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Evaluating inclisiran for children with a rare cholesterol condition

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 (2 to Less Than 12 Years) With Homozygous Familial Hypercholesterolemia and Elevated LDL-cholesterol

Phase 3 Interventional Novartis · NCT06597006

This study is testing if a new cholesterol-lowering drug called inclisiran is safe and effective for children aged 2 to under 12 who have a rare condition that causes very high cholesterol levels.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment9 (estimated)
Ages2 Years to 11 Years
SexAll
SponsorNovartis Industry-sponsored
Locations18 sites (San Francisco, California and 17 other locations)
Trial IDNCT06597006 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 2 to less than 12 years who have been diagnosed with homozygous familial hypercholesterolemia (HoFH) and exhibit elevated levels of low-density lipoprotein cholesterol (LDL-C). The study consists of two parts: a one-year double-blind comparison of inclisiran versus placebo followed by a one-year open-label phase where all participants receive inclisiran. Participants must be on a stable background lipid-lowering therapy, and the study will monitor their response to the treatment over the course of two years.

Who should consider this trial

Good fit: Ideal candidates are children aged 2 to less than 12 years with genetically confirmed homozygous familial hypercholesterolemia and elevated LDL-C levels.

Not a fit: Patients with known null mutations in both LDLR alleles will not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly lower cholesterol levels in children with HoFH, improving their long-term health outcomes.

How similar studies have performed: Other studies have shown promise with similar approaches using inclisiran in adults, but this specific application in children is novel.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* Male or female participants, 2 to \<12 years of age at screening
* HoFH diagnosed by genetic confirmation

  \- Note: Participants with known null (negative) mutations in both LDLR alleles are not eligible (see also exclusion criteria)
* 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.

Exclusion Criteria:

* 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

Where this trial is running

San Francisco, California and 17 other locations

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 - HomozygousHomozygous familial hypercholesterolemia,LDL-cholesterol, children, pediatric,small interfering ribonucleic acid,inclisiran,Familial Hypercholesterolemia,Homozygous FH,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.