Within-Patient Comparison of Serum Transthyretin Response to Tafamidis Versus Acoramidis: A Pilot Crossover Study in Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
Transthyretin amyloid cardiomyopathy (ATTR-CM) is a rare condition where proteins fold incorrectly and build up in the heart muscle. Over time, this makes the heart stiff and less able to pump blood efficiently. Two medicines, tafamidis (Vyndaqel) and acoramidis (BEYONTTRA), work by stabilising these proteins in the blood to stop them building up. This study is a 'crossover' trial, which means each person will take one medicine for four weeks, and at another time take the other medicine. Doctors will take blood tests before and after each four-week treatment period. By measuring the level of protein in your blood, the study will help doctors see whether one drug works better than the other in the same person. The goal is to gather clear information that can help doctors choose the best options for people with this condition in the future.
At a glance
What is this study about?
Transthyretin amyloid cardiomyopathy (often shortened to ATTR-CM) is a serious heart condition. It happens when a natural protein in the blood called transthyretin becomes unstable and breaks apart. These pieces build up inside the heart muscle, making the heart walls thick and stiff. As a result, the heart has to work much harder to pump blood around the body, which can lead to breathlessness, tiredness, and swelling in the legs.
Researchers have developed medicines called 'stabilisers' to treat this. These medicines attach to the protein to stop it falling apart and settling in the heart. Two such treatments are tafamidis (brand name Vyndaqel) and acoramidis (brand name BEYONTTRA). While both medicines aim to do the same job, doctors want to know if one is better at stabilising the protein than the other.
This study uses what is called a 'crossover' design. Instead of comparing two different groups of people, each person will test both medicines at different times. You will take one medicine for four weeks, have routine blood tests, and then take the other medicine for four weeks. This allows researchers to compare how your own body responds to each drug.
By comparing the results within the same patients, doctors can get very clear and reliable evidence. This will help doctors across the UK and worldwide make more informed decisions about which treatment might be best suited to each individual patient.
Key takeaways
- The study compares two medicines (tafamidis and acoramidis) for ATTR amyloid cardiomyopathy.
- It is open to adults aged 18 and over who have this heart condition.
- Participants will try each medicine for four weeks to see how their blood protein levels change.
- Blood samples will be taken before and after each four-week treatment block.
- Taking part is completely voluntary and will not affect your standard NHS medical care.
Who may be eligible?
This study is open to adults aged 18 and older who have been diagnosed with transthyretin amyloid cardiomyopathy (ATTR-CM). Both men and women can take part.
Because this study compares two specific heart medicines, you must be well enough to take daily tablets or capsules by mouth and be able to attend the required hospital or clinic appointments for check-ups and blood tests.
Your cardiology doctor or study team will review your full medical history, your current heart medicines, and your general health to ensure it is safe and suitable for you to take part.
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 aged 18 or older.
- I have been diagnosed with ATTR amyloid cardiomyopathy (ATTR-CM).
- I am able to take daily tablets and capsules by mouth.
- I can attend regular clinic appointments for check-ups and routine blood tests.
- I am willing to follow the study schedule and try both medicines.
What does participation involve?
If you take part in this study, you will be asked to attend clinic visits at a specialist heart centre. You will take one study medicine (either Vyndaqel capsules or BEYONTTRA tablets) every day for about four weeks. After this, you will switch to the other medicine for another four-week period.
At the start and end of each four-week period, the medical team will carry out standard health checks and take blood samples. These blood tests measure the amount of stabilised protein in your bloodstream. You may also be asked about any symptoms or side effects you notice. The study usually lasts for a few months in total to allow time for both treatment stages and any required break in between.
Potential risks and benefits
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Common questions
What is a crossover study?
A crossover study means you will receive both treatments at different times. Instead of comparing you to someone else, researchers compare how your own body responds to each medicine.
Will taking part cure my heart condition?
No. These medicines do not cure ATTR-CM, but they are designed to stabilise proteins and help slow down the progression of the condition.
Do I have to pay to take part?
No, clinical trials run through hospital clinics are free to join, and the study medications are provided at no cost to you.
Can I leave the study if I change my mind?
Yes, you can leave the study at any time without giving a reason, and your regular doctor will continue your standard care as normal.
How often will I need blood tests?
You will have blood tests at the start and end of each four-week treatment period to measure changes in your blood protein levels.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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