Allogeneic ABCB5-positive Dermal Mesenchymal Stromal Cells for Treatment of Epidermolysis Bullosa (Phase III, Cross-over)

Launched by RHEACELL GMBH & CO. KG · Jul 18, 2022

Keywords

ClinConnect Summary

This clinical trial is looking at a new treatment for a skin condition called Epidermolysis Bullosa (EB), specifically a type known as recessive dystrophic epidermolysis bullosa (RDEB) and another type called junctional epidermolysis bullosa (JEB). The researchers want to find out if a special type of cells (called allo-APZ2-OTS) given through an IV (a small tube in the vein) is safe and effective compared to a placebo (a treatment that doesn't contain the active drug). The trial is open to both boys and girls who have been diagnosed with RDEB or JEB, and have a specific type of wound that fits certain criteria.

To participate, individuals should be generally healthy, have a wound that is at least 5 cm² but not larger than 50 cm², and have had that wound for at least 21 days. They also need to understand the study and agree to participate by signing a consent form. However, some people may not be eligible, including those with certain cancers, allergies to the treatment, or those who are pregnant or breastfeeding. Participants can expect to receive close monitoring during the trial, and their safety and well-being will be a top priority throughout the process.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. Male or female subjects with a diagnosis of RDEB or JEB confirmed by genetic testing or by a skin biopsy with immunofluorescence mapping (IFM);
• • 2. Subject is eligible to participate in this clinical trial based on general health condition;
• • 3. Subject with a target wound meeting the following criteria: 5-50 cm2, ≥ 21 days and \< 9 months, no signs of acute infection;
• • 4. Patient/legal representative understands the nature of the procedure and provide written informed consent/assent prior to any clinical trial procedure;
• • 5. Women of childbearing potential must have a negative urine pregnancy test at Visit 1. Women of childbearing potential, male participants, and their partner must be willing to use highly effective contraceptive methods during the course of the entire clinical trial.
• Exclusion Criteria:
• • 1. Any current tumor diseases, including squamous cell carcinoma and basal cell carcinoma;
• • 2. Any known allergies to components of the IP or premedication;
• • 3. Patient/legal representative anticipated to be unwilling or unable to comply with the requirements of the protocol;
• • 4. Pregnant or lactating women;
• • 5. Current or previous (within 30 days of screening) treatment with another IP, or participation and/or under follow-up in another interventional clinical trial;
• • 6. Previous participation in this clinical trial (except for screening failures);
• • 7. Clinically significant or unstable concurrent disease or other clinical contraindications like an uncontrolled or poorly controlled mental health condition of the subject and/or his/her legal representative that could impact on patient's safety or interfere with study compliance such as inability to attend scheduled study visits; Confidential
• • 8. Employees of the sponsor, or employees or relatives of the investigator.