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Taking people in GermanyPhase 3

Tests inclisiran in young children with a rare inherited high-cholesterol disease

Tests inclisiran against placebo in children aged 2 to under 12 with homozygous familial hypercholesterolemia.

Key facts

Who runs it
Novartis Pharma AG
Where
Frankfurt Am Main, GermanySee the siteAlso in 5 other countries, 6 sites
Trial code
Registry title
Study to evaluate efficacy and safety of inclisiran in children aged 2 to under 12 years of age with homozygous familial hypercholesterolemia
Status
Recruiting
Phase
Phase 3
People taking part
8 people, planned
Started
5 Aug 2025
Expected to end
4 Jan 2030
Type of study
Interventional
Record updated
26 October 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 who have homozygous familial hypercholesterolemia confirmed by a genetic test and high LDL-cholesterol.

Age
2 to under 12 years at screening
Sex
Boys and girls can join.
Disease stage
Homozygous familial hypercholesterolemia (HoFH) confirmed by a genetic test, with fasting LDL-C above 130 mg/dL (3.4 mmol/L) at screening.
Earlier treatment
Children must take an optimal dose of a statin, unless they cannot tolerate statins, with or without other cholesterol-lowering treatment such as ezetimibe. Doses must be stable for at least 30 days (at least 90 days for evinacumab) before screening. LDL-apheresis is allowed if stable for at least 3 months. Children cannot join if they had a PCSK9 antibody within 90 days, or mipomersen or lomitapide within 5 months before screening.
Biomarker or test result
Genetic confirmation of HoFH. Children with known null (negative) mutations in both LDLR alleles cannot join.

Main reasons for not taking part

  • Known null (negative) mutation in both LDLR alleles
  • Heterozygous familial hypercholesterolemia
  • Body weight under 16 kg (35 lb) (ages 6 to under 12) or under 11 kg (24 lb) (ages 2 to under 6)
  • Active liver disease or raised liver tests
  • Pregnant or nursing females

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
  • 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 (≥90 days for evinacumab) 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
  • Recent and/or planned use of other investigational medicinal products or devices
  • Heterozygous familial hypercholesterolemia (HeFH)
  • Pregnant or nursing females

Where in Germany

Also in 5 other countries, 6 sites

  • Goethe University FrankfurtFrankfurt Am Main, GermanyTaking people

About the trial

What the trial wants to find out

Primary objective is to evaluate the effect of inclisiran compared to placebo on reducing LDL-C [percent change] at Day 330 (Year 1) in children (aged 2 to <12 years) with HoFH and elevated LDL-C

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

Print a page for my doctor

Data and sources

Registry record
Listed on the EU's official list of trials (CTIS).
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 (ORION-19)
Plain-language title
Tests inclisiran in young children with a rare inherited high-cholesterol diseaseGenerated from the registry record. The registry title is the official one.
AI summary
From the trial's eligibility criteria. Read by machine on 9 November 2026.
Record updated
26 October 2026