|Secondary Protocol No.
||MT2009-09 Biochemical Correction of Severe Epidermolysis Bullosa by Allogeneic Stem Cell Transplantation
||To determine safety and efficacy of stem cell transplant in treatment of severe epidermolysis bullosa.
||Patients will receive a chemotherapeutic regimen followed by marrow stem cell and mesenchymal stem cell transplant.
||This is a study in patients with epidermolysis bullosa to test the effectiveness of bone marrow or umbilical cord blood from a healthy unaffected donor to correct the collagen, laminin, integrin or plakin deficiency and reduce the skin fragility characteristic of severe forms of EB. The study will also determine whether mesenchymal stem cells from a healthy donor will enhance the safety and effectiveness of the transplant.
||Patient must have a diagnosis of severe epidermolysis bullosa.
|Applicable Disease Sites
||Blood and Marrow Transplant
||BUSULFAN (MYLERAN) [SUSPENDED]
CYCLOPHOSPHAMIDE (CYTOXAN, NEOSAR)
FILGRASTIM (G-CSF, NEUPOGEN) [SUSPENDED]
Mesenchymal Stem Cells (MSC)
Total Body irradiation (TBI)
||Masonic Cancer Center
||Patients must be between the ages of 0 and 25 years of age. All must have adequate organ function and have an available healthy related or unrelated donor. Anyone with an active infection at time of transplant, history of HIV infection, had prior transplantation with donor skin or evidence of squamous cell carcinoma are not eligible. Women who are pregnant or nursing are not eligible. A signed assent form, informed consent and HIPAA authorization are required.