A Phase 1b, Open-Label, Multiple Ascending Dose, Multicenter Study of VRG50635 in Participants With Sporadic and Familial Amyotrophic Lateral Sclerosis Followed by Long-Term Treatment
This research is looking into a new experimental medicine, VRG50635, for people living with Amyotrophic Lateral Sclerosis, often called ALS or motor neurone disease. This is an early-stage study (Phase 1b), which means the main goals are to understand how safe the medicine is, how much of it people can take, and how it behaves in the body. Researchers also want to see if it might be helpful. The study involves a small number of people and is happening in several locations. Participants will take different amounts of the medicine, and everyone will know what treatment they are receiving. After the main part, there will also be a long-term treatment option. Both people with the more common form of ALS and those with an inherited form are invited to take part.
At a glance
What is this study about?
This study is about a new medicine called VRG50635, which is being investigated for people who have Amyotrophic Lateral Sclerosis (ALS). ALS is a serious condition that affects the nerves in the brain and spinal cord, making it harder to move, speak, swallow, and breathe over time. This research aims to find out more about this new medicine.
This is an early-stage study, known as Phase 1b. In these early phases, the main focus is on safety. Doctors want to understand if the medicine has any unwanted side effects and how well people tolerate it. They also want to figure out the right amount (dose) of the medicine to give and how it travels through and leaves the body. Because it's an early study, we don't yet know if the medicine will be effective in treating ALS, but learning about its safety and how it works is an important first step.
The study is 'open-label,' which means that everyone involved – both the participants and the study team – will know that the participants are receiving VRG50635. It's also a 'multiple ascending dose' study, meaning that different groups of participants will receive increasing amounts of the medicine, allowing researchers to find the best and safest dose. The study will take place in several hospitals or clinics.
Key takeaways
- This study is testing a new medicine (VRG50635) for ALS.
- It's an early-stage study focusing on safety and dosage.
- Participants will receive the active medicine, not a placebo.
- Both common and inherited forms of ALS are included.
- Adults aged 18 and over can participate.
- Your health and safety will be carefully monitored.
Who may be eligible?
To be part of this study, you need to have been diagnosed with Amyotrophic Lateral Sclerosis (ALS). This includes both the more common type of ALS and forms that run in families.
Participants must be adults, aged 18 years or older. There is no upper age limit, meaning older adults are welcome to join if they meet all other requirements.
Both men and women can take part in this research. The study team will check a few other things to make sure the study is a good fit for you and that it's safe for you to participate.
- Are you 18 years old or older?
- Do you have a diagnosis of Amyotrophic Lateral Sclerosis (ALS), either common or inherited?
- Are you willing to visit the study clinics regularly for check-ups?
- Are you comfortable with receiving an experimental medicine?
- Are you able to understand and provide informed consent?
This is a guide only — the research team will confirm whether you can take part.
What does participation involve?
If you join this study, you'll be given the new medicine, VRG50635, and both you and the study team will know you are receiving it. You won't receive a placebo (a dummy pill). The study will involve several visits to the clinic for check-ups. These visits will include health assessments, blood tests, and other tests to see how the medicine is affecting your body and your ALS symptoms. The exact number of visits and tests will be explained to you by the study team.
You will take the study medicine for a specific period, and after that, there might be an option for you to continue receiving the medicine as part of a long-term treatment plan, if you and the study doctors agree. Your total participation time will vary depending on which part of the study you are in and whether you choose to continue with the long-term treatment. Everything will be explained in detail before you decide to join.
Potential risks and benefits
Locations (5)
- —Finland
- —Belgium
- —Netherlands
- —Spain
- —Sweden
Common questions
What is ALS?
ALS stands for Amyotrophic Lateral Sclerosis. It's a condition that affects nerve cells in your brain and spinal cord, making it difficult to control your muscles over time.
What does 'Phase 1b' mean?
Phase 1b is an early stage of testing a new medicine. It mainly focuses on figuring out if the medicine is safe, what the right dose is, and how it works in the body.
Will I know if I'm getting the real medicine?
Yes, in this study, everyone knows they are receiving the study medicine, VRG50635. It's not like studies where some people get a dummy medicine (placebo).
How long will the study last for me?
The initial part of the study will have a set duration. If you meet the criteria and wish to, there may be an option to continue with long-term treatment after that. The study team will explain the exact timeline.
Can I stop participating if I want to?
Yes, you are free to leave the study at any time, for any reason, without it affecting your regular medical care. Your decision will be respected.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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