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Atorvastatin, Placebo, and No Treatment in Adults with Familial Hypercholesterolaemia and Statin-Associated Muscle Symptoms

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This study is being done in adults with familial hypercholesterolaemia, a condition that causes very high levels of cholesterol in the blood. It is looking at atorvastatin, a medicine used to lower cholesterol, and placebo. The purpose of the study is to find out whether muscle symptoms are truly caused by atorvastatin or whether they are linked to other reasons.

The study uses a step-by-step treatment plan. Each person will have periods with atorvastatin, periods with placebo, and a period with no study treatment. The order of these periods is arranged by chance. During the study, muscle symptoms are checked over time to compare how they change during each period.

The study is designed to help understand statin-associated muscle symptoms, which are muscle problems that some people notice while taking statin medicines such as atorvastatin. It also looks at whether some symptoms may be nocebo or misattributed, meaning they are felt as real symptoms but may not be caused by the medicine itself.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Trial treatment periods begin
After joining the study, you enter a series of treatment periods in a randomized order. Randomized means the order is assigned by chance.
The trial is double-blinded, which means neither you nor the study staff know whether you are taking atorvastatin or placebo during a treatment period.
Some periods include atorvastatin 80 mg taken by mouth once daily. The tablet is a film-coated tablet.
Some periods include placebo tablets taken by mouth once daily. A placebo looks like a study tablet but does not contain active medicine.
Some periods include no study treatment.
Step 2
Crossover sequence continues
You move through the different study periods in a crossover design. This means you receive more than one type of study period during the trial.
The study includes periods with atorvastatin, periods with placebo, and periods with no study treatment.
Each treatment period is used to compare your muscle symptoms across the different study conditions.
Step 3
Symptom rating during treatment
During the trial, you rate your muscle symptoms on a 0 to 100 scale, where higher numbers mean stronger symptoms.
The main comparison uses the average symptom intensity during the last two weeks of week 5 to week 6 in the treatment periods with atorvastatin and placebo.
The trial looks at whether the symptom difference between these periods is at least 10 units and also at least 25% on the 0 to 100 scale.
Step 4
Assessment of study results
Your symptom ratings are used to assess whether your muscle symptoms are more closely linked to atorvastatin, to placebo, or to no study treatment.
The study also examines how well the symptom pattern can distinguish between these treatment periods.

Who can join the trial?

8 criteria

Be an adult, meaning 18 years of age or older when signing the informed consent form.
Have a confirmed diagnosis of heterozygous familial hypercholesterolemia based on a genetic test, or have a clinical diagnosis of familial hypercholesterolemia with a Dutch Lipid Clinic Network Score of 9 or higher. This score is a medical way of measuring how likely the condition is.
Have a history of muscle symptoms that you believed were caused by statin treatment, and these symptoms must have happened with at least two different statins. Statins are medicines used to lower cholesterol.
Have stopped taking statins because of these muscle symptoms, and be off statin treatment for at least 2 weeks before the first study assessment.
If you are a woman who can become pregnant (a premenopausal woman who is not permanently sterile), have a negative pregnancy test before joining the study and agree to use highly effective birth control or to avoid sexual activity during the study treatment period.
Be able and willing to follow all study rules, including the requirements and restrictions described in the informed consent form and study plan.
Have access to a smartphone, tablet, or personal computer for the whole study.
Be able to sign the informed consent form, which means you understand the study and agree to take part.

Who cannot join the trial?

14 criteria

Having been hospitalized for an unplanned heart or blood vessel event caused by blocked arteries in the past 6 months.
Having a life expectancy of less than 24 months because of another medical condition. Life expectancy means how long a person is expected to live.
Having a blood test result showing creatine kinase (CK) that is 5 times or more above the normal limit at the start of the study. CK is a muscle enzyme found in the blood, and a high level can mean muscle injury.
Taking part in another interventional clinical study at the same time. An interventional study is a study where a treatment or procedure is given.
Having a past history of rhabdomyolysis, which is a severe breakdown of muscle that can damage the kidneys.
Having a past history of myopathy, meaning a muscle disease, with creatine kinase 10 times or more above the normal limit.
Having a past history of liver injury linked to statin treatment, shown by alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) levels that were 3 times or more above the normal limit. ALT and AST are blood tests that can show liver damage.
Having had severe kidney disease in the last 12 months, defined as an estimated glomerular filtration rate (eGFR) below 30 mL/min/1.73 m². eGFR is a test that shows how well the kidneys are working.
Having any condition, such as a psychiatric illness, dementia (memory and thinking problems), or substance abuse, that the study doctor thinks could put the person at risk, affect the study results, or make it hard to take part in the study.
Having any reason that makes atorvastatin unsafe to use, including:
- Known allergy or strong sensitivity to atorvastatin or its ingredients.
- ALT level that is 3 times or more above the normal limit at the start of the study.
- Pregnancy.
- Breastfeeding.
- Currently using another medicine that should not be taken with atorvastatin or that is not safe to use with atorvastatin 80 mg.

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Verified sites

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Oslo Universitetssykehus HF
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Oslo, Norway
St. Olavs Hospital HF
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Trondheim, Norway

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Investigated drugs

Atorvastatin is a statin medicine taken by mouth to lower cholesterol. In this trial, it is the active treatment being studied to see whether it causes muscle symptoms in people who think they have had statin-related muscle problems before. The study compares how people feel while taking atorvastatin with how they feel during placebo and no-treatment periods.

Atorvastatin

What is already known about the treatment

Atorvastatin
Atorvastatin is an oral medicine taken as a film-coated tablet, usually swallowed with water once daily. It is a well-known and widely used cholesterol-lowering drug in medical practice and is often discussed in medical literature for preventing heart and blood vessel disease, especially in people with high cholesterol such as familial hypercholesterolemia. It belongs to the statin group, and it works by blocking a liver enzyme called HMG-CoA reductase, which lowers the body’s production of cholesterol and helps remove LDL, the “bad” cholesterol, from the blood.
Encapsulated placebo tablets
Encapsulated placebo tablets are taken by mouth in the same way as the active medicine, but they do not contain any drug substance. They are used in clinical trials as a control treatment to help compare real drug effects with changes caused by expectations or chance, and they have no therapeutic use in routine medical care. A placebo does not act on the body’s disease process and does not have a molecular mechanism of action, but it helps researchers measure whether symptoms are truly caused by the medicine being tested or by other factors.

Investigated diseases

Familial hypercholesterolaemia - Familial hypercholesterolaemia is an inherited disorder that causes very high levels of cholesterol in the blood from an early age. It usually develops because the body cannot remove low-density lipoprotein cholesterol from the blood normally. Over time, cholesterol can build up in the arteries and form deposits in the skin or around the eyes. The condition is present for life and often becomes more noticeable as cholesterol-related changes slowly accumulate.