Minneapolis, Minnesota 55455


This is a single-institution, phase II study to determine the event-free survival at 1 year post allogeneic transplant and serial mesenchymal stem cell (MSC) infusions from a related donor (HLA identical, mismatched or haploidentical) or matched unrelated donor for the biochemical correction of severe epidermolysis bullosa (EB).


Inclusion Criteria: - Diagnosis of severe form of EB characterized by collagen, laminin, integrin, keratin or plakin deficiency (by immunofluorescence staining with protein specific antibodies or Western blotting and by mutation analysis). - Adequate organ function within 4 weeks of study registration defined as: - Renal: glomerular filtration rate within normal range for age - Hepatic: Hepatic: bilirubin, AST/ALT, ALP < 5 x upper limit of normal - Pulmonary: adequate pulmonary function in the opinion of the enrolling investigator - Cardiac: left ventricular ejection fraction ≥ 45%, normal EKG or approved by Cardiology for transplant - Sexually active participants must agree to use adequate birth control for the during the study period (from before the start of the preparative chemotherapy through 1 year post-transplant) - Available donor per section 5: targeted MFI < 1,000 (MFI exceeding 1000 must be approved by the PI and treatment team.) - Voluntary written consent - adult or parent (with information sheet for minors, if applicable) prior to any research related procedures or treatment Exclusion Criteria: - beta 3 laminin JEB mutants - Active untreated systemic infection at time of transplantation (including active infection with Aspergillus or other mold within 30 days) - History of HIV infection - Evidence of squamous cell carcinoma - Pregnant or breast feeding. Females of child-bearing potential must have a negative pregnancy test prior to study registration as the agents administered in this study are Pregnancy Category C and D.



Primary Contact:

Principal Investigator
Jakub Tolar, MD, PhD
Masonic Cancer Center, University of Minnesota

Kim Nelson, RN
Phone: 612-273-2925
Email: knelso62@fairview.org

Backup Contact:


Location Contact:

Minneapolis, Minnesota 55455
United States

Kim Nelson, RN
Phone: 612-273-2925
Email: knelso62@fairview.org

Site Status: Recruiting

Data Source: ClinicalTrials.gov

Date Processed: November 18, 2019

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