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Study on the Safety and Effectiveness of Inclisiran for Children Aged 6 to Under 12 with Familial Hypercholesterolemia

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a condition called Heterozygous Familial Hypercholesterolemia, which is a genetic disorder that causes high levels of low-density lipoprotein cholesterol (LDL-C), often referred to as "bad cholesterol." The study is specifically for children aged 6 to under 12 years who have this condition. The treatment being tested is a medication called Inclisiran, which is also known by its code name KJX839. Inclisiran is given as an injection and is designed to help lower LDL-C levels in the blood.

The purpose of the study is to evaluate the safety and effectiveness of Inclisiran in reducing LDL-C levels in children with this condition. The study is divided into two parts. In the first year, participants will receive either Inclisiran or a placebo, which is a substance with no active medication. In the second year, all participants will receive Inclisiran. The study will monitor changes in LDL-C levels and other health markers over time to assess the impact of the treatment.

Throughout the study, participants will have regular check-ups to track their health and any changes in their cholesterol levels. The study aims to provide valuable information on how well Inclisiran works in children with Heterozygous Familial Hypercholesterolemia and to ensure that it is safe for use in this age group. This research could lead to better treatment options for managing high cholesterol in children with this genetic condition.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment

    The study begins with an initial assessment to confirm eligibility. Participants must be between 6 and less than 12 years old, diagnosed with heterozygous familial hypercholesterolemia, and have a fasting LDL cholesterol level greater than 130 mg/dL.

    Participants aged 8 to less than 12 years should be on a stable dose of statin unless intolerant, with or without other lipid-lowering therapy. Those under 8 years may have different treatment based on the investigator's discretion.

  2. Step 2

    Year 1: double-blind phase

    Participants receive either inclisiran or a placebo through subcutaneous injection. The goal is to evaluate the reduction in LDL cholesterol by Day 330.

    The primary objective is to demonstrate the superiority of inclisiran compared to placebo in reducing LDL cholesterol levels.

  3. Step 3

    Year 2: open-label phase

    All participants receive inclisiran during this phase. The focus is on long-term safety, tolerability, and efficacy.

    The study continues to monitor changes in LDL cholesterol and other lipid parameters up to Day 720.

  4. Step 4

    Monitoring and assessments

    Throughout the study, regular assessments are conducted to monitor LDL cholesterol levels, other lipid parameters, and any adverse events.

    Participants' growth, vital signs, and development are also tracked to ensure safety and well-being.

Who can join the trial?

5 criteria

  • Participants must be **male or female** and between **6 to less than 12 years old** at the time of screening.
  • Participants must have a condition called **Heterozygous Familial Hypercholesterolemia (HeFH)**, which can be diagnosed through genetic testing or based on certain physical characteristics.
  • Participants must have a **fasting LDL-C level** greater than **130 mg/dL (3.4 mmol/L)** at screening. **LDL-C** stands for **low-density lipoprotein cholesterol**, often referred to as "bad" cholesterol.
  • For participants aged **8 to less than 12 years**, they should be on an **optimal dose of statin** medication, unless they cannot tolerate statins. They may also be on other cholesterol-lowering treatments like **ezetimibe**. For participants younger than 8 years, the use of cholesterol-lowering treatments is decided by the study doctor.
  • Participants who are already taking cholesterol-lowering medications, such as **statins** or **ezetimibe**, must have been on a **stable dose for at least 30 days** before screening and should not have any planned changes to their medication or dosage during the study.

Who cannot join the trial?

3 criteria

  • Children who are not between the ages of 6 and 12 years old.
  • Children who do not have a condition called Heterozygous Familial Hypercholesterolemia, which is a genetic disorder that causes high levels of cholesterol in the blood.
  • Children who do not have elevated levels of LDL-C, which stands for low-density lipoprotein cholesterol, often referred to as "bad" cholesterol.
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Investigated drugs

Inclisiran: Inclisiran is a medication used to lower levels of LDL cholesterol, often referred to as "bad" cholesterol, in the blood. It works by helping the liver remove more LDL cholesterol from the bloodstream. This medication is being studied for its effectiveness and safety in children with a condition called heterozygous familial hypercholesterolemia, which causes high levels of LDL cholesterol. The trial aims to see if inclisiran can significantly reduce LDL cholesterol levels in these children.

What is already known about the treatment

Inclisiran – Inclisiran is administered as an injection under the skin. It is currently being studied in clinical trials for its effectiveness and safety in treating children with heterozygous familial hypercholesterolemia, a condition characterized by high levels of LDL cholesterol. The medication works by targeting and reducing the production of a protein called PCSK9, which in turn helps lower LDL cholesterol levels in the blood. Inclisiran is classified as a small interfering RNA (siRNA) therapy, which is a type of medication that interferes with specific genetic information to prevent the production of certain proteins.

Investigated diseases

Heterozygous Familial Hypercholesterolemia – This is a genetic disorder characterized by high levels of low-density lipoprotein cholesterol (LDL-C) in the blood. It is caused by a mutation in one of the genes responsible for removing LDL-C from the bloodstream. Individuals with this condition inherit one copy of the mutated gene from one parent. As a result, they have elevated cholesterol levels from a young age, which can lead to cholesterol deposits in various parts of the body. Over time, these deposits can cause narrowing of the arteries. This condition is often identified in childhood or early adulthood due to its genetic nature.
Trial detailsLast updated 2 Oct 2026
Age0-17PhasePhase IIITrial ID2024-514594-21-00Protocol codeCKJX839C12303Estimated enrolment67 patientsSponsorNovartis Pharma AG

sourced from the EU Clinical Trials Register and site verification

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