An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)
This study aims to understand the long-term safety of a medication called eplontersen for adults who have a specific heart condition known as Transthyretin-Mediated Amyloid Cardiomyopathy, or ATTR-CM. This condition affects the heart. The study is an extension of an earlier trial and will follow participants who previously received eplontersen. Participants will receive regular doses of eplontersen along with vitamin A supplements. The main goal is to check for any side effects and to see how well people tolerate the treatment over an extended period. It will help doctors learn more about how to use this medicine safely and effectively for this heart condition.
एक नजर में
यह अध्ययन किस बारे में है?
This study is looking into a medicine called eplontersen for people who have a heart condition called Transthyretin-Mediated Amyloid Cardiomyopathy, or ATTR-CM. In simple terms, ATTR-CM is a condition where a specific protein called transthyretin builds up in the heart, making it stiff and harder for it to pump blood properly. This medicine, eplontersen, aims to help manage this condition.
This particular study is designed as an 'extension' study. This means it's for people who have already taken part in a previous medical study (called the 'Index Study') where they received eplontersen. The main reason for doing this extension study is to find out more about the long-term safety of eplontersen. While earlier studies provide important information about how a medicine works over a shorter time, extension studies help doctors understand how it affects people over several months or even years.
By following people for a longer period, doctors can see if any new side effects show up, or if existing ones change over time. This information is really important because it helps ensure that new treatments are not only effective but also safe for people to use for a long time. It helps make sure that when a medicine like eplontersen becomes widely available, doctors have a full picture of its benefits and any potential long-term considerations.
मुख्य निष्कर्ष
- This study is about the long-term safety of eplontersen for ATTR-CM.
- It's for adults who have already been in a related eplontersen study.
- Participants will receive eplontersen every four weeks, plus daily vitamin A.
- The study aims to monitor effects and side effects over a period of up to three years.
- It's important for understanding how the medicine works safely over a longer time.
कौन पात्र हो सकता है?
To join this study, you must be an adult, aged 18 years or older, and have already successfully completed a previous study involving eplontersen (either the ION-682884-CS2 study or the ISIS 420915-CS101 study). Your doctor must also agree that this study is suitable for you and be willing to treat you with eplontersen as part of the study.
You also need to be willing to take vitamin A supplements every day, as instructed by the study team. Both your doctor and the study organisers need to agree that you are a good fit for the study and that participating won't be harmful to your health.
You cannot join this study if you stopped taking the study medication permanently during the previous study you were in. Also, if you have any new health problems, or if an existing condition has gotten worse, and your doctor thinks this would make the study unsuitable or difficult for you, then you wouldn't be able to take part.
क्या यह अध्ययन आपके लिए उपयुक्त हो सकता है?
यह देखने के लिए इन त्वरित प्रश्नों का उत्तर दें कि क्या आप पात्र हो सकते हैं। यह केवल एक मार्गदर्शिका है — अंतिम निर्णय शोध टीम द्वारा लिया जाता है।.
- क्या आप 18 वर्ष या उससे अधिक आयु के हैं?
- Have you completed one of the previous eplontersen studies (ION-682884-CS2 or ISIS 420915-CS101)?
- Are you willing to take daily vitamin A supplements?
- Are you willing to have regular check-ups with the study team?
- Did you permanently stop the study drug in your previous trial? (If yes, you might not be eligible).
भागीदारी में क्या शामिल है?
If you join this study, you will receive doses of eplontersen once every four weeks. You will also need to take recommended daily doses of vitamin A supplements. This treatment will continue for up to three years (36 months), or until eplontersen becomes available as a standard treatment in your country, whichever happens first.
Throughout the study, you will have regular visits with the study team. These visits will involve various tests and checks to monitor your health and how you are responding to the medication. These might include physical examinations, blood tests, and other assessments to track the safety and effects of eplontersen. The study team will tell you exactly how often these visits will be and what they will involve. They will also let you know about any follow-up appointments after you finish taking the medication.
संभावित जोखिम और लाभ
Locations (134)
- मेयो क्लिनिकसत्यापित पोस्टकोडस्कॉट्सडेल, संयुक्त राज्य अमेरिका
- Cedars-Sinai Medical Centerसत्यापित पोस्टकोडबेवर्ली हिल्स, संयुक्त राज्य अमेरिका
- Altman Clinical and Translational Research Institute - Center for Clinical Researchसत्यापित पोस्टकोडला जोला, संयुक्त राज्य अमेरिका
- University Of California San Francisco Urology Practiceसत्यापित पोस्टकोडसैन फ्रांसिस्को, संयुक्त राज्य अमेरिका
- Stanford University (Leland Stanford Junior University)सत्यापित पोस्टकोडस्टैनफोर्ड, संयुक्त राज्य अमेरिका
- University of Colorado Hospital - Anschutz Medical Campusसत्यापित पोस्टकोडऑरोरा, संयुक्त राज्य अमेरिका
- MedStar Washington Hospital Centerसत्यापित पोस्टकोडवॉशिंगटन डी.सी., संयुक्त राज्य अमेरिका
- The George Washington Medical Faculty Associates - Foggy Bottom North Pavilionसत्यापित पोस्टकोडवॉशिंगटन डी.सी., संयुक्त राज्य अमेरिका
- क्लीवलैंड क्लिनिक फ्लोरिडासत्यापित पोस्टकोडवेस्टन, संयुक्त राज्य अमेरिका
- Piedmont Heart of Fayettevilleसत्यापित पोस्टकोडअटलांटा, संयुक्त राज्य अमेरिका
- Emory Heart and Vascular Center - Emory Clifton Campusसत्यापित पोस्टकोडअटलांटा, संयुक्त राज्य अमेरिका
- Piedmont Atlanta Hospitalसत्यापित पोस्टकोडफेयेटविले, संयुक्त राज्य अमेरिका
सामान्य प्रश्न
What is ATTR-CM?
ATTR-CM is a heart condition where a protein builds up in the heart, making it stiff and weaker over time.
What is eplontersen?
Eplontersen is the name of the medicine being studied, which aims to help manage ATTR-CM.
यह अध्ययन क्यों हो रहा है?
This study is an extension of previous research, designed to look at the long-term safety of eplontersen over a longer period in people who have already taken it.
Will I get vitamin A?
Yes, if you join the study, you will also receive daily vitamin A supplements as part of the treatment plan.
मेरे लिए अध्ययन कितने समय तक चलेगा?
You could be in the study for up to three years (36 months), or until the medicine becomes widely available in your country, whichever happens first.
और अधिक जानने के लिए कैसे पता करें
किसी अध्ययन में भाग लेने का निर्णय लेने से पहले हमेशा अपने जीपी या विशेषज्ञ से बात करें।.
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