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Taking people in United KingdomPhase 3

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.

Key facts

Who runs it
Novartis Pharmaceuticals
Where
Southampton, United KingdomSee the siteAlso in 10 other countries, 18 sites
Registry title
Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children With Homozygous Familial Hypercholesterolemia
Status
Recruiting
Phase
Phase 3
People taking part
9 people, planned
Started
28 Feb 2025
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
24 July 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 2 to under 12 years with genetically confirmed homozygous familial hypercholesterolemia and high LDL-cholesterol. It tests the safety and effect of inclisiran.

Age
2 to under 12 years
Sex
Boys and girls
Disease stage
Homozygous familial hypercholesterolemia (HoFH), confirmed by a genetic test.
Earlier treatment
Must take an optimal statin dose unless statin intolerant, with or without other lipid-lowering treatment such as ezetimibe. Doses must be stable for at least 30 days before screening. LDL-apheresis for at least 3 months may continue if stable.
Biomarker or test result
Fasting LDL-C above 130 mg/dL (3.4 mmol/L) at screening.

Main reasons for not taking part

  • Known null (negative) mutations in both LDLR alleles
  • Treatment with PCSK9 antibodies within 90 days, or poor past response to them
  • Treatment with mipomersen or lomitapide within 5 months
  • Heterozygous familial hypercholesterolemia or secondary hypercholesterolemia
  • Body weight under 16 kg (35 lb) (ages 6 to under 12) or under 11 kg (24 lb) (ages 2 to under 6)

The registry criteria, word for word

Show all 16 criteriaShow less

Who can take part

  • 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

Who cannot take part

  • 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

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 2 and 11 years old?

Where in United Kingdom

Also in 10 other countries, 18 sites

  • SouthamptonHospital 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 2 to <12 years) with homozygous familial hypercholesterolemia (HoFH) 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 (2 to Less Than 12 Years) With Homozygous Familial Hypercholesterolemia and Elevated LDL-cholesterol
Plain-language title
Tests inclisiran in children with a rare inherited high cholesterol conditionGenerated 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 NCT06597006, who can take part
Record updated
24 July 2026