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Study of Inclisiran for Children with Homozygous Familial Hypercholesterolemia

This study is looking at the safety, how well it's tolerated, and how effective inclisiran is in children aged 2 to under 12 years old who have homozygous familial hypercholesterolemia (HoFH). HoFH is a genetic condition that causes very high levels of "bad" cholesterol (LDL-C). Inclisiran works by targeting a specific protein to help lower cholesterol. Some participants will receive inclisiran, while others will receive a placebo (an inactive substance like saline). The main goal is to see how much inclisiran can lower LDL-C levels after about one year. To join, you must have HoFH confirmed by genetic testing and high LDL-C levels. The study plans to enroll 9 children.

Study design
This is a two-part study: one year where participants receive either inclisiran or a placebo, followed by one year where all participants receive inclisiran. It is a multicenter study.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
The primary endpoint measures changes in LDL-C from the start of the study to Day 330 (approximately one year).

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

NCT06597006

Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children With Homozygous Familial Hypercholesterolemia

Recruiting
PHASE3Ages 2–11InterventionalTreatment
Novartis Pharmaceuticals
~9 participants
Updated 2026-07-24 on ClinicalTrials.gov
What's tested:InclisiranPlacebo

At a glance

Recruiting sites
19 of 19 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 - Homozygous

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Hyperlipoproteinemia, Type IIa

NCT06597006

Where you'd take part

This study runs at 19 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • Childrens National Hospital

    Washington D.C., District of Columbiastudy coordinator listed

    Recruiting

  • Primary Childrens Medical Center

    Salt Lake City, Utahstudy coordinator listed

    Recruiting

  • UC San Francisco Medical Center

    San Francisco, Californiastudy coordinator listed

    Recruiting

  • Washington Univ School Of Medicine

    St Louis, Missouristudy coordinator listed

    Recruiting

  • Novartis Investigative Site

    Vienna, Austriano site contact published

    Recruiting

  • Novartis Investigative Site

    Beijing, Beijing Municipality, Chinano site contact published

    Recruiting

  • Novartis Investigative Site

    Frankfurt am Main, Hesse, Germanyno site contact published

    Recruiting

  • Novartis Investigative Site

    Ioannina, Greeceno site contact published

    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, 2 to \<12 years of age at screening
HoFH diagnosed by genetic confirmation

Exclusion

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.
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

What this trial measures

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

    Evaluate the effect of inclisiran compared to placebo on reducing LDL-C \[percent change\] at Day 330