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 for people with a severe skin condition called epidermolysis bullosa to see if it can help improve their symptoms.

Quick facts

• Phase: PHASE3
• Study type: Interventional
• Enrollment: 74 (estimated)
• Ages: 6 Months and up
• Sex: All
• Sponsor: RHEACELL GmbH & Co. KG (industry)
• Locations: 1 site (Minneapolis, Minnesota)
• Trial ID: NCT05838092 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). Participants will be compared to a placebo group to evaluate the treatment's effectiveness. Eligible candidates include individuals aged 6 months and older with confirmed RDEB and specific wound characteristics. The study aims to provide a new therapeutic option for this challenging skin condition.

Who should consider this trial

Good fit: Ideal candidates are males and females aged 6 months and older diagnosed with recessive dystrophic epidermolysis bullosa.

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: Other studies have explored stem cell therapies for skin conditions, showing promising results, but this specific approach is novel.

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.

Where this trial is running

Minneapolis, Minnesota

• Masonic Cancer Center and Medical Center Minneapolis — Minneapolis, Minnesota, United States (RECRUITING)

Study contacts

• Study coordinator: Cristina Daniele

Conditions: Epidermolysis Bullosa