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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
91
Allogeneic dermal ABCB5-positive Mesenchymal Stromal Cells (ABCB5+ MSCs)
Placebo
Sanatorio Mater Dei
Buenos Aires, Argentina
EB-Haus Austria; Salzburger Landeskliniken (SALK)
Salzburg, Austria
Fundacion Debra Chile
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
Time frame: 6 months
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Santiago, Chile
Universitaetsklinik fuer Dermatologie und Allergologie
Nice, France
Hospital Necker-Enfants Malades
Paris, France
Service de Dermatologie Hospital Saint-Louis
Paris, France
Andreas Syggros Hospital of Cutaneous Venereal Diseases
Athens, Greece
Venereal & Skin Diseases Hospital of Thessaloniki
Thessaloniki, Greece
Semmelweis Egyetem
Budapest, Hungary
UOSD Pediatria-Alta Intensita di Curà, Ospedale Maggiore
Milan, Italy
...and 6 more locations