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Inclisiran for Children with Heterozygous Familial Hypercholesterolemia

This study is looking at a drug called inclisiran to see if it's safe and effective for children aged 6 to 11 who have heterozygous familial hypercholesterolemia (HeFH). HeFH is a genetic condition that causes very high levels of "bad" cholesterol (LDL-C). Inclisiran is being compared to a placebo (an inactive substance like sterile saline). The main goal is to see how much inclisiran changes LDL-C levels after about a year. You might be able to join if you are 6 to 11 years old, have HeFH, and your LDL-C is above a certain level. If you are 8 to 11, you should also be on cholesterol-lowering medication unless you can't tolerate it. The study is currently recruiting participants.

Study design
This is a two-part study involving 60 participants. For the first year, some participants will receive inclisiran and others will receive a placebo, and neither you nor your doctor will know which you are getting. For the second year, all participants will receive inclisiran.
What's involved
The study lasts for two years. Your body weight will be measured on Day 1 and Day 360 to determine the correct inclisiran dose. Your LDL-C levels will be measured at the beginning and again at Day 330.
Compensation
Not stated in the trial record.
Follow-up
Participants are followed for at least one year, with LDL-C levels measured at Day 330.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT06597019

Study to Evaluate Efficacy and Safety of Inclisiran in Children With Heterozygous Familial Hypercholesterolemia

Recruiting
PHASE3Ages 6–11InterventionalTreatment
Novartis Pharmaceuticals
~60 participants
Updated 2026-05-15 on ClinicalTrials.gov
What's tested:InclisiranPlacebo

At a glance

Recruiting sites
64 of 66 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Percentage change in LDL-C from baseline to Day 330 (Year 1)
Measured over Baseline and Day 330

Conditions

Familial Hypercholesterolemia - Heterozygous

Explore related trials

Hyperlipoproteinemia, Type IIa

NCT06597019

Where you'd take part

This study runs at 66 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • Children's National Hospital

    Washington D.C., District of Columbiastudy coordinator listed

    Not yet recruiting

  • Childrens National Hospital

    Washington D.C., District of Columbiastudy coordinator listed

    Recruiting

  • Excel Medical Clinical Trials LLC

    Boca Raton, Floridastudy coordinator listed

    Recruiting

  • Icahn School of Med at Mt Sinai

    New York, New Yorkstudy coordinator listed

    Recruiting

  • Primary Childrens Medical Center

    Salt Lake City, Utahstudy coordinator listed

    Not yet recruiting

  • Primary Childrens Medical Center

    Salt Lake City, Utahstudy coordinator listed

    Recruiting

  • UC San Francisco Medical Center

    San Francisco, Californiastudy coordinator listed

    Recruiting

  • UC San Francisco Medical Center

    San Francisco, Californiastudy coordinator listed

    Recruiting

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Study leadership

  • Novartis Pharmaceuticals · STUDY_DIRECTOR · Novartis Pharmaceuticals

Who to contact

Novartis Pharmaceuticals
[email protected]
1-888-669-6682
Email the study team

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Eligibility criteria

Inclusion

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

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

What this trial measures

  • Percentage change in LDL-C from baseline to Day 330 (Year 1)Baseline and Day 330

    Demonstrate superiority of inclisiran compared to placebo in reducing LDL-C \[percent change\] at Day 330