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
- Coimbra, Lisbon, and Porto, PortugalSee the 3 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 6 and 11 years old?
Source: NCT06597019, who can take part
Where in Portugal
- CoimbraCoimbra, PortugalTaking people
- LisbonLisbon, PortugalTaking people
- PortoPorto, PortugalTaking 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
- 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.