drugset / Trial / NCT01238328
Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis
Non-randomizedSingle-groupOpen-labelTreatment
Summary
The purpose of this study is to evaluate the efficacy and side effects of donor hematopoietic cells using chemotherapy regimen without total-body irradiation in children undergoing a hematopoietic stem cell transplant for Mucopolysaccharidosis. The blood stem cells will be derived from either related donor or unrelated umbilical cord blood or haploidentical donor.
Timeline
- Start
- 2009-09
- Primary completion
- 2012-09
- Completion
- 2012-12
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Background | Antithymocyte globulin (ATG) rabbit | Unknown | 10 mg/kg | — |
| Background | Busulfan | Small molecule | 16 mg/kg | Oral |
| Background | Busulfan | Small molecule | 20 mg/kg | Oral |
| Background | Cyclophosphamide | Other / unclassified | 40 mg/kg | — |
| Background | Cyclophosphamide | Other / unclassified | 200 mg/kg | Intravenous |
| Background | Cyclosporine | Peptide | 9 mg/kg | Intravenous |
| Background | Fludarabine | Small molecule | 160 mg/m2 | — |
| Background | MTX | Unknown | 6 mg/m2 | Intravenous |