drugset / Trial / NCT00775931
Allogeneic Transplantation For Severe Osteopetrosis
Non-randomizedParallel-groupOpen-labelTreatment
Summary
The purpose of this research is to explore what we believe may be a safer and more effective means of performing stem cell transplantation in patients with Osteopetrosis, using chemotherapy and radiation designed to bring about engraftment and lessen transplant mortality. Prior multi-institutional data in past studies found that approximately 30% of Osteopetrosis patients do not engraft. Therefore, in this study, we utilize a reduced intensity design of pre-transplant drugs to try to make transplants safer for this disease, as well as to provide a second infusion of stem cells in patients with matched related or unrelated donors.
Timeline
- Start
- 2008-08
- Primary completion
- 2013-06
- Completion
- 2013-06
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Comparator | Busulfan | Small molecule | 0.8 mg/kg | Intravenous |
| Comparator | Busulfan | Small molecule | 1.1 mg/kg | Intravenous |
| Comparator | Cyclophosphamide | Other / unclassified | 50 mg/kg | Intravenous |
| Comparator | Fludarabine | Small molecule | 35 mg/m2 | Intravenous |
| Comparator | Alemtuzumab | Monoclonal antibody | 0.3 mg/kg | Subcutaneous |