RecruitingPHASE3INTERVENTIONAL

Long-term Safety and Tolerability of Inclisiran in Participants With HeFH or HoFH Who Have Completed the Pediatric ORION-16, ORION-13, ORION-20, or ORION-19 Studies

This study is checking the long-term safety and how well people tolerate a medicine named inclisiran. It's for individuals aged 12 to 100 years old who have a genetic condition causing very high cholesterol, called Heterozygous или Homozygous Familial Hypercholesterolemia (HeFH or HoFH). Participants in this study have already taken part in previous inclisiran studies (like ORION-16, ORION-13, ORION-20, or ORION-19). The main goal is to understand if inclisiran continues to be safe over a longer period and to make sure those who benefited from it can keep receiving the medicine.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

Novartis Pharmaceuticals

Enrolment target

195

Start

10 Feb 2023

Estimated completion

28 Mar 2032

Heterozygous or Homozygous Familial Hypercholesterolemia

What is this study about?

This study is exploring a medicine called inclisiran over a longer period. Inclisiran is used to help people with very high cholesterol, especially if this high cholesterol is caused by a genetic condition called Familial Hypercholesterolemia (FH). There are two main types of FH: Heterozygous (HeFH) and Homozygous (HoFH).

The main purpose of this study is to see how safe inclisiran is when people take it for a long time, and how well their bodies handle it. It's especially for those who have already participated in other inclisiran studies in the past, like the ORION-16, ORION-13, ORION-20, or ORION-19 studies. By continuing to observe these participants, researchers can gather important information about the medicine's long-term effects.

Another important reason for this study is to give individuals who have already benefited from inclisiran in the earlier studies a chance to continue receiving the treatment. This helps ensure that people who need this medicine can keep taking it while more information is collected about its long-term use.

Key takeaways

This study is about the long-term safety of inclisiran for high cholesterol.
It's for people with genetic high cholesterol (HeFH or HoFH).
Only people who were in previous inclisiran ORION studies can join.
Participants will continue to receive inclisiran treatment.
The study aims to provide ongoing treatment access and more safety data.

Who may be eligible?

To join this study, you must be a male or female aged 12 to 100 years old and have been diagnosed with HeFH or HoFH. A key requirement is that you must have already successfully completed one of the previous inclisiran studies called ORION-16, ORION-13, ORION-20, or ORION-19. Your study doctor from those previous trials must also believe that you genuinely benefited from the inclisiran treatment.

There are also some reasons why you might not be able to join. For example, if you didn't finish one of the previous ORION studies for any reason, or if you had serious safety problems or couldn't tolerate inclisiran in those earlier studies, you wouldn't be able to participate. Also, if you have any other serious health conditions that could make it unsafe for you to be in the study or make it hard to understand the study results, you would likely not be eligible.

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.

I am between 12 and 100 years old.
I have been diagnosed with HeFH or HoFH.
I have successfully completed one of the previous ORION-16, ORION-13, ORION-20, or ORION-19 studies.
My study doctor believes I benefited from inclisiran in the previous study.
I don't have any other serious health conditions that would make taking part risky.

Answer every question to see your result.

What does participation involve?

If you join this study, you will continue to receive the inclisiran medicine. Because this is an 'open-label' study, both you and your study team will know you are receiving inclisiran. The study will involve regular visits to a clinic at different times to check your health, blood tests to monitor your cholesterol levels and how your body is reacting to the treatment, and discussions with your study doctor or nurse about any side effects or changes you notice. The study keeps collecting information for an extended period, allowing researchers to understand the long-term effects of inclisiran.

Potential risks and benefits

Taking part in this study may offer the benefit of continuing to receive inclisiran, which has previously helped manage your high cholesterol. However, as with any medicine, there is a possibility of side effects, even in the long term. These could be effects you've experienced before or new ones. The study team will carefully monitor your health throughout. Remember, participating in any study is completely your choice, and you are free to withdraw at any time without it affecting your usual medical care.

Locations (52)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Excel Medical Clinical Trials LLC
Verified postcode
Boca Raton, United States· Completed
Icahn School of Med at Mt Sinai
Verified postcode
New York, United States· Active not recruiting
Cincinnati Childrens Hospital MC
Verified postcode
Cincinnati, United States· Completed
Childrens Hosp Pittsburgh UPMC
Verified postcode
Pittsburgh, United States· Active not recruiting
Primary Childrens Medical Center
Verified postcode
Salt Lake City, United States· Completed
Novartis Investigative Site
Verified postcode
Formosa, Argentina· Active not recruiting
Novartis Investigative Site
Verified postcode
Fortaleza, Brazil· Active not recruiting
Novartis Investigative Site
Verified postcode
São Paulo, Brazil· Completed
Novartis Investigative Site
Verified postcode
São Paulo, Brazil· Active not recruiting
Novartis Investigative Site
Verified postcode
Québec, Canada· Active not recruiting
Novartis Investigative Site
Verified postcode
Prague, Czechia· Active not recruiting
Novartis Investigative Site
Verified postcode
Prague, Czechia· Active not recruiting

Common questions

What is HeFH or HoFH?

These are genetic conditions that cause very high levels of cholesterol in your blood, making you more likely to develop heart problems.

What is inclisiran?

Inclisiran is a medicine designed to help lower cholesterol levels in people with conditions like HeFH or HoFH.

What does 'long-term safety and tolerability' mean?

It means the study will check if inclisiran remains safe to use over many months or even years, and how well people's bodies continue to handle the medicine without too many side effects.

Why is this study only for people who were in previous ORION studies?

This study is an extension, meaning it's a follow-up to earlier studies. It focuses on gathering more long-term information from people who have already started treatment with inclisiran.

Will I know if I'm getting the real medicine?

Yes, in this study, both you and your doctor will know that you are receiving inclisiran. It’s called an 'open-label' study.

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.