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Study on Inclisiran, Alirocumab, and Rosuvastatin for Children with 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 heterozygous familial hypercholesterolemia (HeFH), which is a genetic disorder that causes high levels of cholesterol in the blood. The study aims to compare the effectiveness of different treatments in children with this condition. The treatments being tested include a combination of rosuvastatin, a medication taken orally, and either alirocumab or inclisiran, both of which are given as injections under the skin. These treatments will be compared to the standard therapy of rosuvastatin alone.

The purpose of the study is to see how well these treatments work in lowering cholesterol levels to a specific target after 104 weeks. Participants in the study will receive one of the treatment combinations or the standard therapy. The study will last for about two years, during which participants will have regular check-ups to monitor their cholesterol levels and overall health. The goal is to determine which treatment is most effective in helping children with HeFH reach healthier cholesterol levels.

Throughout the study, participants will be monitored for changes in their cholesterol levels and other health markers. The study will also assess the impact of the treatments on the thickness of the arteries, which is measured using a method called carotid intima-media thickness (CIMT). Additionally, the study will evaluate the quality of life of participants using a questionnaire designed for children. This research is important for finding better ways to manage high cholesterol in children with HeFH and improving their long-term health outcomes.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial visit
Upon joining the study, the first step involves an initial visit. During this visit, informed written consent is obtained for participation in the study, genetic testing, and processing of personal data.
Eligibility is confirmed based on age (10 years to 15 years and 6 months) and specific cholesterol levels. A negative pregnancy test is required for menstruating girls, and consent to use contraceptive methods is obtained if applicable.
Step 2
Baseline measurements
Baseline measurements are taken to assess initial health status. This includes measuring levels of LDL cholesterol, total cholesterol, and other related parameters.
A questionnaire assessing quality of life is completed to establish a baseline for future comparisons.
Step 3
Treatment initiation
Participants are assigned to one of the treatment groups. The treatments include rosuvastatin taken orally, and either alirocumab or inclisiran administered via subcutaneous injection.
The goal of the treatment is to achieve an LDL-C level of less than 100 mg/dl over the course of 104 weeks.
Step 4
Follow-up visits
Follow-up visits occur at 24 weeks, 60 weeks, and 104 weeks. During these visits, cholesterol levels and other health parameters are measured again.
The quality of life questionnaire is repeated to assess any changes since the baseline visit.
Step 5
Final assessment
At the end of the 104-week period, a final assessment is conducted. This includes measuring cholesterol levels and other health parameters to determine the effectiveness of the treatment.
The final quality of life questionnaire is completed to evaluate any long-term changes.

Who can join the trial?

7 criteria

Obtain informed written consent for the patient to participate in the study, for genetic testing, and for the processing of personal data.
Age between 10 years and 15 years and 6 months.
LDL level from screening visit (V1):
- (a) LDL ≥ 190 mg/dl, regardless of family history, or
- (b) LDL ≥ 160 mg/dl with a positive family history (in first-degree relatives and/or siblings: LDL > 190 mg/dl and/or with early cardiovascular disease, and/or corneal stroma, and/or tendonitis), or
- (c) LDL ≥ 130 mg/dl with a confirmed genetic mutation in at least one parent.
- Negative serum pregnancy test in menstruating girls.
- Consent to the use of contraceptive methods as described in the study protocol.

Who cannot join the trial?

8 criteria

Patients who do not have heterozygous familial hypercholesterolemia cannot participate. This is a genetic condition that causes high cholesterol levels.
Patients who are not within the specified age range for the study cannot participate. The study is for a specific age group.
Patients who are not willing or able to follow the study procedures cannot participate.
Patients who have other medical conditions that might interfere with the study cannot participate.
Patients who are taking medications that might interfere with the study cannot participate.
Patients who are pregnant or breastfeeding cannot participate.
Patients who have participated in another clinical trial recently cannot participate.
Patients who have a history of allergic reactions to the study medications cannot participate.

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Verified sites

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

Uniwersyteckie Centrum Kliniczne
Verified
Gdansk, Poland
Wojewodzki Specjalistyczny Szpital Im Dr Wl Bieganskiego
Verified
Lodz, Poland

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Investigated drugs

Alirocumab
is a medication given as an injection under the skin. It is used to help lower levels of "bad" cholesterol, known as LDL cholesterol, in the blood. This medication works by blocking a specific protein in the liver, which helps the body remove more LDL cholesterol from the blood. In this trial, it is being tested to see how well it works in children with a condition that causes high cholesterol levels.
Rosuvastatin
is a medication taken by mouth in the form of a pill. It belongs to a group of drugs called statins, which are used to lower cholesterol levels in the blood. Rosuvastatin works by reducing the amount of cholesterol made by the liver, helping to lower "bad" cholesterol and raise "good" cholesterol. In this trial, it is used as a standard treatment to compare with other therapies.
Inclisiran
is a medication given as an injection under the skin. It is designed to lower LDL cholesterol levels by targeting a specific genetic process in the liver. Inclisiran helps the liver remove more LDL cholesterol from the blood, which can be beneficial for people with high cholesterol levels. In this trial, it is being tested to see how effective it is in children with high cholesterol due to a genetic condition.

What is already known about the treatment

Inclisiran
Inclisiran is administered as a subcutaneous injection, meaning it is given under the skin. It is currently being studied in clinical trials for its effectiveness in treating heterozygous familial hypercholesterolemia, a genetic condition that causes high cholesterol levels. Inclisiran works by targeting a specific molecule in the liver to reduce the production of LDL cholesterol, often referred to as "bad" cholesterol. It belongs to a class of medications known as small interfering RNA (siRNA) therapies, which help lower cholesterol levels in the blood.
Alirocumab
Alirocumab is also administered as a subcutaneous injection. It is being evaluated in clinical trials for its role in managing heterozygous familial hypercholesterolemia. Alirocumab functions by inhibiting a protein called PCSK9, which leads to an increase in the number of receptors on liver cells that remove LDL cholesterol from the blood. This medication is classified as a PCSK9 inhibitor and is used to help lower cholesterol levels.
Rosuvastatin
Rosuvastatin is taken orally in pill form. It is a well-established medication used to treat high cholesterol, including in cases of heterozygous familial hypercholesterolemia. Rosuvastatin works by blocking an enzyme in the liver that is responsible for making cholesterol, thereby reducing the overall cholesterol levels in the blood. It is part of a group of drugs known as statins, which are commonly prescribed to manage cholesterol levels.

Investigated diseases

Heterozygous Familial Hypercholesterolemia – This genetic disorder is characterized by high levels of low-density lipoprotein cholesterol (LDL-C) in the blood, which can lead to early cardiovascular disease. It is caused by a mutation in one of the genes responsible for removing LDL-C from the bloodstream. As a result, individuals with this condition have elevated cholesterol levels from birth. Over time, the excess cholesterol can deposit in the walls of arteries, leading to atherosclerosis. This process can begin in childhood and progress with age, increasing the risk of heart attacks and other cardiovascular issues. The condition is inherited in an autosomal dominant pattern, meaning one copy of the altered gene is sufficient to cause the disorder.