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
9
Allogeneic dermal ABCB5-positive Mesenchymal Stromal Cells (ABCB5+ MSCs)
Placebo
Children's Hospital Colorado
Aurora, Colorado, United States
Masonic Cancer Center and Medical Center Minneapolis
Minneapolis, Minnesota, United States
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
Time to complete target wound closure from baseline
Time frame: 6 Months
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