Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant
This study is investigating a new medication called acoramidis. It's for people who have a faulty gene that tends to cause a condition called transthyretin amyloidosis (ATTR). In ATTR, a protein known as TTR starts to fall apart and forms 'sticky plaques' in various body parts, including the heart and nerves, causing damage. Acoramidis is designed to keep this protein stable. The study aims to find out if taking acoramidis early, before any symptoms appear, can prevent or delay the heart and nerve problems associated with ATTR. This is a crucial step to see if early treatment can make a big difference in people's lives.
במבט חטוף
על מה המחקר הזה?
Imagine your body has tiny building blocks called proteins. One of these proteins, called TTR, sometimes breaks down and forms sticky clumps, a bit like glue. These clumps can then gather in different parts of your body, like your heart and nerves, causing them trouble. This condition is called transthyretin amyloidosis (ATTR).
Some people inherit a faulty gene that makes their TTR protein more likely to break down. This study is for these individuals who have this faulty gene but haven't started to feel unwell or show any signs of the disease yet. The study wants to see if a new medication, called acoramidis, can help. Acoramidis works by holding the TTR protein together, hoping to stop it from breaking down and forming these harmful clumps.
The main goal is to find out if taking acoramidis early can prevent or delay these problems from starting, especially those affecting the heart (which can lead to heart failure) or the nerves (which can cause nerve damage). If successful, this could mean that people with this faulty gene could start treatment before they get sick, potentially avoiding or delaying serious health issues.
נקודות עיקריות
- This study is for people with a genetic risk for ATTR, but no symptoms.
- It tests if a drug called acoramidis can prevent heart or nerve problems.
- Participants will receive either acoramidis or a placebo.
- Regular health checks, including heart scans, will be part of the study.
- Taking part helps research and might benefit your health.
מי עשוי להיות זכאי?
This study is looking for men and women between 18 and 75 years old. To join, you must have had a genetic test that shows you carry a specific faulty gene (called a TTR gene variant) that is known to cause transthyretin amyloidosis (ATTR). Importantly, you should not currently have any symptoms or signs of ATTR, either in your heart or nerves. Your age also needs to be roughly within 10 years (either younger or older) of the typical age when this condition usually starts in families with your specific faulty gene.
There are also some reasons why you might not be able to join. For example, if you are already taking other medicines that work in a similar way to acoramidis, or if you have certain other serious health conditions like significant kidney, liver, or heart disease. You also can't take part if you've recently had major surgery or are planning to have one soon, or if you have an allergy to acoramidis.
האם מחקר זה יכול להתאים לך?
ענה על השאלות המהירות הללו כדי לבדוק אם אתה עשוי להיות זכאי. זהו מדריך בלבד — צוות המחקר מקבל את ההחלטה הסופית.
- Am I aged between 18 and 75 years?
- Do I have a confirmed faulty TTR gene from a genetic test?
- Do I currently have no symptoms or signs of ATTR heart or nerve problems?
- Am I roughly within 10 years of the typical age of onset for ATTR in my family or genetic type?
- Am I not currently taking other similar ATTR-preventing medications?
- Do I not have any serious heart, kidney, or liver conditions?
מה כרוך בהשתתפות?
If you decide to take part in this study, you'll be given either the study drug, acoramidis, or a dummy pill (placebo) to take. You won't know which one you're receiving, and neither will your study doctor – this is done to make sure the results are fair. You'll have regular visits to the clinic where doctors will perform various health checks, including scans like heart MRI scans, blood tests, and nerve tests to see if the drug is working and to monitor your health. The study will involve ongoing treatment and check-ups over a period of time to see if acoramidis can prevent the disease from developing.
סיכונים ותועלות פוטנציאליים
Locations (104)
- University of California, San Diego (UCSD) - Medical Centerמיקוד מאומתלה הויה, ארצות הברית· גיוס
- University of California, Los Angeles (UCLA) - David Geffen School of Medicineמיקוד מאומתלוס אנג'לס, ארצות הברית· גיוס
- University of California, San Francisco (UCSF)מיקוד מאומתסן פרנסיסקו, ארצות הברית· גיוס
- אוניברסיטת סטנפורדמיקוד מאומתסטנפורד, ארצות הברית· גיוס
- University of Colorado Anschutzמיקוד מאומתאורורה, ארצות הברית· גיוס
- Yale University School of Medicine - Section of Cardiologyמיקוד מאומתניו הייבן, ארצות הברית· גיוס
- MedStar Washington Hospital Center - MedStar Heart and Vascular Instituteמיקוד מאומתוושינגטון די.סי., ארצות הברית· גיוס
- Mayo Clinic - Jacksonvilleמיקוד מאומתג'קסונוויל, ארצות הברית· גיוס
- University of Miamiמיקוד מאומתמיאמי, ארצות הברית· גיוס
- Cleveland Clinic Floridaמיקוד מאומתWeston, United States· גיוס
- בית הספר לרפואה של אוניברסיטת אמורימיקוד מאומתאטלנטה, ארצות הברית· גיוס
- John H. Stroger, Jr. Hospital of Cook Countyמיקוד מאומתשיקגו, ארצות הברית· גיוס
שאלות נפוצות
What is Transthyretin Amyloidosis (ATTR)?
It's a condition where a protein in your body breaks down and forms sticky clumps that can damage organs like the heart and nerves.
What does 'asymptomatic carrier' mean?
It means you have a faulty gene that could cause ATTR, but you don't have any symptoms or signs of the disease yet.
What is acoramidis?
It's an experimental drug designed to stop the protein from breaking down and causing those harmful clumps.
Will I know if I'm getting the real drug or a dummy pill?
No, this is a 'blinded' study, meaning neither you nor your doctor will know what you are receiving. This ensures the results are as accurate as possible.
כמה זמן אצטרך להיות במחקר?
The study involves ongoing treatment and check-ups over a period of time, which your doctor can explain in more detail.
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