Clinical trials provide evidence that new or repurposed treatments will be safe and more effective than anything already in use to reduce EB symptoms. 

We award the money raised by our supporters to researchers whose proposals are approved by our rigorous application process, but we do not run the trials. 

The only way to join a clinical trial is through your specialist doctor.

Trials may be funded by other charities or companies and members of DEBRA can choose to participate in whichever trial they are eligible for.

 

Contents:

• Eligibility
• Benefits and risks
• Opportunities to take part in clinical trials
• Opportunities to take part in other research

 

Eligibility

For each clinical trial, there will be a list of ‘eligibility criteria’ that will be different for each trial. These may include: 

• being a certain age and sex
• having a certain type of EB or even a specific genetic change
• being able to attend a specific centre or clinic
• having certain EB symptoms to a defined level of severity. 

Clinical trials recruit a set number of people and offer treatment for a specific length of time. Often people will join at different times rather than all starting together. So, a trial that involves treatment for 12 people for 8 weeks each may take longer than 8 weeks for everyone to complete. There will often be a follow up appointment after the experimental treatment has finished.

Your specialist doctor will be able to help you join an appropriate trial if there is a suitable one for you currently recruiting participants. 

You (or your child) can only take part in a clinical trial if you have understood what the trial involves and given the researcher your permission (informed consent). After you have signed a consent form, you can still change your mind at any point and stop participating in the trial without giving a reason.

 

Benefits and risks

Participating in clinical trials and research may have some pros and cons to consider before you consent to take part. You can withdraw from a clinical trial or research project at any point without giving a reason, no matter what you have agreed to do, or the consent forms you may have signed.

 

 

Access to a new treatment:

A potential benefit of participating in clinical trials may be that you can try out something new that improves your symptoms. However, many trials will have a ‘control group’ of participants who do not receive the new treatment and act as a comparison group to see whether those receiving the new treatment really do benefit. It is also possible that the new treatment might not help at all or even make symptoms worse. 

 

Time and effort:

You may give up your time to travel and take part in research without seeing any personal benefits. The results of the trial may help to show that a new treatment works and should be made available to others. However, the trial may show that it is not effective. This is also valuable as it will save patients in the future from wasting time and money on something that doesn’t work, but you will not have experienced personal benefit.

 

Additional medical treatment:

You may experience additional appointments, discomfort or pain when giving samples for research beyond what was necessary for your personal treatment, although ideally research will be combined with your routine visits. Participation may mean additional contact with specialist healthcare professionals and extra opportunities for health checks.

 

Mental and emotional health:

You may find that being asked to share your experiences of living with EB or caring for someone with EB brings feelings of sadness or anxiety to the forefront of your mind. You may choose to participate in surveys or workshops when you feel emotionally strong and to avoid them if you are feeling vulnerable around certain dates or times that have an emotional significance to you. 

You are under no obligation to participate in research and can withdraw from a study at any point without giving a reason even after you have signed consent forms.

 

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Opportunities to take part in clinical trials:

We will share opportunities to take part in clinical trials in the UK. You can speak to your specialist doctor about joining one of these trials if you think it is suitable for you. 

 

Opportunities to join other research:

As well as clinical trials, there are opportunities for our members to share their experiences of living with EB. 

This could be a research project in its own right, like our 2023 Insight Study, with results that are analysed, published and shared to help future research succeed. 

Alternatively, researchers may ask you, as an Expert by Experience, to help them to design a better and more relevant research project. This is sometimes called Patient and Public Involvement and Engagement (PPI or PPIE) and helps researchers to hear the voices of people living with EB when they are planning what research to do and how to do it. Where projects are co-designed between researchers and people living with EB, the research is more likely to be successful. For example, experts by experience might be asked to suggest changes before a survey opens to see if it can be made more clear, compassionate or relevant, or to discuss whether the number of sample collections or appointments might put people off joining a trial before the researcher applies for funding.

We will share opportunities to take part in research and PPIE that involves surveys, questionnaires and workshops here. You do not need to speak to your specialist doctor about these opportunities but can read the information sheets supplied by the researchers and choose whether to share your experiences. Again, you can stop participating at any point without giving a reason, no matter what you have agreed to.

 

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Image credit: National Human Genome Research Institute.