AuthorisedTherapeutic use (Phase IV)Interventional

Reassessment of statin-associated musscle symptoms in adults with familial hypercholesterolemia: A phase IV randomized double-blinded n-of-1 crossover trial including periods with atorvastatin, placebo and no study treatment

This study is for adults with a genetic condition called familial hypercholesterolaemia, which causes very high cholesterol. Many people who take statins for this condition report muscle pain. This research aims to understand if these muscle symptoms are truly caused by the statin or by other factors. It does this by comparing periods where participants take atorvastatin (a common statin) with periods where they take a dummy pill (placebo) or no treatment at all. The study will carefully measure muscle pain to see how it changes during each period. This helps researchers figure out the real connection between statins and muscle aches.

At a glance

Status

Authorised

Phase

Therapeutic use (Phase IV)

Sponsor

Oslo University Hospital HF

Enrolment target

Start

22 May 2026

Familial hypercholesterolaemia

What is this study about?

This study is designed for adults who have a condition called familial hypercholesterolaemia. This is a genetic condition meaning it runs in families and causes very high levels of cholesterol, which can increase the risk of heart disease. To manage this, people often take medicines called statins. However, some people who take statins report experiencing muscle pain or weakness, and it isn't always clear if these symptoms are actually caused by the statin or by something else.

The main goal of this research is to get a clearer picture of whether a commonly used statin, called atorvastatin, truly causes muscle symptoms in people with familial hypercholesterolaemia. The study wants to find out if there's a real difference in how much muscle pain people feel when they are taking the statin compared to when they are not.

To do this, participants will go through different periods where they take atorvastatin, a dummy pill (placebo), or no treatment at all. The study is set up so that neither the participant nor the study staff will know which treatment is being given at any particular time. This helps to make the results as fair and accurate as possible. By carefully comparing muscle symptoms during these different periods, the researchers hope to better understand the link between statins and muscle pain, which could help many patients in the future.

Key takeaways

Study explores muscle pain in people with familial high cholesterol taking statins.
Compares atorvastatin, a dummy pill, and no treatment.
Helps understand if statins cause muscle symptoms in this group.
Participants and staff won't know which treatment is given at the time.
Aims to improve care for people with familial hypercholesterolaemia.

Who may be eligible?

To be considered for this study, you need to be an adult, at least 18 years old, and have a diagnosed medical condition called familial hypercholesterolaemia. This condition means you have very high cholesterol levels that run in your family.

Both men and women can take part in this study. The researchers are looking for a wide range of adults with familial hypercholesterolaemia to ensure the results apply to many different people.

Apart from having familial hypercholesterolaemia and being an adult, the detailed rules for who can join are very specific. They will consider your overall health, any other medications you take, and other medical conditions. These details will be discussed with you thoroughly by the study team to make sure it's safe and appropriate for you to participate.

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 18 years old or older?
Have you been diagnosed with familial hypercholesterolaemia?
Are you comfortable potentially taking statin or placebo medications?
Are you male or female and interested in taking part?
Are you prepared to attend necessary appointments and follow study instructions?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will go through several different treatment periods. During some periods, you will take capsules that contain atorvastatin, a common statin. In other periods, you will take capsules that look exactly the same but contain no active medicine (a placebo). There will also be periods where you don't take any study medication at all. You won't know which type of capsule you're taking, and neither will the study doctors or nurses, until after the study is complete. This helps to make the results more reliable. Your muscle symptoms will be carefully monitored and rated, likely using a simple scale, particularly during the last two weeks of each treatment period. The overall length of the study for each participant will be discussed with you by the research team, as these types of “n-of-1” studies are tailored to the individual, meaning the total duration can vary.

Potential risks and benefits

Taking part in any study has potential benefits and risks. A potential benefit could be gaining a better understanding of your own muscle symptoms and whether they are linked to statin use. This knowledge might help you and your doctor make more informed decisions about your treatment in the future. As with any medication, even a placebo, there's always a small risk of unexpected reactions, though they are carefully monitored. The study uses a well-known statin, atorvastatin, and its effects are generally well understood. You are absolutely free to leave the study at any time, for any reason, without it affecting your usual medical care.

Locations (1)

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

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Unverified
Norway

Common questions

What is familial hypercholesterolaemia?

It's a common inherited condition that causes very high cholesterol levels from birth, increasing the risk of heart disease.

What is a 'statin'?

Statins are a group of medicines commonly prescribed to lower high cholesterol levels and reduce the risk of heart attacks and strokes.

What is a 'placebo'?

A placebo is a dummy pill that looks like the real medicine but doesn't contain any active drug. It helps compare the effects of the actual medicine fairly.

Why don't I know if I'm getting the statin or the placebo?

This 'blinded' approach ensures that your expectations or the researchers' hopes don't accidentally influence the results, making the study more accurate.

Could I still take my other medications during the study?

The study team will review all your current medications to ensure they don't interfere with the study or your safety. Speak to them for specific advice.