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 52 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 and 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 study looks at whether a medicine called inclisiran is effective and safe for children aged 6 to under 12 with heterozygous familial hypercholesterolaemia (HeFH) — an inherited condition causing high cholesterol. The trial is run by Novartis Pharmaceuticals.
Who can take part
- Boys or girls aged 6 to under 12 at screening
- Diagnosed with heterozygous familial hypercholesterolaemia, either by genetic testing or based on signs and symptoms
- Fasting 'bad' cholesterol (LDL-C) above 130 mg/dL (3.4 mmol/L) at screening
- Children aged 8 to under 12 should be on the best dose of a statin medicine (unless they cannot tolerate it), with or without other cholesterol-lowering medicines such as ezetimibe; for children under 8, taking cholesterol medicine is decided by the study doctor
- If already taking cholesterol-lowering medicines, the dose must have been stable for at least 30 days before screening, with no planned changes during the study
Who may not be able to
- Treatment within the last 90 days with monoclonal antibody medicines targeting PCSK9 (a protein involved in cholesterol control)
- High cholesterol caused by another condition, such as an underactive thyroid or nephrotic syndrome (a kidney problem)
- Homozygous familial hypercholesterolaemia (a more severe form of the condition)
- Body weight under 16 kg at screening or on Day 1
- Active liver disease, including unexplained high liver enzyme levels (ALT or AST over 3 times the normal limit) or high bilirubin (over 2 times the normal limit, except with Gilbert's syndrome)
- Girls who are pregnant or breastfeeding
- Recent or planned use of other experimental medicines or devices
What taking part involves
- • Taking part involves receiving the study medicine, inclisiran, so researchers can assess how effective and safe it is — further details of how it is given and the study groups are not stated — ask the trial team
- • Keeping any existing cholesterol medicines at a stable dose, with no planned changes, throughout the study
Time commitment: Not stated — ask the trial team (the data does not say how long the study lasts, how many visits are needed, or what tests are involved).
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
- 6 Years to 11 Years
- Who
- All
- Number of participants
- 60
- Started
- 2024-12-09
- Last checked
- 2026-05
Plain English Summary
What is this study?
- • Testing a new treatment for familial hypercholesterolemia - heterozygous
- • Phase3 - 60 participants
- • 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)
Who can take part?
- • Ages 6 Years to 11 Years
- • Diagnosed with familial hypercholesterolemia - heterozygous
Where?
- • West Midlands - Novartis Investigative Site
- • London - 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 6 to \<12 years) with heterozygous familial hypercholesterolemia (HeFH) 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 6 to \<12 years) with heterozygous familial hypercholesterolemia (HeFH) 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: 6 Years - 11 Years
- Who can join: All genders
What the study is looking for
- ✓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
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
- ✗Homozygous familial hypercholesterolemia (HoFH)
- ✗Body weight \<16 kg at the screening and/or randomization (Day 1) visit
- ✗Pregnant or nursing females
See the full criteria
Where Is This Study? (2 UK sites)
Novartis Investigative Site
West Midlands B4 6NH, United Kingdom
Novartis Investigative Site
London NW3 2QG, United Kingdom
How to Get in Touch
Novartis Pharmaceuticals
Sponsor contactCONTACT
Novartis Pharmaceuticals
Sponsor contactCONTACT