drugset / Trial / NCT01033552
Biochemical Correction of Severe EB by Allo HSCT and "Off-the-shelf" MSCs
Non-randomizedParallel-groupOpen-labelTreatment
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.
Timeline
- Start
- 2010-01
- Primary completion
- 2021-08-12
- Completion
- 2021-08-12
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | ANTILYMPHOCYTE IMMUNOGLOBULIN (HORSE) | Protein / enzyme biologic | 30 mg/kg | — |
| Subject | ANTILYMPHOCYTE IMMUNOGLOBULIN (HORSE) | Protein / enzyme biologic | 90 mg/kg | — |
| Subject | Busulfan | Small molecule | — | — |
| Subject | Cyclophosphamide | Other / unclassified | 50 mg/kg | Intravenous |
| Subject | Cyclophosphamide | Other / unclassified | 200 mg/kg | Intravenous |
| Subject | Fludarabine | Small molecule | 40 mg/m2 | Intravenous |
| Subject | Fludarabine | Small molecule | 75 mg/m2 | Intravenous |
| Subject | Fludarabine | Small molecule | 500 mg/m2 | Intravenous |