RecruitingRare disease

Study on the Safety and Effectiveness of Inclisiran in Children Aged 2 to Under 12 with Homozygous Familial Hypercholesterolemia

Verified siteRegistered drugNo placebo

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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 homozygous familial hypercholesterolemia, a genetic disorder that causes very high levels of cholesterol in the blood from a young age. The study is testing a treatment called inclisiran, which is also known by its code name KJX839. Inclisiran is a medication given as an injection and is being compared to a placebo to see how well it works in lowering cholesterol levels in children aged 2 to under 12 years.

The purpose of the study is to evaluate the safety and effectiveness of inclisiran in reducing levels of LDL-cholesterol, often referred to as "bad cholesterol," in children with this condition. The study is designed in two parts: the first year involves a comparison between inclisiran and a placebo, while the second year allows all participants to receive inclisiran. This approach helps researchers understand both the immediate and longer-term effects of the treatment.

Participants in the study will receive injections of inclisiran or a placebo and will have regular check-ups to monitor their cholesterol levels and overall health. The study aims to see how much inclisiran can lower LDL-cholesterol and to ensure it is safe for use in children. The trial will also track any side effects and assess the children's growth and development throughout the study period.

The research process

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

Step 1
Initial assessment
Participants aged 2 to under 12 years with a confirmed diagnosis of homozygous familial hypercholesterolemia are assessed for eligibility.
Eligibility includes having a fasting LDL-C level greater than 130 mg/dL and being on a stable dose of lipid-lowering therapy for at least 30 days before the trial.
Step 2
Year 1: double-blind phase
Participants receive either inclisiran or a placebo through subcutaneous injection.
The primary goal is to evaluate the effect of inclisiran on reducing LDL-C levels by Day 330.
Participants continue their existing lipid-lowering therapies without changes during this period.
Step 3
Year 2: open-label phase
All participants receive inclisiran in an open-label manner.
The focus is on monitoring changes in LDL-C and other cholesterol-related markers up to Day 720.
Regular assessments include checking for any side effects, monitoring growth, and evaluating pubertal development.
Step 4
End of study assessments
Final evaluations are conducted to measure the percentage change in LDL-C from the start of the trial to the end.
Additional assessments include the incidence and severity of any adverse effects experienced during the trial.

Who can join the trial?

6 criteria

Participants must be **male or female** and between **2 to less than 12 years old** at the time of screening.
Participants must have a diagnosis of **HoFH** (homozygous familial hypercholesterolemia) confirmed by genetic testing. This means they have a specific genetic condition that affects cholesterol levels.
Participants must have a **fasting LDL-C** (low-density lipoprotein cholesterol) level greater than **130 mg/dL** at screening. LDL-C is often referred to as "bad cholesterol."
Participants should be on an **optimal dose of statin** medication, unless they cannot tolerate statins. Statins are drugs used to lower cholesterol levels. They may also be on other cholesterol-lowering treatments like **ezetimibe**.
If participants are taking cholesterol-lowering medications, they must have been on a **stable dose for at least 30 days** before screening, with no plans to change the medication or dose during the study.
Participants who are undergoing **LDL-apheresis** (a procedure to remove LDL cholesterol from the blood) must have been on a stable regimen for at least **3 months before screening**. The schedule and settings for this procedure must remain stable during the study and should align with study visits.

Who cannot join the trial?

3 criteria

Children who do not have homozygous familial hypercholesterolemia cannot participate. This is a condition where a person has very high levels of cholesterol from birth due to a genetic issue.
Children who are not between the ages of 2 and less than 12 years old cannot participate.
Children who do not have elevated levels of LDL-C cannot participate. LDL-C stands for low-density lipoprotein cholesterol, often called "bad" cholesterol.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

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Amsterdam UMC
Amsterdam, The Netherlands
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Assistance Publique Hopitaux De Paris
Paris, France
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Goethe University Frankfurt
Frankfurt, Germany

Trial locations

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Investigated drugs

Inclisiran is a medication being studied for its ability to lower LDL cholesterol levels in children with a condition called homozygous familial hypercholesterolemia (HoFH). This condition causes very high levels of LDL cholesterol, which can lead to heart problems. Inclisiran works by helping the liver remove more LDL cholesterol from the blood. In this study, the medication is being tested to see how well it works and how safe it is for children aged 2 to less than 12 years.

Inclisiran

What is already known about the treatment

Inclisiran – Inclisiran is administered via subcutaneous injection, meaning it is injected under the skin. It is currently being studied in clinical trials, particularly for its safety, tolerability, and effectiveness in children with homozygous familial hypercholesterolemia, a genetic condition that causes very high cholesterol levels. The main therapeutic use of inclisiran is to lower LDL cholesterol levels. It works by inhibiting a specific protein involved in cholesterol production, leading to reduced levels of LDL cholesterol in the blood. Inclisiran is classified as a small interfering RNA (siRNA) medication, which targets and degrades specific RNA molecules to prevent the production of certain proteins.

Investigated diseases

Homozygous Familial Hypercholesterolemia – This is a genetic disorder characterized by extremely high levels of low-density lipoprotein cholesterol (LDL-C) from birth. It occurs when both parents pass on a defective gene that affects the body's ability to remove LDL-C from the blood. As a result, cholesterol builds up in the bloodstream, leading to the formation of deposits in the skin, tendons, and arteries. Over time, these deposits can cause narrowing and hardening of the arteries, a condition known as atherosclerosis. This disease progresses rapidly, often leading to cardiovascular complications at a young age. It is considered a rare condition and requires careful management to control cholesterol levels.