MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)
This study, called MAGNITUDE, is looking into a new treatment called NTLA-2001 for a specific heart condition called transthyretin amyloidosis with cardiomyopathy (ATTR-CM). This condition occurs when abnormal proteins build up in the heart, making it harder for the heart to pump blood. The study aims to see if a single dose of NTLA-2001 can help people with ATTR-CM, and if it's safe to use. It compares NTLA-2001 to a dummy drug, called a placebo, in a large group of people. The goal is to find out if this new treatment could be a safe and effective option for those living with ATTR-CM.
Em resumo
Sobre o que é este estudo?
This study, known as MAGNITUDE, is investigating a new medication called NTLA-2001 for people who have a heart condition called transthyretin amyloidosis with cardiomyopathy, often shortened to ATTR-CM. This condition happens when a faulty protein builds up in the heart muscle, making it stiff and less able to pump blood efficiently. This can lead to symptoms like breathlessness and swelling, which is commonly referred to as heart failure.
The main purpose of this study is to find out whether a single treatment of NTLA-2001 is both safe and effective in improving the health of people with ATTR-CM. To do this, the study will compare NTLA-2001 with a 'placebo', which is an inactive substance that looks just like the real treatment but has no medicine in it. This comparison helps researchers determine if any changes seen are truly due to the new medication or other factors.
About 1200 people will take part in this study across different countries. Participants will be randomly assigned to receive either the NTLA-2001 infusion or the placebo. Neither you nor your study doctor will know which treatment you are receiving, which is a common practice in research to ensure the results are as unbiased as possible. This careful approach is designed to give us clear answers about the potential benefits and risks of NTLA-2001 for ATTR-CM.
Pontos principais
- This study is testing a new treatment (NTLA-2001) for a heart condition called ATTR-CM.
- It aims to see if a single dose of NTLA-2001 is safe and effective compared to a dummy drug (placebo).
- About 1200 people with ATTR-CM will participate globally.
- Participants will receive either NTLA-2001 or placebo as a single infusion.
- Regular health check-ups and monitoring will be part of the study.
- You can discuss the risks and potential benefits with the study team and can withdraw at any time.
Quem pode ser elegível?
To join this study, you would need to have been diagnosed with transthyretin amyloidosis affecting your heart, and have a history of heart failure. Your heart failure symptoms should be stable and well-managed in the month before starting the study, and a specific blood test for heart failure (NT-proBNP) must fall within a certain range.
There are also some reasons why you might not be able to join. For example, if your heart failure is very severe (classed as New York Heart Association Class IV), or if you are confined to a wheelchair or bed due to nerve damage. You also can't participate if you have certain infections like hepatitis B, hepatitis C, or HIV, or a recent history of cancer.
Additionally, if you've recently used certain other medications for amyloidosis within the last year, or if you have uncontrolled high blood pressure, severe kidney problems, or certain liver diseases, you would not be able to take part. You also need to be willing and able to take vitamin A supplements throughout the study.
Este estudo pode ser adequado para você?
Responda a estas perguntas rápidas para ver se você pode ser elegível. Este é apenas um guia — a decisão final é da equipe de pesquisa.
- Do you have a confirmed diagnosis of ATTR amyloidosis affecting your heart?
- Have you been diagnosed with heart failure?
- Are your heart failure symptoms currently stable and well-managed?
- Are you between 18 and 90 years old?
- Do you have any severe liver or kidney problems?
- Have you recently used other specific amyloidosis medications?
O que envolve a participação?
If you decide to participate in this study, you will receive either the study drug NTLA-2001 or a placebo (a dummy drug) as a single infusion. This will be given in hospital. For the duration of the study, you will have regular check-ups, which include blood tests, heart assessments, and discussions about your health and any side effects you might experience. You will also need to take vitamin A supplements throughout the study. The total duration of your participation and the exact schedule of visits will be explained in detail by the study team, but it will involve ongoing monitoring after your single treatment.
Riscos e benefícios potenciais
Locations (132)
- Universidade do ArizonaCódigo postal verificadoTucson, Estados Unidos· Recrutamento
- Cedars-Sinai Medical CenterCódigo postal verificadoLos Angeles, Estados Unidos· Recrutamento
- Western Greater Los Angeles VA Medical CenterCódigo postal verificadoLos Angeles, Estados Unidos· Recrutamento
- Universidade de StanfordCódigo postal verificadoPalo Alto, Estados Unidos· Recrutamento
- University of California, San Diego (UCSD) - Medical CenterCódigo postal verificadoSan Diego, Estados Unidos· Recrutamento
- University of Colorado DenverCódigo postal verificadoDenver, Estados Unidos· Recrutamento
- Yale University School of MedicineCódigo postal verificadoNew Haven, Estados Unidos· Recrutamento
- MedStar Washington Hospital Center - MedStar Heart and Vascular InstituteCódigo postal verificadoWashington D.C., Estados Unidos· Recrutamento
- Mayo Clinic - JacksonvilleCódigo postal verificadoJacksonville, Estados Unidos· Recrutamento
- Cleveland Clinic of FloridaCódigo postal verificadoWeston, United States· Recrutamento
- Emory University School of MedicineCódigo postal verificadoAtlanta, Estados Unidos· Recrutamento
- Northwestern UniversityCódigo postal verificadoChicago, Estados Unidos· Recrutamento
Perguntas frequentes
What is ATTR-CM?
ATTR-CM is a heart condition where abnormal proteins build up in the heart, making it stiff and affecting its ability to pump blood effectively.
O que é um placebo?
A placebo is an inactive substance that looks exactly like the study medication but does not contain any medicine. It helps researchers objectively compare the effects of the new treatment.
Saberei se estou recebendo o medicamento ativo ou o placebo?
No, this is a 'blind' study, meaning neither you nor your study doctor will know whether you are receiving NTLA-2001 or the placebo. This helps ensure fair and unbiased results.
What does a single infusion mean?
It means the treatment (either NTLA-2001 or placebo) will be given to you just once, typically through a drip into your arm, rather than as a daily tablet or repeated injections.
Posso sair do estudo a qualquer momento?
Yes, your participation is voluntary, and you are free to withdraw from the study at any point, for any reason, without it affecting your medical care.
Como saber mais
Trial Manager at Intellia
Sempre consulte seu médico ou especialista antes de decidir participar de um estudo.
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