A double-blind, randomised, placebo-controlled, first-in-human (FIH) Phase I/IIa, multi-centre trial to assess safety, tolerability, and immune response of single and multiple ascending doses of TOL2 (Immune Tolerising Agent) in patients with generalised myasthenia gravis
This study is an early-stage trial in the UK for a new medicine called TOL2, designed to help people with generalised myasthenia gravis. The main aim is to understand if TOL2 is safe and how well people tolerate it, looking at any side effects. Researchers will also check how the medicine affects the immune system. We’ll be giving different doses of the medicine, both single and multiple, to see how the body reacts. This is the first time this medicine is being given to people, so careful monitoring is in place to ensure patient safety.
At a glance
What is this study about?
This study is looking at a new medicine called TOL2 for people with a condition called generalised myasthenia gravis. Myasthenia gravis is a long-term condition that causes muscle weakness. TOL2 is designed to help reset the body's immune system, which is believed to play a role in myasthenia gravis.
This is a very early-stage trial, meaning it's the first time this medicine is being given to humans. It's called a 'Phase I/IIa' study. The main goals are to find out how safe TOL2 is, what side effects it might cause, and how well people can tolerate it. We'll be giving different doses of the medicine to understand how it works in the body and if it has any effect on the immune system.
Researchers will be carefully monitoring participants for any changes, especially any worsening of myasthenia gravis symptoms or serious reactions. They will also look at how the medicine affects standard health checks like blood tests and heart readings. The hope is that this research will one day lead to better treatments for people living with generalised myasthenia gravis.
Key takeaways
- This is an early-stage study for a new medicine called TOL2 for generalised myasthenia gravis.
- The main goal is to check how safe TOL2 is and what side effects it might cause.
- Participants will receive either TOL2 or a placebo (dummy medicine).
- Close monitoring, including blood tests and check-ups, will be part of taking part.
- You can stop participating in the study at any time.
- This is a 'first-in-human' study, meaning the medicine is new to people.
Who may be eligible?
To join this study, participants must be at least 18 years old. Both men and women can take part.
There might be other health requirements or medicines you're taking that would mean you can't join. For example, if you have certain other medical conditions or are on specific treatments, the researchers might decide it's not safe for you to participate.
The research team will carry out a thorough check-up to make sure the study is right for you and that taking part would be safe.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Are you 18 years old or older?
- Do you have generalised myasthenia gravis?
- Are you able to attend regular hospital visits?
- Are you comfortable with the possibility of receiving a placebo?
- Are you willing to have regular blood tests and physical examinations?
What does participation involve?
If you decide to take part, you will be given the study medicine, TOL2, or a placebo (a dummy medicine with no active ingredients). This will involve receiving either a single dose or several doses, depending on which part of the study you join.
You'll have regular visits to the hospital or clinic. During these visits, the research team will perform various assessments. These will include physical examinations, blood tests, urine tests, heart recordings (ECGs), and checks of your vital signs like blood pressure and heart rate. They will also ask you questions about your symptoms and how you are feeling.
Your participation will involve ongoing monitoring to track any side effects and to see how your body is reacting to the medicine. The total duration of your involvement, including follow-up appointments, will be explained by the research team.
Potential risks and benefits
Locations (3)
- —UnverifiedGermany
- —UnverifiedDenmark
- —UnverifiedSweden
Common questions
What is generalised myasthenia gravis?
It's a long-term condition that causes muscle weakness that can affect muscles all over your body.
What is TOL2?
TOL2 is a new medicine being tested. It's designed to help calm down the body's immune system, which can be overactive in myasthenia gravis.
What does 'first-in-human' mean?
It means this is the first time this specific medicine is being given to people, so we are learning about its effects for the first time.
Will I get the actual medicine or a dummy one?
This is a 'double-blind, randomised, placebo-controlled' study. This means some participants will receive TOL2, and others will receive a placebo (dummy medicine), but neither you nor the study team will know which you are getting.
What are 'side effects'?
Side effects are any unwanted reactions or problems that can happen when taking a medicine. Researchers will monitor these very closely.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
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