A Phase 1b/2 Study to Evaluate NB-4746 in Participants with Amyotrophic Lateral Sclerosis.
This research is investigating a new medication, NB-4746, for individuals living with Motor Neurone Disease, also known as Amyotrophic Lateral Sclerosis (ALS). The study is divided into two main parts. The first part, Phase 1b, focuses on understanding the safety of NB-4746 and identifying any immediate side effects. The second part, Phase 2, continues to monitor safety and also looks at how the medicine affects general health, using tests like blood work and heart scans. There's also an optional longer-term part of the study where participants would continue to receive the treatment and be monitored. The main goal is to find out if NB-4746 can be a safe and helpful new treatment option for ALS.
Em resumo
Sobre o que é este estudo?
This study is testing a new medicine called NB-4746 for people who have Motor Neurone Disease, which doctors often call Amyotrophic Lateral Sclerosis or ALS. ALS is a condition that affects special nerve cells in your brain and spinal cord, making your muscles weaker over time.
The main idea behind this study is to see if NB-4746 can be a safe and effective way to help manage ALS. Because this is a new medicine, the study is split into stages. The initial stage focuses on safety – checking for any side effects and how the body handles the medicine.
Later stages will continue to look at safety but also start to investigate if the medicine has any positive effects on the condition itself. There’s even an option for participants to continue receiving the treatment for a longer period, which helps researchers understand its long-term safety and effects.
Pontos principais
- Tests a new medicine (NB-4746) for Motor Neurone Disease (ALS).
- Focuses on safety and side effects, then checks if it helps the condition.
- Includes regular health checks like blood tests and heart scans.
- Participation involves clinic visits and taking study medication or a placebo.
- Open to adults aged 18 and older with ALS.
- Você pode parar de participar a qualquer momento.
Quem pode ser elegível?
To join this study, participants must be at least 18 years old. Both men and women are welcome to take part.
You would need to have a confirmed diagnosis of Amyotrophic Lateral Sclerosis (ALS), also known as Motor Neurone Disease. The research team will review your medical history and current health to make sure the study is a good fit for you.
It’s important that you are able to attend all the required study visits and follow the study instructions. The medical team will check other specific health requirements to ensure your safety 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.
- Você tem 18 anos ou mais?
- Do you have a diagnosis of Motor Neurone Disease (ALS)?
- Você consegue comparecer a consultas regulares na clínica?
- Are you willing to take new study medication or a placebo?
- Are you currently taking Riluzole (your regular ALS medicine)?
O que envolve a participação?
Taking part in this study would involve several visits to a clinic over a period of time. You would receive either the new medicine, NB-4746, or a dummy pill (placebo) that looks exactly the same, but has no active medicine. Some parts of the study also involve taking your usual medicine for ALS, called Riluzole.
During your visits, you would have checks like blood tests, urine tests, heart scans (ECGs), and your vital signs (like blood pressure and heart rate) would be measured. These checks help the doctors monitor your health and how you're responding to the treatment. The total length of your participation would depend on which part of the study you join, but it could involve an initial treatment period followed by an optional longer-term follow-up.
Riscos e benefícios potenciais
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Perguntas frequentes
What is ALS?
ALS stands for Amyotrophic Lateral Sclerosis, also known as Motor Neurone Disease. It's a condition that affects nerve cells, leading to muscle weakness over time.
What is NB-4746?
NB-4746 is a new medicine being tested in this study for people with ALS. We are trying to find out if it is safe and if it can help with the condition.
O que é um 'placebo'?
A placebo is a dummy pill that looks like the real medicine but contains no active ingredients. It helps researchers understand if the study medicine is truly working.
Quanto tempo durará o estudo?
The study has different parts, so the exact length of your participation would be discussed by the study team. Some parts are shorter, while others include an optional longer-term follow-up.
Will I still take my regular ALS medicine?
Yes, some parts of this study involve taking the usual ALS medicine, Riluzole, alongside the study treatment. The study team will explain all the details.
Como saber mais
Sempre consulte seu médico ou especialista antes de decidir participar de um estudo.
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