Phase III Trial of AMX0035 for Amyotrophic Lateral Sclerosis Treatment
The Phoenix Trial is a research study looking at a new treatment called AMX0035 for Amyotrophic Lateral Sclerosis (ALS), or Motor Neurone Disease. We are trying to find out if AMX0035 is safe and can help to slow down how the disease progresses. Some people will get the active medicine, AMX0035, while others will get a dummy medicine (placebo) – neither you nor your doctor will know which one you are receiving until the trial ends. The study will last for 48 weeks and we'll check how your physical abilities change, if it helps you live longer, and your general quality of life. This is a very important step to find new ways to manage ALS.
Em resumo
Sobre o que é este estudo?
Amyotrophic Lateral Sclerosis (ALS), often called Motor Neurone Disease, is a condition that affects nerve cells in the brain and spinal cord, leading to muscle weakness and wasting. There's currently no cure, but researchers are working hard to find new treatments that can slow its progression. This particular study, called the Phoenix Trial, is focusing on a medicine named AMX0035. Researchers believe AMX0035 might work by protecting these important nerve cells from damage, which could help to maintain muscle function for longer.
To find out if AMX0035 really works and is safe, we compare it to a 'dummy' medicine, called a placebo. This means that some participants will receive AMX0035, and others will receive the placebo. Neither you nor your study doctor will know which one you are taking until the study is over. This is a standard way to test new medicines, ensuring that any improvements or side effects we see are truly due to the medicine being tested, and not just what we expect to happen.
Taking part in a study like this is a big decision and will involve regular check-ups over 48 weeks. We'll be carefully monitoring your health, how your condition progresses, and your overall well-being. The goal is to see if AMX0035 can slow down the physical changes caused by ALS and potentially improve quality of life and survival for people living with this condition.
Pontos principais
- This study tests a new medicine, AMX0035, for ALS (Motor Neurone Disease).
- It aims to see if AMX0035 is safe and can slow down the disease over 48 weeks.
- Participants will receive either AMX0035 or a dummy medicine (placebo).
- Regular clinic visits and health checks are part of taking part.
- The study may help find new ways to manage ALS.
Quem pode ser elegível?
To be considered for this study, you need to be at least 18 years old and have a definite or very likely diagnosis of ALS. It's important that your ALS symptoms started less than 24 months ago. If you're already taking other ALS medications like riluzole or edaravone, these should have been at a steady dose for a set period before you join.
There are also some reasons why you might not be able to join. For example, if you need a breathing machine (tracheostomy or permanent assisted ventilation) or if your breathing capacity is very low (less than 55% of what's expected). You also can't have certain allergies, severe liver or kidney problems, or currently be pregnant or breastfeeding. The research team will do medical checks to make sure it's safe for you to take part.
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?
- Was your ALS diagnosed less than 2 years ago?
- Você consegue comparecer a consultas regulares na clínica?
- Do you not need permanent breathing support?
- Do you not have severe liver or kidney problems?
O que envolve a participação?
If you decide to take part, you'll be assigned either to receive the study drug (AMX0035) or a dummy drug (placebo). You won't know which one you're getting. You'll need to attend regular clinic visits over a period of 48 weeks. During these visits, the study team will carry out various checks, including assessing your physical abilities, breathing function, and taking blood samples. You'll also be asked about your quality of life. You'll continue taking the study medication for the full 48 weeks. After this, there might be some follow-up checks. Women who can become pregnant and men will need to use effective birth control during the study and for three months after their last dose.
Riscos e benefícios potenciais
Locations (69)
- Barrow Neurological InstituteCódigo postal verificadoPhoenix, Estados Unidos
- Universidade do Sul da CalifórniaCódigo postal verificadoLos Angeles, Estados Unidos
- University of California IrvineCódigo postal verificadoOrange, Estados Unidos
- California Pacific Medical Center Research InstituteCódigo postal verificadoSão Francisco, Estados Unidos
- Universidade do ColoradoCódigo postal verificadoAurora, Estados Unidos
- Universidade da FlóridaCódigo postal verificadoGainesville, Estados Unidos
- University of South FloridaCódigo postal verificadoTampa, Estados Unidos
- Universidade EmoryCódigo postal verificadoAtlanta, Estados Unidos
- Augusta University Neuroscience CenterCódigo postal verificadoAugusta, Estados Unidos
- Northwestern UniversityCódigo postal verificadoChicago, Estados Unidos
- Johns Hopkins University School of Medicine Outpatient CenterCódigo postal verificadoBaltimore, Estados Unidos
- Healey & AMG Center for ALS Research at Massachusetts General HospitalCódigo postal verificadoBoston, Estados Unidos
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 a 'Phase III' trial?
A Phase III trial is usually the last step before a new medicine might be approved. It involves more people and aims to confirm the medicine's safety and effectiveness.
O que é um 'placebo'?
A placebo is a 'dummy' medicine that looks like the real thing but contains no active drug. It helps researchers compare the effects of the actual medicine fairly.
Quanto tempo estarei no estudo?
The main treatment period for this study is 48 weeks, which is about 11 months. There may be some follow-up checks after this.
Will I know if I'm getting the real medicine?
No, you and your doctor won't know if you're receiving AMX0035 or the placebo until the trial ends. This helps ensure the study results are unbiased.
Como saber mais
Sempre consulte seu médico ou especialista antes de decidir participar de um estudo.
Discussão
Discussão da comunidade
Desenvolvido pelo nosso fórum em community.patient.info. Por favor, seja respeitoso — isto não é um conselho médico.