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Study on ABCB5+ MSCs for Recessive Dystrophic and Junctional Epidermolysis Bullosa Patients

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What is this trial about?

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

This clinical trial is focused on studying a rare skin condition called epidermolysis bullosa, which causes the skin to be very fragile and to blister easily. The study is specifically looking at two types of this condition: recessive dystrophic epidermolysis bullosa (RDEB) and junctional epidermolysis bullosa (JEB). The treatment being tested is called allo-APZ2-OTS, which involves using special cells known as ABCB5-positive mesenchymal stromal cells. These cells are derived from skin and are administered through an injection into the bloodstream.

The purpose of the study is to evaluate the safety and effectiveness of this treatment compared to a placebo. Participants will receive the treatment or placebo over a period of time, and their progress will be monitored to see if there is any improvement in their condition. The study will also include an open-label part, where all participants will receive the treatment, to further assess its safety and effectiveness.

Throughout the study, participants will have regular check-ups to monitor their health and the condition of their skin. The study aims to see if the treatment can help close wounds, reduce pain and itching, and improve the overall quality of life for those with epidermolysis bullosa. The trial will also look at any potential side effects or immune reactions to the treatment. The study is expected to continue until 2025, with participants being closely monitored throughout the process.

The research process

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

  1. Step 1

    Initial visit and consent

    Upon joining the study, you will attend an initial visit where the study team will explain the trial in detail. You will be asked to provide written informed consent, which means you agree to participate after understanding the study's purpose and procedures.

    During this visit, a series of tests will be conducted to confirm your eligibility. These tests include genetic testing or a skin biopsy to confirm a diagnosis of recessive dystrophic epidermolysis bullosa (RDEB) or junctional epidermolysis bullosa (JEB).

  2. Step 2

    Baseline assessment

    If eligible, a baseline assessment will be conducted. This includes measuring your target wound size and checking for any signs of infection. Your general health will be assessed through blood tests and other evaluations to ensure you meet the health criteria for the trial.

  3. Step 3

    Treatment administration

    You will receive the study treatment, which involves the administration of allo-APZ2-OTS intravenously. This means the treatment is given through a vein. The frequency and duration of administration will be explained to you by the study team.

  4. Step 4

    Follow-up visits

    Regular follow-up visits will be scheduled to monitor your health and the effects of the treatment. During these visits, the study team will assess the closure of your target wound and any changes in your condition.

    You will be asked to report any side effects or changes in your health. Blood samples may be taken to monitor your body's response to the treatment.

  5. Step 5

    Final assessment

    At the end of the study period, a final assessment will be conducted. This will include a comprehensive evaluation of your health and the condition of your wounds.

    The study team will discuss the results with you and provide information on any further steps or follow-up care that may be needed.

Who can join the trial?

9 criteria

  • Participants can be male or female from birth with a confirmed diagnosis of Recessive Dystrophic Epidermolysis Bullosa (RDEB) or Junctional Epidermolysis Bullosa (JEB). This diagnosis must be confirmed by genetic testing or a skin biopsy using a method called immunofluorescence mapping (IFM).
  • Participants must weigh at least 5 kg (about 11 pounds).
  • Participants must have a general health condition that meets specific lab values:
    • Hematology: A type of white blood cell count called absolute neutrophil count must be greater than 1000 per cubic millimeter, and platelet count must be greater than 150,000 per microliter.
    • Hepatic (liver function): Levels of liver enzymes called AST and ALT must be less than 2 times the upper limit of normal for the participant's age.
    • Renal (kidney function): Creatinine levels must be less than 2 times the upper limit of normal for the participant's age.
    • Pulmonary (lung function): Oxygen levels in the blood must be greater than 92% when breathing room air, without needing extra oxygen.
    • Participants must have a target wound that is between 5 and 50 square centimeters in size, less than 9 months old, and shows no signs of acute infection.
    • The participant or their legal representative must understand the nature of the clinical trial and provide written informed consent or assent before any trial procedures begin.
    • Women who can become pregnant must have a negative urine pregnancy test at the first visit. Women who can become pregnant, male participants, and their partners must agree to use highly effective birth control methods throughout the entire clinical trial.

Who cannot join the trial?

10 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 the required visits.
  • Patients who have received certain medications or treatments that could interfere with the study.
  • Patients who have a history of drug or alcohol abuse.
  • Patients who have a mental health condition that could affect their ability to participate in the study.
  • Patients who have a family history of certain genetic disorders that could affect the study.
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Investigated drugs

  • ABCB5-positive mesenchymal stromal cells (ABCB5+ MSCs)

    are a type of cell therapy being tested in this clinical trial. These cells are special because they have a marker called ABCB5 on their surface. They are being studied for their potential to help treat a skin condition called epidermolysis bullosa (EB), which causes the skin to be very fragile and to blister easily. The idea is that these cells might help repair or strengthen the skin, making it less prone to damage.

  • allo-APZ2-OTS

    is another type of cell therapy being tested in this trial. It is given through an intravenous infusion, which means it is delivered directly into the bloodstream. This therapy is being studied for its potential to help people with a severe form of epidermolysis bullosa called recessive dystrophic epidermolysis bullosa (RDEB). The goal is to see if this treatment can improve the condition of the skin and make it more resilient.

What is already known about the treatment

allo-APZ2-OTS – This medication is administered intravenously, meaning it is given directly into a vein through an injection. It is currently being studied in a phase III clinical trial to assess its safety and effectiveness for treating two rare skin conditions: Recessive Dystrophic Epidermolysis Bullosa (RDEB) and Junctional Epidermolysis Bullosa (JEB). The main therapeutic use of allo-APZ2-OTS is to help manage these conditions, which cause fragile skin that easily blisters and tears. At the molecular level, the medication involves ABCB5-positive mesenchymal stromal cells, which are believed to aid in skin healing and regeneration. It falls under the pharmacological classification of cell-based therapies, which use living cells to treat or manage diseases.

Investigated diseases

  • Recessive Dystrophic Epidermolysis Bullosa

    This genetic disorder is characterized by fragile skin that easily blisters and tears from minor friction or trauma. It is caused by mutations in the COL7A1 gene, which affects the production of collagen, a protein that helps strengthen and support the skin. As the disease progresses, repeated blistering can lead to scarring and fusion of fingers and toes, as well as other complications like esophageal strictures. The skin's fragility often results in chronic wounds that are slow to heal. Over time, individuals may experience a reduction in mobility and increased risk of infections due to open wounds. The condition is present from birth and affects various parts of the body, including the skin and mucous membranes.

  • Junctional Epidermolysis Bullosa

    This genetic condition leads to severe blistering of the skin and mucous membranes, often starting at birth. It is caused by mutations in genes responsible for proteins that help attach the outer layer of the skin to the underlying layers. As the disease progresses, affected individuals may develop chronic wounds, scarring, and potential complications such as respiratory and gastrointestinal issues. The skin is extremely fragile, and even minor friction can cause painful blisters. Over time, the condition can lead to significant discomfort and challenges in daily activities due to the persistent skin damage. The severity and specific symptoms can vary depending on the exact genetic mutations involved.

Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2022-500266-10-00Protocol codeallo-APZ2-EB-IIIEstimated enrolment167 patientsSponsorRheacell GmbH & Co. KG

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