Tests inclisiran in children with inherited high cholesterol
Tests inclisiran against placebo in children aged 6 to 11 with high cholesterol.
Plain-language title generated from the registry record
Registry title: Study to Evaluate Efficacy and Safety of Inclisiran in Children With Heterozygous Familial Hypercholesterolemia
Key facts
- Where
- Budapest, HungarySee the site
- Phase
- Phase 3
AI summary: who the study is looking for
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for children aged 6 to under 12 years with heterozygous familial hypercholesterolemia (HeFH) and high LDL cholesterol.
- Age
- 6 to under 12 years
- Sex
- Boys and girls can join.
- Disease stage
- Heterozygous familial hypercholesterolemia (HeFH), diagnosed by genetic testing or by typical signs. Fasting LDL-C above 130 mg/dL (3.4 mmol/L).
- Earlier treatment
- Children aged 8 to under 12 take an optimal statin dose unless they cannot tolerate statins. Children under 8: the investigator decides on lipid-lowering treatment. Lipid-lowering medicines must be at a stable dose for at least 30 days before screening.
- Biomarker or test result
- Fasting LDL-C above 130 mg/dL (3.4 mmol/L) at screening.
Main reasons for not taking part
- Treatment with PCSK9 monoclonal antibodies within 90 days of screening
- Secondary hypercholesterolemia, for example hypothyroidism or nephrotic syndrome
- Homozygous familial hypercholesterolemia (HoFH)
- Body weight under 16 kg (35 lb)
- Active liver disease, or pregnant or nursing females
Read by machine on 12 November 2026.
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 6 and 11 years old?
Source: NCT06597019, who can take part
Where in Hungary
- BudapestHospital not named in the registryContact: Novartis PharmaceuticalsTaking 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?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorTrial details
- Who runs it
- Novartis Pharmaceuticals
- Trial code
- NCT06597019
- Record updated
- 15 May 2026
- Original description
This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in children (aged 6 to <12 years) with heterozygous familial hypercholesterolemia (HeFH) 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 (6 to Less Than 12 Years) With Heterozygous 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 6 to <12 years) with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDLC).
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; 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); Homozygous familial hypercholesterolemia (HoFH); Body weight <16 kg at the screening and/or randomization (Day 1) visit; Pregnant or nursing females; Recent and/or planned use of other investigational medicinal products or devices.