MT2009-09: Biochemical Correction of Severe Epidermolysis Bullosa by Allogeneic Stem Cell Transplantation and "Off-the-shelf" Mesenchymal Stem Cells

NCT01033552 Phase 1/Phase 2 COMPLETED Results posted

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.

Details

Lead sponsor	Masonic Cancer Center, University of Minnesota
Phase	Phase 1/Phase 2
Status	COMPLETED
Enrolment	32
Start date	2010-01
Completion	2021-08-12

Conditions

• Epidermolysis Bullosa

Interventions

• Cyclophosphamide
• Fludarabine
• Anti-thymocyte globulin
• Myeloablative Busulfan
• Mesenchymal stem cell transplantation
• Total body irradiation
• Bone marrow or umbilical cord blood (UCG) stem cell transplantation

Primary outcomes

• Percentage of Participants With Event-free Survival — 1 year and 2 Years Post-transplant
  Event-free survival rate, with an event defined as death or failure to have a demonstrable increase in collagen, laminin, intergrin, keratin or plakin deposition. Assessed at follow up appointments through questionnaire and patient samples.

Countries

United States