently discontinued from the treatment/study for any reason or had serious safety or tolerability issues related to inclisiran treatment * Any uncontrolled or serious disease, or any medical, physical, or surgical condition, that may either interfere with participation in the clinical study or interpretation of clinical study results, and/or put the participant at significant risk

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Conditions

The condition(s) this trial relates to.

familial hypercholesterolemia homozygous familial hypercholesterolemia Hyperlipoproteinemia Type II

As listed by the trial registrant

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

Heterozygous or homozygous familial hypercholesterolemia

How to take part

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

  1. The places running it

    2 sites in 2 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 Hosp Pittsburgh UPMC

    COMPLETED

    Pittsburgh, Pennsylvania, 15224, United States

  • Cincinnati Childrens Hospital MC

    COMPLETED

    Cincinnati, Ohio, 45229-3039, United States

  • Excel Medical Clinical Trials LLC

    COMPLETED

    Boca Raton, Florida, 33434, United States

  • Icahn School of Med at Mt Sinai

    ACTIVE_NOT_RECRUITING

    New York, New York, 10029, United States

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Formosa, Formosa Province, P3600MPD, Argentina

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Fortaleza, Ceará, 60430-275, Brazil

  • Novartis Investigative Site

    COMPLETED

    São Paulo, São Paulo, 04023-900, Brazil

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    São Paulo, São Paulo, 05403-000, Brazil

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Québec, Quebec, G1V 4W2, Canada

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Prague, 128 08, Czechia

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Prague, 150 06, Czechia

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Besançon, 25030, France

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Toulouse, 31059, France

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Frankfurt am Main, Hesse, 60590, Germany

  • Novartis Investigative Site

    COMPLETED

    Freiburg im Breisgau, 79100, Germany

  • Novartis Investigative Site

    COMPLETED

    Athens, 115 27, Greece

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Athens, 185 47, Greece

  • Novartis Investigative Site

    COMPLETED

    Pécs, Baranya, 7623, Hungary

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Jerusalem, 9112001, Israel

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Ramat Gan, 5265601, Israel

  • Novartis Investigative Site

    COMPLETED

    Milan, MI, 20162, Italy

  • Novartis Investigative Site

    COMPLETED

    Modena, MO, 41124, Italy

  • Novartis Investigative Site

    COMPLETED

    Roma, RM, 00161, Italy

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Roma, RM, 00165, Italy

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Irbid, 22110, Jordan

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Beirut, 1100 2070, Lebanon

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Beirut, 113-0236, Lebanon

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Sungai Buloh, Selangor, 47000, Malaysia

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Kuala Lumpur, 59100, Malaysia

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Amsterdam, North Holland, 1105 AZ, Netherlands

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Rotterdam, South Holland, 3015 GD, Netherlands

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Oslo, 0514, Norway

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Gdansk, 80-952, Poland

  • Novartis Investigative Site

    COMPLETED

    Lodz, Łódź Voivodeship, 93-338, Poland

  • Novartis Investigative Site

    COMPLETED

    Kemerovo, 650002, Russia

  • Novartis Investigative Site

    COMPLETED

    Moscow, 127412, Russia

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Poprad, 058 01, Slovakia

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Ljubljana, 1000, Slovenia

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Bloemfontein, Free State, 9301, South Africa

  • Novartis Investigative Site

    COMPLETED

    Cape Town, Western Cape, 7130, South Africa

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Pamplona, Navarre, 31008, Spain

  • Novartis Investigative Site

    COMPLETED

    Oviedo, Principality of Asturias, 33011, Spain

  • Novartis Investigative Site

    COMPLETED

    A Coruña, 15001, Spain

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Córdoba, 14004, Spain

  • Novartis Investigative Site

    COMPLETED

    Geneva, 1211, Switzerland

  • Novartis Investigative Site

    RECRUITING

    Taipei, 111045, Taiwan

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Istanbul, Fatih, 34098, Turkey (Türkiye)

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Adana, Saricam, 01330, Turkey (Türkiye)

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Ankara, Yenimahalle, 06500, Turkey (Türkiye)

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Izmir, 35100, Turkey (Türkiye)

  • Novartis Investigative Site

    ACTIVE_NOT_RECRUITING

    Middlesex, UB9 6JH, United Kingdom

  • Primary Childrens Medical Center

    RECRUITING

    Salt Lake City, Utah, 84113, United States