Biochemical Correction of Severe EB by Allo HSCT and "Off-the-shelf" MSCs

  • End date
    Oct 25, 2021
  • participants needed
  • sponsor
    Masonic Cancer Center, University of Minnesota
Updated on 26 January 2021
stem cell transplantation
mycophenolate mofetil
ejection fraction
mutation analysis
allogeneic hematopoietic stem cell transplant
antigen level


This is an open-label, single institution, phase II study in patients with epidermolysis bullosa (EB). The underlying hypothesis is that the infusion of bone marrow or umbilical cord blood from a healthy unaffected donor will correct the collagen, laminin, integrin, or plakin deficiency and reduce the skin fragility characteristic of severe forms of EB. A secondary hypothesis is that mesenchymal stem cells from a healthy donor will enhance the safety and efficacy of the allogeneic hematopoietic stem cell transplant as well as serve as a source of renewable cells for the treatment of focal areas of residual blistering.


The primary objective of this study is to estimate the event-free survival rate by 1 year post-transplant with an event defined as a death or failure to have a demonstrable increase in collagen, laminin, integrin, keratin or plakin deposition by 1 year post-transplant or other biochemical, structural or physical measure of improvement.

The secondary objectives of this study are to i) determine the incidence of transplant-related mortality (TRM) at 180 days; ii) describe the pattern of biochemical improvement as measured by an increase in protein expression (collagen, laminin, integrin, keratin or plakin) and related structural and physical changes; iii) describe health quality of life at day 365 and 730 as compared to pretreatment results; iv) describe the pattern and durability of HSC and third party MSC engraftment in the skin; v) determine the probability of survival at 1 year.

Patients with severe epidermolysis bullosa will be screened to meet the eligibility requirements, related or unrelated donor marrow or UCB will be infused, and subjects will be followed for a minimum of 5 years after stem cell transplant. A target accrual of 75 subjects over 5 years will be recruited to the study.

Condition Epidermolysis Bullosa
Treatment anti-thymocyte globulin, cyclophosphamide, mycophenolate mofetil, Fludarabine, Total body irradiation, Cyclosporine A, Mesenchymal stem cell transplantation, Mesenchymal stem cell transplantation, Bone marrow or umbilical cord blood (UCG) stem cell transplantation
Clinical Study IdentifierNCT01033552
SponsorMasonic Cancer Center, University of Minnesota
Last Modified on26 January 2021


Yes No Not Sure

Inclusion Criteria

Is your age less than or equal to 25 yrs?
Gender: Male or Female
Do you have Epidermolysis Bullosa?
Do you have any of these conditions: Do you have Epidermolysis Bullosa??
Do you have any of these conditions: Do you have Epidermolysis Bullosa??
Do you have any of these conditions: Do you have Epidermolysis Bullosa??
Diagnosis of severe form of epidermolysis bullosa (EB) characterized by collagen, laminin, integrin, keratin or plakin deficiency. Assessment criteria for severe EB
Documented collagen, laminin, integrin, keratin or plakin deficiency (by immunofluorescence staining with protein specific antibodies or Western blotting and by mutation analysis)
Adequate Organ Function Criteria
Renal: glomerular filtration rate within normal range for age
Hepatic: bilirubin, aspartate aminotransferase/alanine aminotransferase (AST/ALT), Alkaline phosphatase (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 electrocardiogram (EKG) or approved by Cardiology for transplant
Available Healthy HSC Donor (order of preference)
Related Donor (marrow or UCB)
HLA-A, B, C, DRB1 genotypic identical (sibling) donor
HLA-A, B, C, DRB1 phenotypic identical donor
8 HLA matched donor at HLA-A, B, C, DRB1
Unrelated Donor
HLA-A, B, C, DRB1 phenotypic identical donor
8 HLA matched donor at HLA-A, B, C, DRB1
HLA-A, B (antigen level) and DRB1 (allele level) matched donor
6 HLA matched donor at HLA-A, B, DRB1
6 HLA matched donor at HLA-A, B, DRB1
Voluntary written consent

Exclusion Criteria

Active systemic infection at time of transplantation (including active infection with Aspergillus or other mold within 30 days)
History of human immunodeficiency virus (HIV) infection
Evidence of squamous cell carcinoma
Donor has EB
Pregnancy females of child-bearing age must have a documented negative pregnancy test and agree to use contraception as a condition for enrollment
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