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.
Överblick
Vad handlar denna studie om?
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.
Viktiga punkter
- 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.
Vem kan vara berättigad?
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.
Kan denna studie passa dig?
Svara på dessa snabba frågor för att se om du kan vara berättigad. Detta är endast en vägledning — forskarteamet fattar det slutgiltiga beslutet.
- Är du 18 år eller äldre?
- 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).
Vad innebär deltagande?
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.
Potentiella risker och fördelar
Locations (134)
- Mayo ClinicVerifierat postnummerScottsdale, USA
- Cedars-Sinai medicinska centrumVerifierat postnummerBeverly Hills, USA
- Altman Clinical and Translational Research Institute - Center for Clinical ResearchVerifierat postnummerLa Jolla, USA
- University Of California San Francisco Urology PracticeVerifierat postnummerSan Francisco, USA
- Stanford University (Leland Stanford Junior University)Verifierat postnummerStanford, USA
- University of Colorado Hospital - Anschutz Medical CampusVerifierat postnummerAurora, USA
- MedStar Washington Hospital CenterVerifierat postnummerWashington D.C., USA
- The George Washington Medical Faculty Associates - Foggy Bottom North PavilionVerifierat postnummerWashington D.C., USA
- Cleveland Clinic FloridaVerifierat postnummerWeston, USA
- Piedmont Heart of FayettevilleVerifierat postnummerAtlanta, USA
- Emory Heart and Vascular Center - Emory Clifton CampusVerifierat postnummerAtlanta, USA
- Piedmont Atlanta HospitalVerifierat postnummerFayetteville, United States
Vanliga frågor
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.
Varför sker denna studie?
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.
Hur länge kommer studien att pågå för mig?
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.
Hur du får reda på mer
Prata alltid med din läkare eller specialist innan du bestämmer dig för att delta i en studie.
Intresserad av att delta?
Diskussion
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