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Scientific research contributes to our goal of a world where no one suffers from EB symptoms because it is the best way to try to find out what actually causes a symptom and what can really make a difference to it.  

Research happens in partnership with lots of different experts and groups because it would be hard for anyone to know everything about all the parts of the body that are involved in the symptoms of EB. 

Companies, charities, doctors, scientists and people living with EB all contribute to research projects and help us to know more about EB and how to fight it.

Watch the short video below to find out more about how research happens.

Scientific research begins with an observation: seeing something happen. If a baby is born with blistered skin, this symptom is an observation. Some researchers will study how often it happens, when and where and how. The observation leads researchers to consider what causes the symptom and how it can be treated.

Doctors don’t need to know exactly what causes a symptom before they try to treat it but knowing more can help researchers choose which existing treatments might work best and which could make things worse. 

It is important to know how well different potential treatments work. Clinical trials are carried out by researchers to compare them to each other.

DEBRA UK funds medical research for clinical trials on treatments being developed, to repurpose existing treatments and basic science.

 

How do clinical trials work? 

Clinical trials are carried out to demonstrate whether a treatment, medical strategy or device is safe and whether it is as effective to treat or prevent symptoms as the approaches already in use.

Diagram which shows how clinical trials work

Initially a trial may only be carried out on a small number of people in case there are unforeseen side effects. If a phase 1 trial shows a new treatment is safe, it can be trialled on people with symptoms in a phase 2 trial. This stage can be skipped if a treatment already in use for another condition is being considered as it will already have been shown to be safe to use. This is called Drug Repurposing and is an important part of our research strategy as it has the potential to provide effective treatments more quickly to families living with EB. 

If a phase 2 trial shows the treatment appears to be safe and effective in a small number of people with EB, a phase 3 trial can be carried out on a larger number of individuals to show that this new treatment will work better than what is currently being used. This phase can also provide information about the safety and effectiveness of different doses or frequency of treatment and discover more about the risks of potential side effects.

When a treatment passes a phase 3 trial successfully it can be approved and licensed for use and manufacture in different countries. This process can take a few more months but helps to ensure that the treatments available are genuinely safe and effective.

Once a treatment is in use more widely, phase 4 trials can continue as more and more people start using it. This allows researchers to fine tune what is known about a treatment and adjust the way it is used to make it even more effective.

You can read more about how different types of clinical trial work in this article.

 

 

Who is involved in a clinical trial?

To carry out a clinical trial, a lot of funding is required to support a research team including many different experts such as doctors, nurses, social workers, health care professionals, scientists, data managers, and clinical trial coordinators. It also requires willing volunteers to participate in a trial and this can involve both risks and benefits. Participants will always be asked to give their “informed consent” before joining a trial. This means that they aren’t just agreeing because they trust their healthcare provider but have fully understood what their involvement will mean before they agree. It is also important for participants to know that they can choose to withdraw from a clinical trial at any point without giving any reason. At the end of a clinical trial, the number of participants who didn’t choose to complete it can give an idea of how difficult the treatment was to tolerate. The progress of the clinical trial will be monitored, and some are stopped early if the risks appear to outweigh the benefits but the nature of medical research studies means that some risks are unavoidable. 

The results of clinical trials add to medical knowledge and provide reliable information to assist in health care decision-making and better guidelines for treatment.

 

What treatments for EB are currently undergoing clinical trial? 

There are various treatments being trialled for EB. These include topical treatments that are applied to wounds and systemic treatments that are taken by mouth as tablets or are transfused directly into the blood. There are new treatments involving genetic engineering developed specifically for EB and medicines that have been used in other conditions that affect the skin and may help reduce EB symptoms. Some are just beginning the journey at phase 1 while others are closer to approval.

Here is a list of some of the potential treatments for EB that are currently undergoing clinical trials.

Clinical trials involving EB are listed here  by the US National Library of Medicine if you would like more details on any of these studies.

To find out about specific research studies that you could be involved in, please talk to your specialist doctor or nurse. They will be able to find out if you could participate in research local to you.