RecruitingPHASE3INTERVENTIONAL

Study to Evaluate Efficacy and Safety of Inclisiran in Children With Heterozygous Familial Hypercholesterolemia

This important study is looking into a medicine called inclisiran for children between 6 and 11 years old. These children have a genetic condition known as heterozygous familial hypercholesterolemia (HeFH), which causes high levels of 'bad' cholesterol (LDL-C). The main goal is to find out if inclisiran is safe and effective in helping to lower these cholesterol levels. Half of the children will receive inclisiran, and the other half will get a placebo (a dummy medicine) for the first year. After that, all children will receive inclisiran. All participants will also continue their usual cholesterol-lowering treatments. The study will last for two years.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

Novartis Pharmaceuticals

Enrolment target

Start

09 Dec 2024

Estimated completion

15 Apr 2029

Familial Hypercholesterolemia - Heterozygous

What is this study about?

This study is about a new medicine called inclisiran, and it's being tested in children aged 6 to 11 who have a condition called heterozygous familial hypercholesterolemia (HeFH). HeFH is a genetic condition passed down through families that causes very high levels of 'bad' cholesterol (LDL-C) from a young age. High cholesterol can lead to serious heart problems later in life, so finding effective ways to manage it early on is really important.

The study aims to see two main things: first, if inclisiran is safe for these children, and second, if it can effectively lower their high 'bad' cholesterol levels. For the first year, some children will receive inclisiran, while others will get a placebo (a treatment with no active medicine). This helps researchers fairly compare how well inclisiran works. For the second year, all children in the study will receive inclisiran.

All children taking part will also continue with their usual cholesterol-lowering medicines, such as statins, if they are already on them. This study is crucial because it could help doctors understand if inclisiran can be a new and safe option to help children with HeFH manage their cholesterol and reduce their risk of heart disease in the future.

Key takeaways

Tests a new cholesterol medicine (inclisiran) for children with HeFH.
Aimed at children aged 6-11 years with high 'bad' cholesterol.
Compares inclisiran to a dummy medicine (placebo) for one year, then all get inclisiran.
Children will continue their usual cholesterol medicines.
Focuses on safety and how well it lowers cholesterol over two years.
Helps researchers find new ways to treat high cholesterol in children.

Who may be eligible?

The study is looking for boys and girls who are between 6 and 11 years old. They must have a diagnosis of heterozygous familial hypercholesterolemia (HeFH), either confirmed by a genetic test or by their doctor based on specific criteria. Their 'bad' cholesterol (LDL-C) levels must also be above a certain level.

Children aged 8 to 11 should already be on a stable dose of their usual cholesterol-lowering medicine, like a statin, unless they can't take them. For younger children (under 8), the decision about background medication is up to their doctor. Anyone on cholesterol-lowering medicines must have been on a steady dose for at least one month before starting the study and expect to keep that dose throughout.

However, some children won't be able to join. This includes those who have recently had treatment with certain other cholesterol-lowering drugs (monoclonal antibodies towards PCSK9), have other health problems like certain liver diseases, or types of cholesterol problems not related to HeFH. Girls who are pregnant or breastfeeding also cannot participate. Your doctor will carefully check all these requirements to see if this study is right for your child.

Quick self-check

Is my child between 6 and 11 years old?
Does my child have a diagnosis of heterozygous familial hypercholesterolemia (HeFH)?
Does my child have high 'bad' cholesterol (LDL-C) despite their current treatment (if any)?
If my child is 8-11, are they on a stable dose of a statin or similar medicine, or cannot take them?
Is my child generally healthy, without other serious liver issues or certain other conditions?
Is my child a girl who is not pregnant or breastfeeding?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If your child takes part, the study will last for two years. For the first year, they will either receive the study medicine, inclisiran, or a placebo (a dummy medicine without active ingredients), given as injections. Neither you nor the study team will know which one your child is getting during this first year. In the second year, all children will receive inclisiran.

Throughout the study, your child will have regular visits to the clinic for health checks, blood tests to measure their cholesterol and general health, and to receive their study medication. They will continue to take any other cholesterol-lowering medicines their doctor has prescribed, provided the dose stays the same. The doctors and nurses will monitor your child's health closely to look for any side effects and to see how well the medicine is working. You will be given full details of the visit schedule if you decide to consider participation.

Potential risks and benefits

Taking part in this study could potentially offer some benefits to your child. If inclisiran proves to be effective, it might help lower their high cholesterol levels, which is important for their long-term heart health. You will also receive close medical attention and monitoring throughout the study. However, like all medicines, inclisiran could have side effects, and some blood tests or procedures might cause minor discomfort. It's also possible that your child will receive the placebo for the first year and therefore not experience any direct medical benefit from the study drug during that period. You have the right to withdraw your child from the study at any time, for any reason, without it affecting their ongoing medical care.

Locations (66)

UC San Francisco Medical Center
San Francisco, United States· Recruiting
UC San Francisco Medical Center
San Francisco, United States· Recruiting
Children's National Hospital
Washington D.C., United States· Not yet recruiting
Childrens National Hospital
Washington D.C., United States· Recruiting
Excel Medical Clinical Trials LLC
Boca Raton, United States· Recruiting
Icahn School of Med at Mt Sinai
New York, United States· Recruiting
Primary Childrens Medical Center
Salt Lake City, United States· Not yet recruiting
Primary Childrens Medical Center
Salt Lake City, United States· Recruiting
Virginia Commonwealth University
Richmond, United States· Recruiting
West Virginia Childrens Hospital
Morgantown, United States· Recruiting
West Virginia Childrens Hospital
Morgantown, United States· Recruiting
Novartis Investigative Site
Buenos Aires, Argentina· Recruiting

+54 more sites — see the official record for the full list.

Common questions

What is HeFH?

HeFH (heterozygous familial hypercholesterolemia) is a genetic condition meaning your body handles cholesterol differently, leading to very high levels of 'bad' cholesterol (LDL-C) from childhood.

How is the study medicine given?

The study medicine, inclisiran, is given as an injection, but your doctor or nurse will explain exactly how and when this will happen.

Will my child still take their usual medicines?

Yes, your child will continue to take their usual cholesterol-lowering medicines at a stable dose throughout the study.

How long does the study last?

The study will last for two years, with regular check-ups and monitoring during that time.

Can I take my child out of the study?

Yes, you can withdraw your child from the study at any time, for any reason, and it won't affect their medical care.

How to find out more

Novartis Pharmaceuticals

novartis.email@novartis.com 1-888-669-6682 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.