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

Launched by RHEACELL GMBH & CO. KG · Apr 20, 2023

Keywords

ClinConnect Summary

This clinical trial is investigating a new treatment for a skin condition called recessive dystrophic epidermolysis bullosa (RDEB), which causes very fragile skin that blisters easily. The treatment being studied involves giving patients special cells (called allo-APZ2-OTS) through an IV to see if it is safe and effective in helping their skin heal, compared to a placebo, which looks like the treatment but has no active ingredients. The trial is currently recruiting participants of all genders, ages 6 months and older, who have been diagnosed with RDEB and have a specific type of wound that meets certain size and duration criteria.

To participate, individuals must be generally healthy and meet specific requirements, like having a target wound for at least 21 days but less than 9 months. They also need to be able to provide consent or have a legal representative who can. Participants can expect to be closely monitored throughout the trial, and there are some exclusions, such as having certain types of tumors or ongoing medical conditions that could affect safety. This trial will help researchers understand if this new treatment can improve the lives of those living with RDEB.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. Male or female subjects from 6 months of age with a diagnosis of RDEB confirmed by genetic testing or by a skin biopsy with immunofluorescence mapping
• • 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.