Not recruitingRare disease

Study on the Safety of Allogeneic Adipose Tissue-Derived Mesenchymal Stromal Cells for Patients with Epidermolysis Bullosa

Verified siteInvestigationalNo placebo

Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying the safety and effectiveness of a new treatment for epidermolysis bullosa, a rare genetic skin condition that causes the skin to be very fragile and to blister easily. The treatment being tested involves the use of allogeneic adipose tissue-derived mesenchymal stromal cells, which are special cells taken from fat tissue and used in a form that can be injected into the skin. These cells are known for their potential to help repair and regenerate damaged tissues.

The main goal of the study is to assess the safety of these cells when they are injected into the skin of patients with epidermolysis bullosa. Participants in the study will receive injections of the cell treatment, and some may receive a placebo. The study will monitor participants for any side effects, such as severe reactions at the injection site or allergic responses, as well as any changes in the condition of their skin. The study will also look at how the treatment affects the healing of skin lesions and the overall area of affected skin.

Throughout the study, participants will be asked to report on their experiences, including any itching or ease of changing wound dressings. The study is designed to gather important information that could lead to new treatment options for people living with epidermolysis bullosa. The trial is expected to continue until 2025, providing valuable insights into the potential benefits and risks of using these special cells for treating this challenging condition.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Joining the study
Upon joining the study, eligibility is confirmed based on criteria such as age, clinical diagnosis of epidermolysis bullosa, and extent of the disease.
Participants must agree to use prescribed contraceptive methods during the study and for at least six months after the last dose of the study medication.
Step 2
Initial assessment
An initial assessment is conducted to evaluate the extent of the disease, which must cover at least 10% of the body surface.
Baseline measurements are taken, including the total area of lesions on the body and patient-reported outcomes such as itching and ease of wound dressing change.
Step 3
Administration of study medication
The study medication, allogeneic adipose tissue-derived mesenchymal stromal cells, is administered as a suspension for injection.
The medication is given through intradermal use, which means it is injected into the skin.
Step 4
Monitoring and follow-up
Participants are monitored for any adverse events, including severe local reactions at the injection site, systemic allergic reactions, or infectious complications.
Regular follow-up visits are scheduled to assess the safety and efficacy of the treatment, including changes in the non-healed area of lesions and patient-reported outcomes.
Step 5
Completion of the study
The study is estimated to end by August 29, 2025.
Upon completion, final assessments are conducted to evaluate the overall safety and efficacy of the treatment.

Who can join the trial?

5 criteria

Must be older than 2 years.
Must have a clinical diagnosis of **hereditary epidermolysis bullosa (EB)**, which is confirmed by special tests like **electron microscopy** (a detailed look at cells) and **immunofluorescence antigen mapping** (a test to see specific proteins in the skin), as well as **DNA analysis** (a test to look at genes).
The disease must affect at least 10% of the body surface.
If you can have children, you must agree to use specific birth control methods during the study and for at least 6 months after the last dose of the study medication. For women, this includes using reliable birth control methods like hormonal pills, patches, or devices, or having a partner who has had a vasectomy. For men, this means using a condom or not having sexual intercourse.
Must be willing and able to follow the rules for taking care of the study-related skin areas and attend all scheduled visits and tests during the study.

Who cannot join the trial?

8 criteria

Patients who have any other serious health conditions that could interfere with the study.
Patients who are currently participating in another clinical trial.
Patients who have had a recent infection or illness that could affect the study results.
Patients who are pregnant or breastfeeding.
Patients who have a history of allergic reactions to similar treatments.
Patients who are unable to follow the study procedures or attend all required visits.
Patients who have a condition that affects their immune system, making them more vulnerable to infections.
Patients who have received certain medications or treatments that could interfere with the study.

Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.

We usually reply within a few days

Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Fakultni Nemocnice Brno
Verified
Brno, Czechia

Trial locations

Where you can join this trial

Countries are shaded by recruitment status. Click a recruiting country to ask about joining there.

Not recruiting

Not finding your country?

Not sure what to do next?

Joining a clinical trial can seem overwhelming. We guide you step by step, so you know exactly what to expect and how we support you through the process.

See the full process and FAQ

Investigated drugs

Allogenic Adipose Tissue-Derived Mesenchymal Stromal Cells are special cells taken from donated fat tissue. These cells are used in the trial to see if they can help treat patients with a skin condition called epidermolysis bullosa. The cells are injected into the skin to check if they are safe and if they can help improve the condition.

Allogeneic Adipose Tissue-Derived Mesenchymal Stem Cells Expanded

What is already known about the treatment

Allogeneic Adipose Tissue-Derived Mesenchymal Stromal Cells – This medication is administered through intradermal injections. It is currently being studied in clinical trials, specifically in Phase I/II, to evaluate its safety and effectiveness. The main therapeutic indication is for treating epidermolysis bullosa congenita, a rare skin condition. At the molecular level, these cells are believed to aid in tissue repair and regeneration by modulating the immune response and promoting healing. They are classified under cell-based therapies in pharmacology.

Investigated diseases

Epidermolysis bullosa congenita – This is a rare genetic condition that causes the skin to be extremely fragile, leading to easy blistering and tearing from minor friction or trauma. The disease is present from birth and can affect not only the skin but also mucous membranes inside the body. Blisters can form anywhere on the body, including the hands, feet, and areas subject to friction. Over time, repeated blistering can lead to scarring and changes in skin texture. The severity of the condition can vary widely, with some individuals experiencing mild symptoms and others facing more significant challenges. The disease is caused by mutations in genes responsible for skin integrity, affecting proteins that help bind skin layers together.