A Phase 2b/3 Study of Safety and Efficacy of AMX0035 in Progressive Supranuclear Palsy (ORION)
The ORION study is a major clinical trial looking into a new treatment, AMX0035, for people living with Progressive Supranuclear Palsy (PSP). PSP is a rare brain condition that gradually affects movement, balance, speech, and swallowing. This study is in its final stages (Phase 3), meaning it's designed to confirm whether AMX0035 is both safe and effective at treating PSP. Participants will either receive the active drug or a placebo (a dummy drug). Researchers will closely monitor changes in PSP symptoms over 52 weeks to understand if AMX0035 can help slow the disease's progression and improve daily life. They will also carefully track any side effects to ensure the treatment is well-tolerated.
Em resumo
Sobre o que é este estudo?
This study, known as ORION, is looking for people with a condition called Progressive Supranuclear Palsy (PSP) to take part. PSP is a serious and complex condition that affects the brain, causing problems with movement, balance, speech, and eyesight. Unfortunately, there isn't a cure for PSP yet, and treatments mainly focus on managing symptoms. This research aims to find out if a new drug called AMX0035 can make a real difference for those living with PSP.
The main goal of the study is to see if AMX0035 can slow down how quickly PSP symptoms get worse over 52 weeks (about a year). To do this, some participants will receive AMX0035, while others will receive a placebo – a substance that looks exactly like the drug but contains no active medicine. This is a common way to test new drugs reliably. Neither the participant nor their doctor will know who is getting the active drug and who is getting the placebo until the study ends. Researchers will carefully measure how PSP symptoms change using specific rating scales.
Another very important part of the study is to check for safety. The research team will be keeping a close eye on any side effects or unexpected health problems that participants might experience. This helps them understand if AMX0035 is not only effective but also safe for people with PSP. If this study shows that AMX0035 is safe and helps people with PSP, it could potentially become a new treatment option in the future, offering hope for many.
Pontos principais
- The ORION study is testing a new drug (AMX0035) for Progressive Supranuclear Palsy (PSP).
- It aims to see if AMX0035 is safe and can slow down PSP symptoms over one year.
- Participants will receive either the active drug or a dummy pill (placebo).
- Both men and women, aged 18 and over, with PSP may be eligible.
- Sua saúde será monitorada de perto durante todo o estudo.
Quem pode ser elegível?
To be able to take part in this study, participants should be aged 18 or older – there's no upper age limit. Both men and women are welcome to join.
Beyond age and sex, there will be other specific health requirements that the study team will need to check. For example, they'll need to confirm that you have a clear diagnosis of Progressive Supranuclear Palsy. They will also look at your overall health to make sure that taking part in the study would be safe for you.
It's important to remember that these are just general guidelines. The study doctors and nurses will need to do a full medical check-up to decide if this particular study is right for you. They will explain all the details and answer any questions you have.
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?
- Have you been diagnosed with Progressive Supranuclear Palsy (PSP)?
- Are you able to attend regular clinic visits for about a year?
- Are you willing to potentially receive a placebo instead of the active drug?
O que envolve a participação?
If you decide to take part in the ORION study, you would receive either the study drug AMX0035 or a placebo (a dummy drug) over 52 weeks, which is about a year. You wouldn't know which one you are receiving. During this time, you would have regular visits to the study clinic. At these visits, doctors and nurses will assess your PSP symptoms using special questionnaires and examinations. They will also monitor your general health and check for any side effects. You might also have blood tests or other checks as needed. After the 52 weeks are up, there would likely be a follow-up period to see how you are doing. The exact number and timing of visits would be explained in detail by the study team.
Riscos e benefícios potenciais
Locations (10)
- —Não verificadoBulgária
- —Não verificadoÁustria
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- —Não verificadoSuécia
- —Não verificadoBélgica
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- —Não verificadoPolônia
- —Não verificadoFrança
- —Não verificadoAlemanha
- —Não verificadoItália
Perguntas frequentes
What is Progressive Supranuclear Palsy (PSP)?
PSP is a rare brain condition that causes problems with movement, balance, speech, and eye movements. It gets worse over time.
What is AMX0035?
AMX0035 is a new drug being tested to see if it can help slow down the progression of PSP symptoms.
O que é um placebo?
A placebo is a 'dummy' treatment that looks like the real drug but contains no active medicine. It helps researchers fairly test if the real drug works.
Quanto tempo dura o estudo?
The main treatment period for participants in this study is 52 weeks, which is about one year.
Will I know if I'm getting the drug or the placebo?
No, during the study, neither you nor your doctor will know if you are receiving the active drug or the placebo. This helps make the study results more reliable.
Como saber mais
Sempre consulte seu médico ou especialista antes de decidir participar de um estudo.
Interessado em participar?
Discussão
Discussão da comunidade
Desenvolvido pelo nosso fórum em community.patient.info. Por favor, seja respeitoso — isto não é um conselho médico.