Ongoing, recruitingPhase I and Phase II (Integrated)- OtherInterventional

Safety and efficacy of allogenic adipose tissue-derived mesenchymal stromal cells in patients with epidermolysis bullosa: Clinical trial phase I/II

This clinical trial is looking into a new treatment for Epidermolysis Bullosa (EB), a condition that makes your skin very fragile and prone to blistering. The treatment uses special cells called mesenchymal stromal cells, which are taken from fat tissue. The main goal of this study is to find out if these cells are safe for people with EB. Researchers will be carefully monitoring for any unwanted side effects, such as allergic reactions or infections, especially around the injection site. They also want to see if this treatment can help the skin heal better, reduce the size of skin lesions, and improve daily symptoms like itching. The study is open to adult patients and combines two phases of research to get a good understanding of both safety and how well the treatment works.

At a glance

Status

Ongoing, recruiting

Phase

Phase I and Phase II (Integrated)- Other

Sponsor

Masarykova Univerzita

Enrolment target

Start

11 Nov 2024

Epidermolysis bullosa congenita

What is this study about?

This research study is about finding new ways to help people living with a difficult skin condition called Epidermolysis Bullosa (EB). If you have EB, your skin can be very fragile and might blister or tear easily. This trial is investigating a new treatment that uses special cells called mesenchymal stromal cells. These cells come from healthy fat tissue and are thought to have properties that could help with healing and inflammation.

The main purpose of this study is to check how safe this new cell treatment is for people with EB. Doctors will be very carefully looking for any side effects, particularly serious reactions where the cells are injected, any widespread allergic reactions, or infections. They also want to see if the treatment actually helps. This means they will measure how well your skin wounds heal, check if the total area of affected skin on your body changes, and ask about your daily symptoms like itching and how easy it is to change your wound dressings.

By taking part, you would be helping doctors understand if this cell treatment could be a safe and effective option for people with EB in the future. It's a combination of earlier research stages (Phase I and Phase II), which means they are looking at both safety and how well the treatment works at the same time.

Key takeaways

Exploring a new cell treatment for Epidermolysis Bullosa (EB).
Primary focus is on the safety of the treatment.
Also looking for improvements in skin healing and symptoms like itching.
Adults aged 18 and over with EB can inquire about participation.
Combines early research phases (Phase I/II) to assess both safety and effectiveness.

Who may be eligible?

This study is looking for adult volunteers. You must be at least 18 years old to be considered for participation. There is no upper age limit, meaning older adults are also welcome to inquire.

Both men and women are encouraged to take part in this research. The study aims to gather information from a broad range of adults living with Epidermolysis Bullosa.

Essentially, if you are an adult with Epidermolysis Bullosa, you might be eligible for this study. However, the research team will do a thorough check to make sure the study is a good fit for your specific health situation.

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 Epidermolysis Bullosa?
Are you willing to attend regular check-ups and follow study instructions?
Are you comfortable with receiving a new type of cell treatment?
Are you able to provide your informed consent?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will receive the special cell treatment. The study doctors and nurses will closely monitor your health right from the start to make sure you are safe and to watch how your body responds to the treatment. This will involve regular check-ups, where they will look at your skin, especially the areas where you have blisters or wounds. They will also ask you questions about how you are feeling, any new symptoms you might have, and how your daily life is affected by your skin condition, including things like itching and changing your dressings. They will also measure the size of your wounds and the total area of affected skin. The total duration of your participation in the study and the number of visits will be explained in detail by the research team.

Potential risks and benefits

Taking part in a clinical trial always involves some potential benefits and risks. A potential benefit of this study is that the new cell treatment might help improve your skin condition, speed up healing, and reduce uncomfortable symptoms like itching. However, it's also possible that the treatment may not help you, or you might experience side effects. The main focus of this study is to carefully look for potential risks, such as local reactions where the cells are injected, severe allergic reactions, or infections. The research team will monitor you very closely for any of these issues. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (1)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Czechia

Common questions

What is Epidermolysis Bullosa (EB)?

EB is a rare genetic skin condition that makes your skin very fragile and prone to blistering and tearing, sometimes with very little friction.

What are 'mesenchymal stromal cells'?

These are special cells, often taken from fat tissue, that have properties which may help with healing and reducing inflammation in the body.

What does 'Phase I and Phase II' mean?

These are stages of clinical research. Phase I usually checks safety, and Phase II looks at how well the treatment works. This study combines both to learn about safety and effectiveness at the same time.

Will I know if I'm getting the actual treatment?

The study design will determine this. Sometimes, participants receive the treatment, and others might receive a placebo (dummy treatment) for comparison, or a different standard treatment. The research team will explain this to you.

What does a 'primary safety endpoint' mean?

This is the main thing the researchers are looking at to determine if the treatment is safe. In this study, it's about checking for serious side effects like reactions at the injection site, severe allergies, or infections.