A study to learn how safe KER-0193 is, the activity of KER-0193 in the body over a period of time, and the effect of KER-0193 to the body in adult healthy people taking single and multiple doses by the mouth that are increased a little at a time
This research is investigating a new medication, KER-0193, that could potentially help people with Fragile X syndrome. This early-stage study, called a Phase 1 trial, is specifically designed for healthy adult volunteers. The main goal is to understand how safe KER-0193 is when taken by mouth. Researchers will also be observing how the medicine acts in the body over time — how much stays in the body, how it's used, and if it causes any effects. Participants will start with a very small dose, which will be slowly increased, to help scientists find the safest and most effective amount for future studies. This is the very first time this medicine is being given to people.
At a glance
What is this study about?
This research is focused on a new medicine called KER-0193. It's hoped that this medicine might one day help people who have Fragile X syndrome. Fragile X syndrome is a genetic condition that can cause a range of learning, developmental, and behavioural challenges.
Because KER-0193 is a new medicine, researchers first need to carefully study it in healthy volunteers to make sure it's safe. This type of study is called a Phase 1 clinical trial, and it's always the first step when a new medicine is given to people. The main aim is to find out if the medicine causes any side effects, how the body handles the medicine (for example, how long it stays in the system), and what effects it might have.
Volunteers in this study will be healthy adults. They will be given KER-0193 by mouth, starting with a very small amount. This dose will then be slowly and carefully increased. This careful approach helps researchers understand the medicine's safety at different levels and how the body reacts. The information from this study is vital because it will help guide further research into KER-0193 and, if successful, could eventually lead to a new treatment option for people with Fragile X syndrome.
Key takeaways
- This is an early-stage study for a new medicine called KER-0193 for Fragile X syndrome.
- It aims to find out how safe the medicine is and what it does in the body of healthy adults.
- Participants will take the medicine by mouth, starting with small, increasing doses.
- Regular visits and medical checks will monitor health and side effects.
- No direct health benefit is expected for healthy participants; the benefit is to future patients.
- You can withdraw from the study at any time without affecting your normal medical care.
Who may be eligible?
This study is looking for healthy adults to take part. This means you should be at least 18 years old.
To be considered healthy, you shouldn't have any major ongoing health problems. The healthcare team running the study will check your health carefully to make sure it's safe for you to participate. Both men and women are welcome to join.
There might be other specific health checks or conditions that mean you can or cannot join the study, even if you feel healthy. These will be explained to you in detail if you are interested in taking part.
- Are you 18 years old or older?
- Do you consider yourself generally healthy with no major ongoing medical conditions?
- Are you comfortable taking a new medicine that has not been given to people before?
- Are you able to attend several clinic appointments as required by the study team?
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 will be given the new medicine, KER-0193, by mouth. You will start with a small dose, which will then be slowly increased over time at planned intervals. During the study, you will need to attend several appointments at the study clinic. At these visits, doctors and nurses will monitor your health very closely to check for any changes or side effects.
These check-ups will likely include some common medical tests, such as blood tests, urine tests, and checking your blood pressure and heart rate. You will also be asked questions about how you are feeling. The exact number of visits, the length of time you'll be taking the medicine, and the total duration of the study will be fully explained to you before you agree to participate.
Potential risks and benefits
Locations (1)
- —Belgium
Common questions
What is Fragile X syndrome?
Fragile X syndrome is a genetic condition that can cause a range of learning difficulties, developmental delays, and behavioural challenges.
Why is this study only for healthy adults?
This is an early-stage 'first-in-human' study. It's done in healthy volunteers first to carefully check the medicine's safety and how it works in the body before it's given to people with the condition.
Will I get paid to take part?
Payment for participation or help with travel costs can vary between studies. This would be clearly explained to you by the study team if you express interest.
Do I have to take the medicine every day?
The schedule for taking the medicine will be clearly explained. It will include starting with specific doses and potentially increasing them over time, as guided by the study doctors.
What if I feel unwell during the study?
The study team will monitor your health very closely. If you feel unwell at any point, you must tell the study doctors or nurses immediately so they can provide care and assess your situation.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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