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Allogeneic Hematopoietic Stem Cell Transplant for Epidermolysis Bullosa

2014-08-27 03:17:13 | BioPortfolio

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.

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Conditions

Epidermolysis Bullosa

Intervention

Busulfan, Cyclophosphamide, Fludarabine, Granulocyte colony-stimulating factor, Cyclosporine A, Mycophenolate mofetil, Hematopoietic stem cell transplant, Mesenchymal stem cell transplant

Location

University of Minnesota Masonic Cancer Center and Medical Center
Minneapolis
Minnesota
United States
55455

Status

Recruiting

Source

Masonic Cancer Center, University of Minnesota

Results (where available)

View Results

Links

Published on BioPortfolio: 2014-08-27T03:17:13-0400

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Medical and Biotech [MESH] Definitions

Glycoproteins found in a subfraction of normal mammalian plasma and urine. They stimulate the proliferation of bone marrow cells in agar cultures and the formation of colonies of granulocytes and/or macrophages. The factors include INTERLEUKIN-3; (IL-3); GRANULOCYTE COLONY-STIMULATING FACTOR; (G-CSF); MACROPHAGE COLONY-STIMULATING FACTOR; (M-CSF); and GRANULOCYTE-MACROPHAGE COLONY-STIMULATING FACTOR; (GM-CSF).

Receptors that bind and internalize GRANULOCYTE COLONY-STIMULATING FACTOR. Their MW is believed to be 150 kD. These receptors are found mainly on a subset of myelomonocytic cells.

Granulocyte colony stimulating factors prepared by recombinant DNA technology.

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