A Phase 3 Global, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of ION-682884 in Patients with Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)
Transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) is a rare condition where abnormal proteins build up in the heart, making it stiff and difficult to pump blood normally. This can lead to heart failure and other serious heart problems. This Phase 3 clinical trial is looking at a new medicine called ION-682884. Researchers want to see if regular injections under the skin can reduce serious heart issues, prevent hospital stays, and improve survival over about two and a half to three years. Participants will be placed by chance into one of two groups: one receives the study medicine and the other receives a dummy injection (placebo). Neither you nor your doctor will know which one you are receiving. The study involves regular check-ups to track your heart health and general wellbeing closely throughout the trial.
At a glance
What is this study about?
Transthyretin-mediated amyloid cardiomyopathy, often called ATTR-CM, is a condition that affects how the heart works. In people with ATTR-CM, a protein called transthyretin becomes unstable and clumps together. These clumps build up in the walls of the heart muscle, causing it to become thick and stiff. Over time, the heart cannot pump blood around the body as well as it should, which can cause breathlessness, fatigue, and heart failure.
This trial is testing an experimental medicine known as ION-682884. The medicine is designed to stop or reduce the buildup of these harmful protein clumps in the heart. Researchers hope that by tackling the root cause of the condition, the treatment will help the heart stay healthier for longer and ease symptoms.
Because this is a Phase 3 study, it is one of the final stages of testing before a new medicine can be approved for general use. The study compares ION-682884 against a placebo (an inactive dummy treatment) to see if the medicine really makes a difference to people's health, helps avoid emergency hospital visits, and helps people live longer, fuller lives.
Key takeaways
- This study tests a new injection (ION-682884) for people with ATTR-CM, a serious heart condition.
- The goal is to see if the treatment reduces hospital visits, improves heart health, and helps patients live longer.
- Participants have an equal chance of receiving either the active study medicine or an inactive placebo.
- The study lasts around 140 weeks (about 2.5 to 3 years) and involves regular hospital check-ups.
- Taking part is voluntary, and you can withdraw at any time without losing your standard care.
Who may be eligible?
This study is open to adults aged 18 and older who have been diagnosed with ATTR-CM. Both men and women can take part.
To be suitable, participants generally need to have confirmed signs of heart involvement from this condition, but still be well enough to attend regular clinic appointments and manage daily activities.
The study team will check your full medical history, run heart tests, and review any other health conditions or treatments you have before deciding if it is safe and appropriate for you to join.
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-CM (cardiac amyloidosis).
- I am able and willing to attend regular hospital appointments for around 2.5 to 3 years.
- I am comfortable receiving injections just under the skin.
- I am willing to accept that I might receive a placebo rather than the active medicine.
What does participation involve?
If you take part, you will be in the study for up to 140 weeks (around two and a half to three years). The study medicine or placebo is given as a small injection just under the skin (subcutaneous injection), similar to how insulin is given.
You will need to attend regular clinic visits at the hospital or trial centre. During these visits, doctors and nurses will check your heart, take blood and urine samples, perform heart scans or traces (such as ECGs), and ask how you are feeling. The study team will also closely monitor whether you need any extra hospital treatment or emergency heart care during the study period.
Potential risks and benefits
Locations (12)
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Common questions
What is a placebo?
A placebo is a dummy treatment that looks just like the real medicine but contains no active drug. It helps researchers see if the real medicine actually works better than no treatment at all.
Can I choose whether I get the real medicine or the placebo?
No. A computer will randomly decide which group you are in. Neither you nor your study doctor can choose, and neither of you will know which one you are having during the trial.
How is the medicine given?
It is given as an injection of a small amount of liquid just beneath the skin, usually in the tummy area or thigh.
Will taking part cost me anything?
No, the study medication and all trial tests are provided free of charge. Many trials also help cover travel expenses to and from your appointments.
What happens if I want to stop?
You can leave the trial at any time. Simply tell your study doctor. Your standard medical care will continue as normal.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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