Biochemical Correction of Severe EB by Allo HSCT and "Off-the-shelf" MSCs
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- STATUS
- Recruiting
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- End date
- Oct 25, 2021
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- participants needed
- 75
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- sponsor
- Masonic Cancer Center, University of Minnesota
Summary
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.
Description
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.
Details
| 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 Identifier | NCT01033552 |
| Sponsor | Masonic Cancer Center, University of Minnesota |
| Last Modified on | 26 January 2021 |
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