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

NCT05464381 | Active Not Recruiting Phase 3 DMC

• 1 other identifier: allo-APZ2-EB-III
• Study Type: interventional
• Target: 91
• Locations: 12 countries
• Sites: 16

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

Study Arms (2)

Verum

EXPERIMENTAL

Drug: allo-APZ2-OTS

Placebo

PLACEBO COMPARATOR

Drug: Placebo

Interventions

allo-APZ2-OTS DRUG

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

Verum

Placebo DRUG

Placebo

Placebo

Eligibility Criteria

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

You may qualify if:

• Male or female subjects with a diagnosis of RDEB or JEB confirmed by genetic testing or by a skin biopsy with immunofluorescence mapping (IFM). A minimal body weight of at least 5 kg is required;
• 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, 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:

• Signs and/or a history of skin precancerous and cancerous lesions at screening or any current tumor diseases, including squamous cell carcinoma and basal cell carcinoma;
• Known chronic lung disease;
• Clinically significant laboratory values for coagulation and thrombocytes at screening;
• Thromboembolic events of any grade in medical history;
• Immunoreactions of any grade in medical history;
• 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;
• Employees of the sponsor, or employees or relatives of the investigator.

Sponsors & Collaborators

• RHEACELL GmbH & Co. KG: lead

Study Sites (16)

Sanatorio Mater Dei

Buenos Aires, Argentina

Location

EB-Haus Austria; Salzburger Landeskliniken (SALK)

Salzburg, 5020, Austria

Location

Fundacion Debra Chile

Santiago, Chile

Location

Universitaetsklinik fuer Dermatologie und Allergologie

Nice, France

Location

Hospital Necker-Enfants Malades

Paris, France

Location

Service de Dermatologie Hospital Saint-Louis

Paris, France

Location

Andreas Syggros Hospital of Cutaneous Venereal Diseases

Athens, Greece

Location

Venereal & Skin Diseases Hospital of Thessaloniki

Thessaloniki, Greece

Location

Semmelweis Egyetem

Budapest, Hungary

Location

UOSD Pediatria-Alta Intensita di Curà, Ospedale Maggiore

Milan, Italy

Location

Ospedale Pediatrico Bambin Gesu

Roma, Italy

Location

Medical Concierge Centrum Medyczne

Warsaw, Poland

Location

Centro Hospitalar e Universitario de Comibra HUC

Coimbra, Portugal

Location

Hospital La Paz

Madrid, Spain

Location

Department of Dermatology, National Cheng Kung University

Tainan, Taiwan

Location

Great Ormond Street Hospital (GOSH)

London, United Kingdom

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: July 5, 2022
• First Posted: July 19, 2022
• Study Start: June 13, 2023
• Primary Completion: August 29, 2025
• Study Completion (Estimated): January 1, 2027
• Last Updated: December 10, 2025

Record last verified: 2025-12

Locations

CL (1); PT (1); PL (1); TW (1); HU (1); IT (2); AU (1); GR (2); FR (3); AR (1); GB (1); ES (1)