drugset / Trial / NCT00881556
Allogeneic Stem Cell Transplantation (ALLOSCT) in Recessive Dystrophic Epidermolysis Bullosa (RDEB)
NaSingle-groupOpen-labelTreatment
Summary
Reduced Intensity Conditioning (RIC) and Allogeneic Stem Cell Transplantation (AlloSCT) from family-related donors and unrelated cord blood (UCB) donors will be safe and well tolerated in selected patients with RDEB. To determine the event-free survival (EFS) and overall survival (OS) following RIC consisting of busulfan/fludarabine/alemtuzumab (BFA) and AlloSCT in selected patients with RDEB.
Timeline
- Start
- 2009-08-20
- Primary completion
- 2015-09
- Completion
- 2015-09
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Background | Alemtuzumab | Monoclonal antibody | 20 mg/m2 | Intravenous |
| Background | Busulfan | Small molecule | 4 mg/kg | Intravenous |
| Background | Fludarabine | Small molecule | 30 mg/m2 | Intravenous |
| Background | Lorazepam | Small molecule | 0.02 mg/kg | — |
| Background | Lorazepam | Small molecule | 0.05 mg/kg | — |
| Background | Palifermin | Protein / enzyme biologic | 60 ug/kg | — |
| Background | Tacrolimus | Small molecule | 0.03 mg/kg | — |