RecruitingPHASE3INTERVENTIONAL

A Phase 3 Study of Zodasiran in Adolescent and Adult Subjects With Homozygous Familial Hypercholesterolemia (YOSEMITE)

This research study, called YOSEMITE, is looking into a new medicine called zodasiran. It's for teenagers and adults, aged 12 and over, who have a rare and severe inherited condition called homozygous familial hypercholesterolemia (HoFH). HoFH causes extremely high cholesterol levels from birth, which can lead to serious heart problems at a young age. The study aims to find out if zodasiran injections are safe and effective at lowering these high cholesterol levels. Some participants will receive zodasiran, while others will receive a dummy medicine (placebo) first, before everyone eventually gets the active drug. Your participation could help improve treatments for HoFH in the future.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

Arrowhead Pharmaceuticals

Enrolment target

Start

17 Jun 2025

Estimated completion

20 Aug 2027

Homozygous Familial Hypercholesterolemia

What is this study about?

Imagine your body struggling to clear out bad cholesterol, leading to dangerously high levels from a very young age. This is what happens to people with Homozygous Familial Hypercholesterolemia (HoFH), a rare and serious inherited condition. These extremely high cholesterol levels can cause serious health problems, including heart disease, much earlier in life than usual.

This study, named YOSEMITE, is testing a new medicine called zodasiran. Researchers want to see if zodasiran can safely and effectively lower the very high cholesterol levels in people aged 12 and over with HoFH. By taking part, you could help doctors understand more about how to manage this condition and potentially improve future treatments.

The study is 'double-blind', meaning neither you nor your doctor will know if you are receiving zodasiran or a dummy medicine (placebo) at first. This helps ensure the results are accurate. After this initial period, everyone will have the chance to receive the actual zodasiran medicine in a longer, open-label part of the study.

Key takeaways

This study is testing a new medicine for severe inherited high cholesterol (HoFH).
It's for people aged 12 and above.
Participants will receive either zodasiran or a dummy medicine first.
Everyone can eventually receive the active medicine.
The goal is to see if zodasiran can safely lower cholesterol levels.
Your participation could help advance HoFH treatment.

Who may be eligible?

To join this study, you would need to be at least 12 years old and weigh at least 35 kg (about 5 stone 7 pounds). If you can become pregnant, you shouldn't be pregnant or breastfeeding, and should not plan to become pregnant during the study.

You would need to have a confirmed diagnosis of HoFH, either through a genetic test or based on certain cholesterol levels and other signs like fatty deposits on your skin or tendons from a young age. Your 'bad' cholesterol (LDL-C) levels would also need to be above a certain amount, even if you're already taking cholesterol-lowering medicines.

There are also some health conditions or medicines that would prevent you from joining. For example, if you've recently used certain other trial medicines or have severe kidney problems, you might not be able to take part. The study also requires you to be stable on your current cholesterol-lowering medicines.

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.

Are you 12 years old or older?
Do you have a diagnosis of HoFH?
Are you currently on cholesterol-lowering medicines?
If female, are you not pregnant or breastfeeding, and not planning to become pregnant during the study?
Are your cholesterol levels still high despite existing treatment?
Do you meet the weight requirement of at least 35 kg?

Answer every question to see your result.

What does participation involve?

If you decide to join, you'll first be randomly assigned to receive either the study medicine, zodasiran, or a dummy medicine (placebo) through injections under your skin. Neither you nor your study doctor will know which you are receiving at this stage. This initial 'double-blind' period will last for several months.

Throughout the study, you'll have regular visits to the clinic for health checks, blood tests, and to make sure the treatment is working and you're not experiencing any problems. After the initial period, you'll have the option to continue in a longer part of the study where everyone will receive the active zodasiran medicine. We don't have the exact number of visits or the total length of the study available right now, but these details would be explained fully by the study team.

Potential risks and benefits

Taking part in a study like this can have potential benefits, such as receiving a new treatment that might help lower your cholesterol levels. You'll also receive close medical attention and contribute valuable information that could help others with HoFH in the future. However, there are also potential risks, including side effects from the study medicine or discomfort from injections and blood tests. Remember, you can stop participating in the study at any time, for any reason, without it affecting your usual medical care.

Locations (43)

Research Site 7
Verified postcode
Park Ridge, United States· Recruiting
Research Site 2
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New York, United States· Recruiting
Research Site 1
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Cincinnati, United States· Recruiting
Research Site 14
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Pittsburgh, United States· Recruiting
Research Site 13
Verified postcode
Camperdown, Australia· Recruiting
Research Site 9
City only
Saint Leonards, Australia· Recruiting
Research Site 21
Verified postcode
Heidelberg, Australia· Recruiting
Research Site 3
Verified postcode
Nedlands, Australia· Recruiting
Research Site 40
Verified postcode
Innsbruck, Austria· Recruiting
Research Site 39
Verified postcode
Linz, Austria· Recruiting
Research Site 42
Verified postcode
Vienna, Austria· Recruiting
Research Site 37
Verified postcode
Vienna, Austria· Recruiting

Common questions

What is HoFH?

HoFH (Homozygous Familial Hypercholesterolemia) is a very rare inherited condition that causes extremely high cholesterol levels from birth, leading to a high risk of heart disease at a young age.

What is zodasiran?

Zodasiran is an investigational medicine being tested in this study to see if it can help lower very high cholesterol levels in people with HoFH.

What is a 'placebo'?

A placebo is a dummy treatment that looks exactly like the study medicine but doesn't contain any active drug. It helps researchers accurately see if the real medicine is effective.

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

For the first part of the study, neither you nor your doctor will know if you're getting zodasiran or the placebo. Later, everyone will have the option to receive the active zodasiran.

How is the medicine given?

The study medicine, zodasiran, is given as an injection under the skin.

How to find out more

Medical Monitor

yosemite@arrowheadpharma.com 626-304-3400 Official trial record

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