Using stem cells to treat a skin condition called Epidermolysis Bullosa

A Double-blind, Randomized, Placebo-controlled, Interventional, Multicenter, Phase III Clinical Trial to Investigate the Safety and Efficacy of ABCB5-positive Mesenchymal Stromal Cells (ABCB5+ MSCs) on Epidermolysis Bullosa (EB)

This study is testing a new stem cell treatment to see if it can help improve skin healing for people with severe skin conditions called epidermolysis bullosa.

Quick facts

• Phase: Phase 3
• Study type: Interventional
• Enrollment: 74 (estimated)
• Sex: All
• Sponsor: RHEACELL GmbH & Co. KG (Industry-sponsored)
• Locations: 1 site (Salzburg)
• Trial ID: NCT05464381 on ClinicalTrials.gov

What this trial studies

This clinical trial investigates the safety and efficacy of allo-APZ2-OTS, an allogeneic stem cell therapy, administered intravenously to patients with recessive dystrophic epidermolysis bullosa (RDEB) and junctional epidermolysis bullosa (JEB). The study includes a placebo-controlled phase and an open-label arm for younger patients with RDEB. Participants will be monitored for improvements in wound healing and overall skin condition. The trial aims to provide a new treatment option for individuals suffering from these severe skin disorders.

Who should consider this trial

Good fit: Ideal candidates are individuals diagnosed with RDEB or JEB, aged under one year or with specific wound characteristics.

Not a fit: Patients with current tumor diseases or known allergies to the treatment components may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve wound healing and quality of life for patients with Epidermolysis Bullosa.

How similar studies have performed: While this approach is innovative, similar studies using stem cell therapies for skin conditions have shown promise, indicating potential for success.

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.

Where this trial is running

Salzburg

• EB-Haus Austria; Salzburger Landeskliniken (SALK) — Salzburg, Austria (Recruiting)

Study contacts

• Study coordinator: Cristina Daniele

Conditions Epidermolysis Bullosa