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Taking people in United KingdomPhase 3

Tests inclisiran in children with inherited high cholesterol

Tests inclisiran against placebo in children aged 6 to 11 with high cholesterol.

Key facts

Who runs it
Novartis Pharmaceuticals
Where
London and West Midlands, United KingdomSee the 2 sitesAlso in 22 other countries, 58 sites
Registry title
Study to Evaluate Efficacy and Safety of Inclisiran in Children With Heterozygous Familial Hypercholesterolemia
Status
Recruiting
Phase
Phase 3
People taking part
60 people, planned
Started
9 Dec 2024
Expected to end
15 Apr 2029
Type of study
Interventional, 2 groupsOne group gets a placebo (a dummy treatment), for comparison.
Treatments tested
  • Inclisiran
  • Placebo
Record updated
15 May 2026

Who can take part

AI summary

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

The registry criteria, word for word

Show all 12 criteriaShow less

Who can take part

  • 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

Who cannot take part

  • 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

Check if you might fit

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?

Where in United Kingdom

Also in 22 other countries, 58 sites

  • LondonHospital not named in the registryContact: Novartis PharmaceuticalsTaking people
  • West Midlands, BirminghamHospital not named in the registryContact: Novartis PharmaceuticalsTaking people

About the trial

What the trial wants to find out

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).

The 2 groups in the trial

  • InclisiranTreatment being tested

    Year 1 - inclisiran sodium subcutaneous injection (given at Days 1, 90, and 270) Day 360 only - placebo subcutaneous injection Year 2 - inclisiran sodium subcutaneous injection (given at Days 450 and 630)

  • PlaceboPlacebo, for comparison

    Year 1 - placebo subcutaneous injection (given at Days 1, 90 and 270) Year 2 - inclisiran sodium subcutaneous injection (given at Days 360, 450, and 630)

Questions to ask the study team

  1. How often do I need to come in, and for how long each time?
  2. Are travel costs paid?
  3. Can I keep my own doctor?
  4. What happens if I want to stop?

Contact the study team: see the registry record → Contacts and Locations

Print a page for my doctor

Data and sources

Registry record
Listed on ClinicalTrials.gov.
Official title
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
Plain-language title
Tests inclisiran in children with inherited high cholesterolGenerated from the registry record. The registry title is the official one.
AI summary
From the trial's eligibility criteria. Read by machine on 12 November 2026.
Fit questions
Built from NCT06597019, who can take part
Record updated
15 May 2026