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New shot could help teens with inherited high cholesterol

NCT ID NCT04652726

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 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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 tested a drug called inclisiran in teenagers aged 12 to 18 who have a genetic condition that causes very high cholesterol. The goal was to see if the drug is safe and can lower bad cholesterol. Participants received either inclisiran or a placebo for one year, then everyone received 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

141 people

The number who actually took part.

Started

Jan 2021

Finished

Nov 2024

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 to 17 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: * Heterozygous Familial Hypercholesterolemia (HeFH) diagnosed either by genetic testing or on phenotypic criteria * Fasting LDL-C \>130 mg/dL (3.4 mmol/L) at screening * Fasting triglycerides \<400 mg/dL (4.5 mmol/L) at screening * On maximally tolerated dose of statin (investigator's discretion) with or without other lipid-lowering therapy; stable for ≥ 30 days before screening Exclusion Criteria: * Homozygous familial hypercholesterolemia (HoFH) * Active liver disease * Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome * Previous treatment with monoclonal antibodies directed towards PCSK9 (within 90 days of screening) * Recent and/or planned use of other investigational medicinal products or devices

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Conditions

The condition(s) this trial relates to.

familial hypercholesterolemia

As listed by the trial registrant

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

Familial hypercholesterolemia - heterozygous

Contacts and locations

Locations

  • Childrens Hospital Pittsburgh of UPMC

    Pittsburgh, Pennsylvania, 15224, United States

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229-3039, United States

  • Far Eastern Memorial Hospital

    New Taipei City, 22060, Taiwan

  • Gazi University Medical Faculty

    Ankara, 06500, Turkey (Türkiye)

  • Heart Institute (InCOr) HCMFUSP

    São Paulo, São Paulo, 05403 000, Brazil

  • Hippokrateion General Hospital of Athens Greece

    Athens, 115 27, Greece

  • Hospital Abente y Lago

    A Coruña, 15001, Spain

  • Hospital Central de Asturias

    Oviedo, Principality of Asturias, 33011, Spain

  • Hospital Reina Sofia

    Córdoba, Andalusia, 14004, Spain

  • Hospital Virgen de la Vcitoria

    Málaga, Andalusia, 29010, Spain

  • Hotel Dieu de France Hospital

    El Achrafiyé, 166830, Lebanon

  • Icahn School of Medicine at Mount Sinai

    New York, New York, 10029, United States

  • Institute of Internal Prev. Med.

    Novosibirsk, 630090, Russia

  • Institute of the complex problems of cardiovascular disease

    Kemerovo, 650002, Russia

  • KKIM UK Frankfurt/Main

    Frankfurt, 60590, Germany

  • Lipid Research

    Jerusalem, 9112001, Israel

  • Lipids Center Sheba Medical Center, Israel

    Ramat Gan, 52621, Israel

  • Metropolitan Hospital

    Athens, 18547, Greece

  • Novartis Investigative Site

    Formosa, Formosa Province, P3600, Argentina

  • Novartis Investigative Site

    Québec, Quebec, G1V 4W2, Canada

  • Novartis Investigative Site

    Prague, 12808, Czechia

  • Novartis Investigative Site

    Prague, 150 06, Czechia

  • Novartis Investigative Site

    Besançon, 25030, France

  • Novartis Investigative Site

    Bron, 69677, France

  • Novartis Investigative Site

    Toulouse, 31059, France

  • Novartis Investigative Site

    Pécs, 7623, Hungary

  • Novartis Investigative Site

    Milan, MI, 20162, Italy

  • Novartis Investigative Site

    Modena, MO, 41124, Italy

  • Novartis Investigative Site

    Roma, RM, 00161, Italy

  • Novartis Investigative Site

    Roma, RM, 00165, Italy

  • Novartis Investigative Site

    Irbid, 22110, Jordan

  • Novartis Investigative Site

    Rotterdam, South Holland, 3015 GD, Netherlands

  • Novartis Investigative Site

    Amsterdam, 1105 AZ, Netherlands

  • Novartis Investigative Site

    Oslo, 0514, Norway

  • Novartis Investigative Site

    Gdansk, 80 952, Poland

  • Novartis Investigative Site

    Lodz, 93-338, Poland

  • Novartis Investigative Site

    Moscow, 127412, Russia

  • Novartis Investigative Site

    Poprad, 058 01, Slovakia

  • Novartis Investigative Site

    Bloemfontein, Free State, 9301, South Africa

  • Novartis Investigative Site

    Somerset West, Western Cape, 7130, South Africa

  • Novartis Investigative Site

    Cape Town, 7925, South Africa

  • Novartis Investigative Site

    Pamplona, Navarre, 31008, Spain

  • Novartis Investigative Site

    Geneva, 1211, Switzerland

  • Novartis Investigative Site

    Istanbul, TUR, 34098, Turkey (Türkiye)

  • Novartis Investigative Site

    Adana, 01330, Turkey (Türkiye)

  • Novartis Investigative Site

    Izmir, 35100, Turkey (Türkiye)

  • Novartis Investigative Site

    Middlesex, UB9 6JH, United Kingdom

  • Nucleo de Pesquisa Clinica do Rio Grande do Sul

    Porto Alegre, Rio Grande do Sul, 90430-001, Brazil

  • Primary Children's Medical Center

    Salt Lake City, Utah, 84113, United States

  • Setor de Lípides, Aterosclerose e Biologia

    São Paulo, São Paulo, 04023-900, Brazil

  • Taipei Veterans General Hospital

    Taipei, 11217, Taiwan

  • Tucson Medical Center

    Tucson, Arizona, 85712, United States

  • UiTM Sungai Buloh

    Sungai Buloh, Selangor, 47000, Malaysia

  • Unidade de pesquisa clinica - Hospital Universitario Walter Cantidio

    Fortaleza, Ceará, 60430275, Brazil

  • Universitaetsklinikum Freiburg

    Freiburg im Breisgau, 79106, Germany

  • Universitaetsmedizin Mannheim

    Mannheim, Baden-Wurttemberg, 68305, Germany

  • University General Hospital of Ioannina

    Ioannina, GR, 455 00, Greece

  • University Medical Centre Ljubljana, Div. of Pediatric Dept. of Endocrinology, Diabetes and Metabolic Diseases

    Ljubljana, 1000, Slovenia

  • Wake Forest U of Health Sciences

    Winston-Salem, North Carolina, 27157, United States

More trials for these conditions

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