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New shot could slash Sky-High cholesterol in teens with rare gene condition

NCT ID NCT04659863

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 rare inherited condition that causes extremely high cholesterol. The goal was to see if the drug safely lowers their LDL (bad) cholesterol. Participants received either inclisiran or a placebo for one year, followed by open-label inclisiran for a second year.

What this could mean

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

Active substance
Inclisiran
What this could lead to
If successful, inclisiran could offer a new, convenient treatment option to lower dangerously high cholesterol in teenagers with this rare genetic condition.
What could go wrong
This is a small, early-phase study with only 13 participants, so results may not apply to all patients. Long-term safety and effectiveness 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

13 people

The number who actually took part.

Started

Feb 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: * Homozygous Familial Hypercholesterolemia (HoFH) diagnosed by genetic confirmation * Fasting LDL-C \>130 mg/dL (3.4 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 * Male or female participants \>=12 to \<18 years of age at screening Exclusion Criteria: * Documented evidence of a null (negative) mutation in both LDLR alleles * Heterozygous familial hypercholesterolemia (HeFH) * Active liver disease * Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome * Previous treatment with monoclonal antibodies directed towards PCSK9 (within 90 days of screening) * Treatment with mipomersen or lomitapide (within 5 months 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.

homozygous familial hypercholesterolemia

As listed by the trial registrant

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

Familial hypercholesterolemia - homozygous

Contacts and locations

Locations

  • American University of Beirut Medical Center

    Beirut, 1107 2020, Lebanon

  • Excel Medical Clinical Trials LLC

    Boca Raton, Florida, 33434, United States

  • Hotel Dieu de France Hospital

    El Achrafiyé, 166830, Lebanon

  • Metropolitan Hospital

    Athens, 18547, Greece

  • Novartis Investigative Site

    Québec, Quebec, G1V 4W2, Canada

  • Novartis Investigative Site

    Bron, 69677, France

  • Novartis Investigative Site

    Kuala Lumpur, 59100, Malaysia

  • Novartis Investigative Site

    Amsterdam, 1105 AZ, Netherlands

  • Novartis Investigative Site

    Istanbul, TUR, 34098, Turkey (Türkiye)

  • Novartis Investigative Site

    Izmir, 35100, Turkey (Türkiye)

  • University General Hospital of Ioannina

    Ioannina, GR, 455 00, Greece

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