CARDIO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
This study is looking into a new medicine called eplontersen for a serious heart condition known as ATTR amyloidosis (Transthyretin-Mediated Amyloid Cardiomyopathy). In this condition, abnormal proteins build up in the heart, making it stiff and hard to pump blood. The main goal of the study is to find out if eplontersen is safe and more effective than a look-alike dummy medicine (placebo) in improving the health of people living with ATTR amyloidosis. Participants will receive either eplontersen or the placebo as a simple injection under the skin every month, alongside their usual heart treatments. This is a large-scale study involving many people across different centres, so the results can give us a clear picture of how well eplontersen works.
At a glance
What is this study about?
This research study is about a heart condition called Transthyretin-Mediated Amyloid Cardiomyopathy, often shortened to ATTR amyloidosis. In this condition, a protein called transthyretin doesn't fold correctly and forms sticky clumps, called amyloid deposits, which build up in the heart. This buildup makes the heart muscle stiff and unable to pump blood as it should, leading to symptoms like shortness of breath and tiredness.
The study is testing a new medicine called eplontersen. Researchers want to compare eplontersen with a placebo, which looks exactly like the actual medicine but contains no active drug. This is to clearly see if eplontersen has a real effect on the heart and on people's health. All participants will also continue to receive the usual care recommended for their heart condition.
The study is designed to be 'double-blind,' meaning neither you nor your study doctor will know whether you are receiving eplontersen or the placebo. This helps make sure the results are fair and unbiased. The findings from this study will help doctors understand if eplontersen could be a new and effective treatment option for people with ATTR amyloidosis.
Key takeaways
- The study tests a new medicine, eplontersen, for ATTR amyloidosis affecting the heart.
- It compares eplontersen to a placebo (dummy medicine) to see how effective it is.
- Participants will receive monthly injections and continue their usual heart care.
- Neither you nor your doctor will know if you're getting the active drug or placebo.
- Regular clinic visits and tests will monitor your health.
- You can withdraw from the study at any time if you change your mind.
Who may be eligible?
To join this study, people generally need to be between 18 and 90 years old. Both men and women can take part. Women who could become pregnant and men whose partners could become pregnant must agree to use reliable birth control during the study.
A key requirement is a confirmed diagnosis of ATTR amyloidosis affecting the heart. This diagnosis needs to be clearly shown by special tests like biopsies (taking a small tissue sample) or specific heart scans. Your heart's main pumping chamber wall also needs to be a certain thickness, and your heart failure symptoms should fall within a specific range (mild to moderate).
There are also reasons why someone might not be able to join. For example, if you've had a recent heart attack, stroke, or major surgery within the last three months, you wouldn't be eligible. Also, if your heart problems are mainly due to other conditions like high blood pressure or valve disease, and not primarily ATTR amyloidosis, you would not be able 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 between 18 and 90 years old?
- Have you been diagnosed with ATTR amyloidosis affecting your heart?
- Do you have mild to moderate heart failure symptoms?
- Have you avoided major heart events or surgery in the last 3 months?
- Are you able to use reliable birth control if needed?
What does participation involve?
If you join this study, you'll be assigned by chance (like a coin toss) to receive either the study medicine, eplontersen, or a dummy medicine (placebo). Neither you nor your doctor will know which one you are getting. Both are given as injections under the skin, once every four weeks. You'll also take daily vitamin A supplements throughout the study.
Taking part will involve regular visits to the study clinic for check-ups, blood tests, and heart assessments to see how you're responding to the treatment. These visits will help the doctors monitor your health and the effects of the medicine. The total length of your participation in the study will be explained by the study team.
Potential risks and benefits
Locations (142)
- Mayo Clinic - ArizonaVerified postcodePhoenix, United States
- Altman Clinical and Translational Research Institute Center for Clinical ResearchVerified postcodeLa Jolla, United States
- Cedars-Sinai Medical CenterVerified postcodeLos Angeles, United States
- University of California, San Francisco (UCSF) - Medical CenterVerified postcodeSan Francisco, United States
- Stanford HospitalVerified postcodeStanford, United States
- University of Colorado Hospital - Anschutz Medical CampusVerified postcodeAurora, United States
- MedStar Washington Hospital CenterVerified postcodeWashington D.C., United States
- The George Washington Medical Faculty Associates - Foggy Bottom North PavilionVerified postcodeWashington D.C., United States
- Cleveland Clinic FloridaVerified postcodeWeston, United States
- Emory Heart and Vascular Center - Emory Clifton CampusVerified postcodeAtlanta, United States
- Piedmont Heart of FayettevilleVerified postcodeFayetteville, United States
- Northwestern UniversityVerified postcodeChicago, United States
Common questions
What is ATTR amyloidosis?
It's a condition where abnormal protein clumps (amyloid) build up in the heart, making it stiff and harder to pump blood.
What is eplontersen?
Eplontersen is a new medicine being tested to see if it can help stop or reduce the protein buildup in ATTR amyloidosis.
Will I know if I'm getting the real medicine or the dummy one?
No, neither you nor your doctor will know. This helps ensure the study results are fair and unbiased.
How is the medicine given?
It's given as a simple injection under the skin, once every four weeks.
Can I leave the study if I change my mind?
Yes, you can withdraw from the study at any time without it affecting your current medical care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
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