Tests inclisiran in children with a rare inherited high cholesterol condition
Tests inclisiran against placebo in children aged 2 to 11 with homozygous familial hypercholesterolemia.
Plain-language title generated from the registry record
Registry title: Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children With Homozygous Familial Hypercholesterolemia
Key facts
- Where
- Salt Lake City, San Francisco, St Louis, and Washington D.C., United StatesSee the 4 sites
- Phase
- Phase 3
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 2 and 11 years old?
Source: NCT06597006, who can take part
Where in United States
- Primary Childrens Medical CenterSalt Lake City, United StatesTaking people
- UC San Francisco Medical CenterSan Francisco, United StatesTaking people
- Washington Univ. School of MedicineSt Louis, United StatesTaking people
- Childrens National HospitalWashington D.C., United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Novartis Pharmaceuticals
- Trial code
- NCT06597006
- Record updated
- 24 July 2026
- Original description
This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in children (aged 2 to <12 years) with homozygous familial hypercholesterolemia (HoFH) and elevated low density lipoprotein cholesterol (LDLC).Read full original descriptionShow less
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
This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in children (aged 2 to <12 years) with homozygous familial hypercholesterolemia (HoFH) and elevated low density lipoprotein cholesterol (LDLC).
Inclusion: 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: Previous treatment (within 90 days of screening) with monoclonal antibodies directed towards PCSK9; Treatment with mipomersen or lomitapide (within 5 months of screening); History of poor response to therapy with any monoclonal antibody directed towards PCSK9 (e.g. <15% reduction in LDL-C); Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome; 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); 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); Documented evidence of a null (negative) mutation in both LDLR alleles; Heterozygous familial hypercholesterolemia (HeFH); Pregnant or nursing females; Recent and/or planned use of other investigational medicinal products or devices.