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

NCT05838092 | Active Not Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: allo-APZ2-EB-III-01
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 2

Brief Summary

The aim of this clinical trial is to investigate the safety and efficacy of allo-APZ2-OTS administered intravenously to subjects with recessive dystrophic epidermolysis bullosa (RDEB) compared to placebo. An additional baseline-controlled open-label arm will be included to investigate the safety and efficacy of allo-APZ2-OTS administered intravenously to subjects with JEB and to RDEB subjects \< 1 year.

Conditions

Epidermolysis Bullosa

Outcome Measures

Primary Outcomes (1)

• Proportion of subjects with complete target wound closure

  Proportion of subjects with complete healing of target wound, determined by the investigator, as compared to baseline

  6 Months

Secondary Outcomes (1)

• Time to complete target wound closure from baseline

  6 Months

Study Arms (2)

Verum

EXPERIMENTAL

Drug: allo-APZ2-OTS

Placebo

PLACEBO COMPARATOR

Drug: Placebo

Interventions

Placebo DRUG

Placebo

Placebo

allo-APZ2-OTS DRUG

Allogeneic dermal ABCB5-positive Mesenchymal Stromal Cells (ABCB5+ MSCs)

Verum

Eligibility Criteria

• Age: 6 Months+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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
• Subject is eligible to participate in this clinical trial based on general health condition;
• Subject with a target wound meeting the following criteria: 5-50 cm2, ≥ 21 days and \< 9 months, no signs of acute infection;
• Patient/legal representative understands the nature of the procedure and provide written informed consent/assent prior to any clinical trial procedure;
• 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.

You may not qualify if:

• Any current tumor diseases, including squamous cell carcinoma and basal cell carcinoma;
• Any known allergies to components of the IP or premedication;
• Patient/legal representative anticipated to be unwilling or unable to comply with the requirements of the protocol;
• Pregnant or lactating women;
• Current or previous (within 30 days of screening) treatment with another IP, or participation and/or under follow-up in another interventional clinical trial;
• Previous participation in this clinical trial (except for screening failures);
• 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
• Employees of the sponsor, or employees or relatives of the investigator.

Sponsors & Collaborators

• RHEACELL GmbH & Co. KG: lead

Study Sites (2)

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

Masonic Cancer Center and Medical Center Minneapolis

Minneapolis, Minnesota, 55455, United States

Location

MeSH Terms

Conditions

Epidermolysis Bullosa

Condition Hierarchy (Ancestors)

Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Skin Diseases; Skin and Connective Tissue Diseases; Skin Diseases, Vesiculobullous

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 20, 2023
• First Posted: May 1, 2023
• Study Start: October 18, 2023
• Primary Completion: August 29, 2025
• Study Completion (Estimated): July 1, 2026
• Last Updated: December 10, 2025

Record last verified: 2025-12

Locations

US (2)