Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children With Homozygous Familial Hypercholesterolemia
This important study is investigating a new medication, inclisiran, for children aged 2 to 11 who have a rare and severe inherited condition called homozygous familial hypercholesterolemia (HoFH). Children with HoFH have very high levels of 'bad' cholesterol (LDL-C) from birth, which can lead to serious health problems. The study aims to find out if inclisiran is a safe and effective way to help lower these high cholesterol levels. It will compare inclisiran against a placebo (a dummy drug) over one year, followed by another year where all participants receive inclisiran. Participants will already be on their usual cholesterol-lowering treatments. The findings could help improve treatments for children with HoFH.
At a glance
What is this study about?
This research study is about a condition called homozygous familial hypercholesterolemia, often shortened to HoFH. This is a very rare genetic condition where children are born with extremely high levels of 'bad' cholesterol (LDL-C). This isn't caused by diet or lifestyle, but by faulty genes inherited from both parents. Because their cholesterol is so high from a young age, children with HoFH are at a much higher risk of developing serious heart problems early in life.
The study is looking at a new medicine called inclisiran. It's designed to help the body get rid of more 'bad' cholesterol from the blood. The main purpose of this study is to see if inclisiran is safe for children with HoFH, if their bodies can handle it well, and if it's effective at lowering their high cholesterol levels when added to their existing treatments.
This study is a 'Phase 3' trial, which means it's a key step in checking if new medicines work and are safe before they can be made widely available. The results of this study will help doctors and researchers understand if inclisiran could be a valuable new treatment option for children living with HoFH.
Key takeaways
- Targets very high cholesterol (HoFH) in children aged 2-11.
- Compares a new medicine, inclisiran, to a placebo first.
- Aims to check safety, how well it's tolerated, and effectiveness.
- Study lasts for two years, with regular clinic visits.
- Participation is voluntary, and you can withdraw at any time.
Who may be eligible?
This study is looking for children aged between 2 and 11 years old. To be considered, children must have a confirmed diagnosis of homozygous familial hypercholesterolemia (HoFH) through genetic testing. They also need to have high levels of 'bad' cholesterol (LDL-C) that are above a certain point, even with their current treatments.
Children currently taking cholesterol-lowering medicines like statins or ezetimibe must have been on a stable dose for at least one month before joining the study, with no plans to change these doses. If a child receives a treatment called LDL-apheresis (a procedure to remove cholesterol from the blood), they must have been doing this regularly for at least three months, and this treatment schedule needs to stay the same during the first part of the study.
Some children would not be able to take part. This includes those with a specific genetic mutation called a 'null mutation' in both their LDLR genes, or if they've recently used or had a poor response to similar cholesterol-lowering medications. Children with other medical problems, like active liver disease or certain serious health conditions, would also be excluded.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Is your child aged between 2 and 11 years old?
- Does your child have a confirmed diagnosis of homozygous familial hypercholesterolemia (HoFH)?
- Is your child's LDL-C ('bad' cholesterol) still high despite their current treatments?
- Has your child been on a stable dose of their current cholesterol medicines for at least one month?
- Does your child currently weigh enough for their age group?
What does participation involve?
If your child takes part in this study, it will last for two years. For the first year, your child would be randomly assigned to receive either the study medicine, inclisiran, or a placebo (a dummy treatment that looks like inclisiran but has no active medicine). Neither you nor the study team will know which treatment your child is receiving during this first year. Both inclisiran and the placebo are given as injections.
After the first year, all children in the study will then receive inclisiran for another year. Throughout the study, your child will continue their usual cholesterol-lowering treatments. There will be regular visits to the clinic, which will involve check-ups, blood tests to monitor cholesterol levels and general health, and assessments to see how your child is feeling and responding to the treatment. The total duration of participation in the study for each child would be two years.
Potential risks and benefits
Locations (18)
- UC San Francisco Medical CenterVerified postcodeSan Francisco, United States· Recruiting
- UC San Francisco Medical CenterVerified postcodeSan Francisco, United States· Recruiting
- Childrens National HospitalVerified postcodeWashington D.C., United States· Recruiting
- Washington Univ School Of MedicineVerified postcodeSt Louis, United States· Recruiting
- Novartis Investigative SiteVerified postcodeVienna, Austria· Recruiting
- Novartis Investigative SiteVerified postcodeBeijing, China· Recruiting
- Novartis Investigative SiteVerified postcodeFrankfurt am Main, Germany· Recruiting
- Novartis Investigative SiteVerified postcodeIoannina, Greece· Recruiting
- Novartis Investigative SiteVerified postcodeThessaloniki, Greece· Recruiting
- Novartis Investigative SiteVerified postcodeKota Bharu, Malaysia· Recruiting
- Novartis Investigative SiteVerified postcodeAmsterdam, Netherlands· Recruiting
- Novartis Investigative SiteVerified postcodeBloemfontein, South Africa· Recruiting
Common questions
What is homozygous familial hypercholesterolemia (HoFH)?
HoFH is a rare genetic condition where children are born with very high levels of 'bad' cholesterol (LDL-C) due to faulty genes inherited from both parents, leading to an increased risk of heart problems.
What is inclisiran?
Inclisiran is a new investigational medicine designed to help lower 'bad' cholesterol (LDL-C) levels in the blood.
Will my child get the actual medicine?
For the first year, your child will either receive inclisiran or a look-alike placebo. For the second year, all children will receive inclisiran.
How long will the study last for my child?
If your child participates, their involvement in the study will last for two years.
Can we stop participating at any time?
Yes, you are free to withdraw your child from the study at any point, and their regular medical care will not be affected.
How to find out more
Novartis Pharmaceuticals
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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