At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- Inclisiran (drug), Placebo (drug)
- How long the study runs
- Study runs about 50 months (dates as stated)
- About the drug or intervention
- Inclisiran — drug: Inclisiran (inclisiran sodium 300 mg subcutaneous (s.c.) for participants with body weight ≥23 kg, inclisiran sodium 180 mg s.c. · Placebo — drug: Sterile normal saline (0.9% sodium chloride in water for subcutaneous injection)
- Patient visit burden
- Not specified by the sponsor
In plain English
This Novartis Pharmaceuticals study is testing how safe, tolerable and effective a medicine called inclisiran is for children aged 2 to under 12 with homozygous familial hypercholesterolemia (HoFH) — an inherited condition causing very high cholesterol from birth. The study aims are to evaluate safety, tolerability and efficacy. How long the study lasts is not stated — ask the trial team.
Who can take part
- Boys and girls aged 2 to under 12 at screening
- HoFH confirmed by genetic testing
- Fasting LDL cholesterol (low-density lipoprotein cholesterol, often called 'bad cholesterol') above 130 mg/dL (3.4 mmol/L) at screening
- Taking an optimal dose of a statin medicine unless statin-intolerant, with or without other cholesterol-lowering medicines such as ezetimibe
- Any cholesterol-lowering medicines must be at a stable dose for at least 30 days before screening, with no planned changes during the study
- Children having LDL-apheresis (a blood-filtering treatment that removes cholesterol) for at least 3 months before screening may continue it during the study, but the schedule must stay the same during the double-blind part of the trial and must allow a session at each study visit
Who may not be able to
- A 'null' (negative) mutation in both LDLR genes
- Treatment with PCSK9 monoclonal antibody medicines within 90 days of screening, or a history of poor response to them (for example, less than a 15% drop in LDL cholesterol)
- Treatment with mipomersen or lomitapide within 5 months of screening
- High cholesterol caused by another condition, such as an underactive thyroid or nephrotic syndrome
- Heterozygous familial hypercholesterolemia (HeFH), a different inherited form of high cholesterol
- Body weight under 16 kg for children aged 6 to under 12, or under 11 kg for children aged 2 to under 6
- Active liver disease or unexplained raised liver blood tests (ALT or AST more than 3 times the upper limit of normal, or bilirubin more than 2 times the upper limit of normal, except Gilbert's syndrome)
- Girls who are pregnant or nursing
- Recent or planned use of other experimental medicines or devices
What taking part involves
- • The study medicine inclisiran, given alongside existing cholesterol-lowering treatment
- • The trial includes a double-blind period, meaning neither the family nor the study team knows who is receiving which treatment (for example, inclisiran or a dummy medicine) — details of what is given at each stage are not stated — ask the trial team
Time commitment: Not stated — ask the trial team. The data says children keep taking their existing cholesterol medicines at unchanged doses, apheresis sessions (if used) must line up with each study visit, and blood samples are taken at screening, but the number of visits and total study length are not stated.
Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.
- Type of study
- Testing a treatment
- Ages
- 2 Years to 11 Years
- Who
- All
- Number of participants
- 9
- Started
- 2025-02-28
- Last checked
- 2026-07
Plain English Summary
What is this study?
- • Testing a new treatment for familial hypercholesterolemia - homozygous
- • Phase3 - 9 participants
- • 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)
Who can take part?
- • Ages 2 Years to 11 Years
- • Diagnosed with familial hypercholesterolemia - homozygous
Where?
- • Southampton - Novartis Investigative Site
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
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).
More detail
This is a two-part (1 year double-blind inclisiran versus placebo / 1 year open-label inclisiran) multicenter 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 (LDL-C) on stable standard of care background lipid-lowering therapy.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 2 Years - 11 Years
- Who can join: All genders
What the study is looking for
- ✓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...
- ✓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...
Who cannot take part
- ✗Documented evidence of a null (negative) mutation in both LDLR alleles
- ✗Previous treatment (within 90 days of screening) with monoclonal antibodies directed towards PCSK9
- ✗History of poor response to therapy with any monoclonal antibody directed towards PCSK9 (e.g. \<15% reduction in LDL-C)
- ✗Treatment with mipomersen or lomitapide (within 5 months of screening)
- ✗Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome
See the full criteria
Where Is This Study? (1 UK site)
Novartis Investigative Site
Southampton SO16 6YD, United Kingdom
How to Get in Touch
Novartis Pharmaceuticals
Sponsor contactCONTACT
Novartis Pharmaceuticals
Sponsor contactCONTACT