Phase 1/2 Investigation Of Novel Experimental Regimen in Amyotrophic Lateral Sclerosis (PIONEER-ALS): An Open-Label, Uncontrolled, Multicenter Study to Assess the Safety and Tolerability of Two Doses of VTx-002.
This is an early-stage clinical trial (Phase 1/2) for people with Motor Neurone Disease (ALS). The study is called PIONEER-ALS and aims to try out a new treatment named VTx-002. Firstly, researchers want to carefully check how safe VTx-002 is and what side effects people might experience. They will test two different dose levels of the treatment. Because it's an early trial, it's open-label, meaning everyone involved knows who is receiving the new treatment. While the main goal is safety, the study will also look for any early hints that the treatment could help with ALS symptoms over 6 to 12 months. This trial is an important step in developing potential new therapies for ALS.
At a glance
What is this study about?
This study is called PIONEER-ALS and it's looking into a new experimental medicine, VTx-002, for people living with Motor Neurone Disease (MND), also known as Amyotrophic Lateral Sclerosis (ALS). MND causes muscle weakness and can make everyday activities difficult. This research is a 'Phase 1/2' trial, which means it's one of the very first times this medicine is being given to people. The main purpose of these early stage trials is to find out if the treatment is safe and what side effects it might have.
The study will involve giving two different amounts (doses) of VTx-002 to participants. Because it's an 'open-label' study, everyone involved – patients and doctors – will know that the treatment being given is VTx-002. Researchers will be carefully monitoring participants for any changes in their health.
While safety is the top priority, the study will also look for early signs of how VTx-002 might affect the disease. This includes checking if it impacts muscle function, breathing, or how long people live over a period of 6 to 12 months. This research is a crucial step to see if VTx-002 could be a promising future treatment for MND.
Key takeaways
- This study is for an investigational treatment (VTx-002) for Motor Neurone Disease (ALS).
- It's an early-stage (Phase 1/2) trial, mainly focused on checking safety and side effects.
- Participants will receive the experimental treatment; there is no placebo group.
- The study will track changes in muscle function, breathing, and overall health over 6-12 months.
- Participation involves regular hospital visits for assessments and monitoring.
- You can withdraw from the study at any time without affecting your medical care.
Who may be eligible?
To be able to join this study, people generally need to be adults, aged 18 years or older. Both men and women can take part.
There will be other detailed criteria that the study doctors will check to make sure the treatment is safe for you and that you fit what the study needs. For example, they will likely need to know about your specific type of MND, how long you've had it, and any other health conditions or medicines you are taking.
It's important to remember that not everyone who has MND will be able to join this study. The research team will carefully review your medical history and conduct tests to see if this particular study is right for you.
- Are you 18 years old or older?
- Have you been diagnosed with Motor Neurone Disease (ALS)?
- Are you able to attend regular hospital visits for up to a year?
- Are you open to trying an experimental treatment that is still being investigated?
This is a guide only — the research team will confirm whether you can take part.
What does participation involve?
If you decide to take part in this study, you would receive doses of the experimental medicine, VTx-002. The study will involve regular visits to a hospital or clinic over a period of up to 12 months. During these visits, the research team will conduct various checks to monitor your health and how you're responding to the treatment.
These checks will include blood tests, MRI scans to look at your brain and spinal cord, and assessments of your nerve function. You will also complete questionnaires about your symptoms and well-being, including questions about your mood. The study team will meticulously record any side effects you experience. There will also be follow-up appointments after you finish receiving the treatment to continue monitoring your health. The total duration of your participation, including treatment and follow-up, could be up to 12 months.
Potential risks and benefits
Locations (3)
- —Netherlands
- —Belgium
- —Spain
Common questions
What is Motor Neurone Disease (MND) or ALS?
MND, or ALS, is a condition that affects the nerves in your brain and spinal cord. It causes muscle weakness and can make everyday tasks like walking, speaking, and breathing difficult over time.
What does 'Phase 1/2' mean?
This means it's an early stage clinical trial. Phase 1 focuses on checking if a new treatment is safe for people, and Phase 2 looks at both safety and if the treatment shows any early signs of working.
What is VTx-002?
VTx-002 is the name of the new experimental medicine being tested in this study for people with MND. It's not yet approved and is still under investigation.
Will I get a placebo (dummy treatment)?
No, this study is 'open-label,' which means all participants will receive doses of the new experimental treatment, VTx-002. No one will receive a placebo in this particular study.
How long will the study last if I join?
If you join the study, your participation, including receiving the treatment and follow-up appointments, could last for up to 12 months.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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