A Phase 3, Randomized, Double-blind, Placebo-controlled Multicenter Study to Evaluate the Efficacy and Safety of Patisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy (ATTR Amyloidosis with Cardiomyopathy)
This research study is looking into a new treatment called Onpattro for people who have a heart condition known as ATTR amyloidosis with cardiomyopathy. This condition affects your heart's ability to pump blood properly. The study aims to see how well Onpattro works and if it's safe. Participants will be given either the study medicine or a dummy treatment (placebo) without knowing which one they receive. Researchers will check changes in walking ability and other heart health measures over 12 months. This type of study helps doctors understand if new medicines can effectively treat serious conditions and improve patients' lives.
De un vistazo
¿De qué trata este estudio?
This study is about a heart condition called ATTR amyloidosis with cardiomyopathy. In this condition, abnormal proteins build up in your heart, making it stiff and harder for it to pump blood around your body. This can lead to symptoms like shortness of breath and tiredness.
The main goal of this study is to see if a medication called Onpattro can help people with this heart condition. Researchers want to find out if Onpattro can improve your ability to walk and generally make you feel better and healthier compared to people who don't receive the active medicine. They will also be carefully checking for any side effects.
This is a "Phase 3" study, which means the medicine has already been tested in smaller groups and is now being looked at in a larger group of people to confirm its benefits and safety. By comparing Onpattro to a 'dummy' treatment (a placebo), doctors can get a clear picture of how effective the new medicine truly is. This information is crucial for deciding if Onpattro could become a new standard treatment for ATTR amyloidosis.
Puntos clave
- This study investigates Onpattro for a serious heart condition.
- It aims to improve walking ability and heart health.
- Participants receive either Onpattro or a dummy treatment.
- The study lasts about 12 months with ongoing follow-up.
- Regular health checks and walking tests are part of participation.
- It's a Phase 3 study, looking to confirm benefits and safety.
¿Quién puede ser elegible?
This study is looking for adults aged 18 years and older. Both men and women can take part.
To be considered for the study, you must have been diagnosed with Transthyretin Amyloidosis with Cardiomyopathy. There will be other specific medical checks to make sure the study is right for you and safe for you to take part in.
¿Podría este estudio ser adecuado para ti?
Responde estas preguntas rápidas para ver si puedes ser elegible. Esto es solo una guía: el equipo de investigación toma la decisión final.
- ¿Tengo 18 años o más?
- Have I been diagnosed with Transthyretin Amyloidosis with Cardiomyopathy?
- Am I able to attend regular hospital visits for assessments?
- Am I comfortable completing walking tests and health questionnaires?
¿En qué consiste la participación?
If you decide to take part, you will be randomly assigned to receive either the study medicine, Onpattro, or a dummy treatment (placebo). Neither you nor your doctor will know which one you are receiving – this is standard practice in these types of studies and helps us get accurate results. Both Onpattro and the dummy treatment will be given through a drip (infusion) into your vein.
You will have regular hospital visits for medical checks, blood tests, and to assess your heart health and how well you can walk. These assessments typically involve a "6-minute walk test" where you walk as far as you can in six minutes. You will also fill out questionnaires about your quality of life and symptoms.
These visits will happen over about 12 months. After this initial period, there will be ongoing follow-up to monitor your health. The medical team will explain the exact schedule of visits and what each will involve if you are interested.
Riesgos y beneficios potenciales
Locations (8)
- —No verificadoDinamarca
- —No verificadoBélgica
- —No verificadoItalia
- —No verificadoSuecia
- —No verificadoChequia
- —No verificadoPaíses Bajos
- —No verificadoFrancia
- —No verificadoPortugal
Preguntas comunes
What is ATTR amyloidosis with cardiomyopathy?
It's a heart condition where abnormal proteins build up in your heart, making it less effective at pumping blood.
What is Onpattro?
Onpattro is the new experimental medicine being tested in this study for ATTR amyloidosis with cardiomyopathy.
Will I know if I'm getting the real medicine or a dummy treatment?
No, neither you nor your doctor will know. This is a standard way to ensure reliable study results.
¿Cuánto tiempo estaré en el estudio?
The main part of the study lasts about 12 months, with follow-up beyond that.
¿Puedo abandonar el estudio en cualquier momento?
Yes, you can choose to leave the study at any point, and your regular medical care will not be affected.
Cómo obtener más información
Siempre hable con su médico de cabecera o especialista antes de decidir participar en un estudio.
¿Interesado en participar?
Discusión
Discusión comunitaria
Impulsado por nuestro foro en community.patient.info. Por favor, sea respetuoso — esto no es un consejo médico.