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Evaluating Inclisiran for Children with Familial Hypercholesterolemia

Two Part (Double-blind Inclisiran Versus Placebo [Year 1] Followed by Open-label Inclisiran [Year 2]) Randomized Multicenter Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children (6 to Less Than 12 Years) With Heterozygous Familial Hypercholesterolemia and Elevated LDL- Cholesterol

Phase 3 Interventional Novartis · NCT06597019

This study is testing if a new medication called inclisiran can safely lower cholesterol levels in children aged 6 to under 12 who have a genetic condition that causes high cholesterol.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment51 (estimated)
Ages6 Years to 11 Years
SexAll
SponsorNovartis Industry-sponsored
Locations66 sites (San Francisco, California and 65 other locations)
Trial IDNCT06597019 on ClinicalTrials.gov

What this trial studies

This pivotal phase III study aims to assess the safety, tolerability, and efficacy of inclisiran in children aged 6 to less than 12 years who have heterozygous familial hypercholesterolemia (HeFH) and elevated LDL cholesterol levels. The study consists of two parts: a one-year double-blind phase comparing inclisiran to a placebo, followed by a one-year open-label phase where all participants receive inclisiran. Participants must be on stable background lipid-lowering therapy, and the study will monitor their LDL-C levels throughout the trial.

Who should consider this trial

Good fit: Ideal candidates are children aged 6 to less than 12 years diagnosed with heterozygous familial hypercholesterolemia and elevated LDL-C levels.

Not a fit: Patients with homozygous familial hypercholesterolemia or those with secondary causes of hypercholesterolemia may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide a new treatment option for children with HeFH, potentially lowering their LDL cholesterol levels and reducing cardiovascular risk.

How similar studies have performed: Other studies have shown promising results with inclisiran in adult populations, but this specific application in children is novel.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* 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 Criteria:

* 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

Where this trial is running

San Francisco, California and 65 other locations

+16 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Familial Hypercholesterolemia - HeterozygousHeterozygous familial hypercholesterolemia,LDL-cholesterol,Children,pediatric,small interfering ribonucleic acid,inclisiran,Familial Hypercholesterolemia,
Last reviewed 2026-06-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.