An Open-Label Study to Investigate the Safety, Tolerability, and Pharmacokinetics/Pharmacodynamics of Risdiplam (RO7034067) in Adult and Pediatric patients with Spinal Muscular Atrophy
This study is investigating a new medicine, Risdiplam, for individuals with Spinal Muscular Atrophy (SMA). We aim to understand its safety and if it causes any unwanted side effects when taken by adults and children. We will also carefully check how the medicine moves through the body and what effects it has. This is an early-stage study (Phase II) to gather important information about the treatment. Participants will be closely monitored for any changes in their health, and doctors will perform various checks including physical exams, eye tests, and neurological assessments. The main goal is to make sure the medicine is well-tolerated and to learn more about how it works.
De un vistazo
¿De qué trata este estudio?
This study is about a new potential medicine called Risdiplam (also known as RO7034067) for people who have Spinal Muscular Atrophy (SMA). SMA is a condition that affects nerves and muscles, making them weaker over time.
In this study, we want to learn more about how Risdiplam works in the body and if it's safe for people to take. We'll be looking carefully at any side effects people might experience, how well their body handles the medicine, and how much of the medicine is in their blood over time. This is an 'open-label' study, which means everyone involved, including the study doctors and participants, will know that the study medicine is being given.
This kind of study, called a Phase II trial, is an important step in developing new medicines. It helps us understand if the medicine has a good balance of showing helpful signs while also being safe enough to test in larger groups of people. Our main focus is always on the safety and well-being of the participants, so we'll be carrying out many different health checks throughout the study.
Puntos clave
- This study is testing a new SMA medicine called Risdiplam.
- It aims to find out if Risdiplam is safe and how it works in the body.
- Both adults and children with SMA can participate.
- Participants will have regular health checks and close monitoring.
- Your safety and well-being are the top priority throughout the study.
¿Quién puede ser elegible?
This study is looking for adults aged 18 years and older who have been diagnosed with Spinal Muscular Atrophy (SMA). Children with SMA can also take part.
Both men and women are welcome to join the study.
Important note: The study team will review your medical history and current health condition to decide if this study is suitable for you. This is to ensure your safety and that the study is a good fit for your individual circumstances.
¿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.
- Are you 18 years old or older, or are you a child with SMA?
- Have you been diagnosed with Spinal Muscular Atrophy (SMA)?
- Are you able to travel to study visits?
- Are you willing to have regular medical checks?
¿En qué consiste la participación?
If you decide to take part in this study, you will be given the study medicine, Risdiplam. You will have regular visits to the clinic where doctors and nurses will monitor your health very closely. These visits will include various assessments such as physical examinations, blood tests, heart checks (ECGs), eye tests, and neurological exams to check your nerve and muscle function. We will also ask about your general well-being and any symptoms. The study team will tell you the exact schedule of visits and assessments, and the total duration of your participation, which will include taking the medication and follow-up checks.
Riesgos y beneficios potenciales
Ubicaciones (5)
- —No verificadoPaíses Bajos
- —No verificadoItalia
- —No verificadoFrancia
- —No verificadoPolonia
- —No verificadoBélgica
Preguntas comunes
What is Spinal Muscular Atrophy (SMA)?
SMA is a genetic condition that causes muscle weakness and wasting over time, because it affects the nerves that control movement.
¿Qué significa 'etiqueta abierta'?
It means that everyone involved, including the patients and the study doctors, will know that you are receiving the study medicine, Risdiplam.
What is Risdiplam?
Risdiplam is the name of the new medicine being tested in this study for people with SMA.
¿Tendré que pagar para participar en el estudio?
No, you will not have to pay for the study medicine or the assessments related to the study.
Can children join this study?
Yes, both adults and children with SMA can take part in this study.
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.