Clinical Trials and Research
Helping Shape the Future of CMT
Research is helping us learn more about CMT, understand how it affects people differently and move closer towards better treatments and support.
We know that finding clear, reliable information about clinical trials can sometimes feel overwhelming. That’s why we’ve created this page to answer some of the most common questions, explain how clinical trials work and help you make informed decisions alongside your healthcare team.
Taking part in research is always your choice. Our aim is to provide balanced, easy-to-understand information so you can decide what feels right for you.
Research is Becoming Part of Everyday NHS Care
For people living with CMT, research offers hope. Every study, survey and clinical trial helps researchers build a clearer picture of the condition and work towards a future where more effective treatments are available.
Research is no longer something that happens behind the scenes. The NHS is working to make research a more visible and accessible part of healthcare, giving more people the chance to hear about studies and clinical trials that may be suitable for them.
Part of everyday care
The NHS is committed to making research a bigger part of everyday healthcare. This means that, wherever possible, people should have the opportunity to hear about research studies and clinical trials that may be relevant to them as part of their usual care.
Find out more on NIHR Be Part of Research →A 10 year plan for research
The NHS 10 Year Health Plan also aims to make it easier for people to find and take part in clinical research, helping improve access to new treatments and ensuring more people can benefit from advances in healthcare.
Read more about plans to expand clinical trials →For people living with CMT, this is particularly important. As researchers continue to develop new treatments, taking part in research gives us the best opportunity to improve understanding, improve care and create better options for the future.
Did you know?
The NHS Constitution says that patients should be informed about research studies they may be eligible to join.
If you’re interested in research, speak to your healthcare team and ask whether there are any patient registries, research studies or clinical trials that may be suitable for your type of CMT.
You can also search for current studies through NIHR Be Part of Research:
https://bepartofresearch.nihr.ac.uk/search/
Why Clinical Trials Matter
At the moment, there are very few treatments that can slow or stop the progression of CMT. Clinical trials are how we change that.
Before a new treatment can become available through the NHS, it needs to be carefully tested to understand whether it is safe, effective and makes a meaningful difference to people’s lives.
Research isn’t just about testing new medicines. Many studies focus on understanding how CMT changes over time, how symptoms affect everyday life and what matters most to people living with the condition.
This information helps researchers design better clinical trials and develop treatments that focus on the things that matter most to the CMT community.
Research helps us to:
- – Better understand how CMT progresses
- – Identify the symptoms that have the biggest impact on everyday life
- – Improve rehabilitation and symptom management
- – Develop and test potential new treatments
- – Prepare the NHS for future therapies
Every Contribution Matters
Whether you take part in a clinical trial, join a patient registry or complete a research survey, your contribution can make a difference.
Every piece of information helps researchers understand CMT better and brings us closer to improved treatments and support.
Common myths about clinical trials
Click a card to reveal the fact
Myth
I'm too old to take part.
Fact
Many studies welcome adults of different ages. Each study has its own eligibility criteria, so it's always worth finding out whether you may be suitable.
Myth
Once I join a study, I can't leave.
Fact
Taking part in research is always voluntary. You can withdraw from a study at any time without it affecting your usual healthcare.
Myth
Clinical trials are only for people with severe CMT.
Fact
Different studies are looking for different people. Some may focus on people in the early stages of CMT, while others may need participants with more advanced symptoms.
Myth
There's no point taking part if I don't receive the new treatment.
Fact
Every participant provides valuable information that helps researchers understand CMT and improve future research.
What questions should I ask before joining?
It’s completely normal to have questions before taking part in research.
You may want to ask:
- What is the purpose of the study?
- What will taking part involve?
- How long will the study last?
- Are there any possible risks or benefits?
- Will I need to travel?
- Will expenses be covered?
- Who should I contact if I have questions?
There are no wrong questions to ask. It’s important that you feel comfortable, informed and confident before making your decision.
What happens before I start?
If you decide to take part, you will receive a Patient Information Sheet explaining exactly what the study involves.
Take your time to read this carefully and discuss it with your family, carers or healthcare team before deciding.
Before joining the study, you will be asked to give informed consent. This means you understand what taking part involves and are choosing to participate voluntarily.
Even after joining, you can change your mind and leave the study at any time. This will not affect your usual healthcare.
Frequently asked questions
A clinical trial is a research study that looks at whether a medicine, treatment, therapy or medical device is safe and effective.
Before a new treatment can become available through the NHS, it must go through carefully regulated clinical trials. Every study follows strict scientific, ethical and safety standards to protect participants and ensure the results are reliable.
Participant safety is always the top priority.
Before a clinical trial can begin, it must be reviewed by independent experts to make sure it is safe and ethical.
If you decide to take part, you will receive detailed information about the study, have the opportunity to ask questions and can choose to leave the study at any time.
Potential new treatments go through several stages before they can become widely available.
| Phase | What happens |
|---|---|
| Preclinical | Researchers study a potential treatment in the laboratory before testing it in people. |
| Phase 1 | A small group of participants helps researchers understand the safest dose and identify possible side effects. |
| Phase 2 | Researchers begin looking at how well the treatment works while continuing to monitor safety. |
| Phase 3 | Larger groups help confirm how effective and safe the treatment is before approval is considered. |
Because CMT is a rare condition, studies often involve fewer participants than trials for more common conditions.
A placebo looks like the treatment being tested but does not contain any active medicine.
Researchers sometimes compare people receiving the new treatment with people receiving a placebo to understand whether the treatment is making a genuine difference.
In some studies, neither you nor your doctor will know which treatment you are receiving until the study ends. This is known as a double-blind trial and helps make the results as accurate as possible.
Not all research involves testing new medicines.
Some studies look at how CMT changes over time or collect information about symptoms such as mobility, fatigue, pain and quality of life.
These studies are just as important because they help researchers understand CMT and design better clinical trials in the future.
Some clinical trials can run for several years, but this does not mean you will necessarily be involved for the entire time.
Before deciding whether to take part, the research team will explain exactly what your involvement would include and how long it is expected to last.
Every study has its own eligibility criteria.
Researchers may be looking for people with a particular type of CMT, age group, genetic diagnosis or level of mobility. This helps make sure the study can answer the questions it has been designed to investigate.
If you are not suitable for one study, it does not mean you will not be eligible for another in the future.
There are many ways to support CMT research.
You can help by joining a patient registry, taking part in observational studies or completing research surveys.
Every contribution helps researchers build a better understanding of CMT.
Useful Resources
If you’d like to find out more about research and clinical trials, these trusted resources are a good place to start.
NIHR Be Part of Research
Search for research studies and register your interest in taking part.
https://bepartofresearch.nihr.ac.uk/
Take Part in Research
Find out more about what taking part in research involves and what you can expect.
https://bepartofresearch.nihr.ac.uk/take-part-in-research/
Search for Clinical Trials
Browse research studies and clinical trials taking place across the UK.
https://bepartofresearch.nihr.ac.uk/search/
Discover more
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CMTUK is a donation-based charity. We don’t receive government funding – everything we do is made possible thanks to the generosity of our community. Every pound helps us provide vital support, raise awareness, and fund research for people living with Charcot-Marie-Tooth disease.