MT2015-20: Biochemical Correction of Severe EB by Allo HSCT and Serial Donor MSCs

  • End date
    Dec 25, 2024
  • participants needed
  • sponsor
    Masonic Cancer Center, University of Minnesota
Updated on 25 March 2022
stem cell transplantation
mycophenolate mofetil
ejection fraction
mutation analysis


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).

Condition Epidermolysis Bullosa
Treatment cyclophosphamide, mycophenolate mofetil, busulfan, Fludarabine, Tacrolimus, Total body irradiation, Thymoglobulin, Bone marrow infusion, Donor mesenchymal stem cell infusions
Clinical Study IdentifierNCT02582775
SponsorMasonic Cancer Center, University of Minnesota
Last Modified on25 March 2022


Yes No Not Sure

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
Available donor per section 5: targeted MFI < 1,000 (MFI exceeding 1000 must be approved by the PI and treatment team.)
the study period (from before the start of the preparative chemotherapy
through 1 year post-transplant)
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
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