drugset / Trial / NCT00176917
Stem Cell Transplantation for Hurler
NaSingle-groupOpen-labelTreatment
Summary
The purpose of this study is to determine the safety and engraftment of donor hematopoietic cells using this conditioning regimen in patients undergoing a hematopoietic (blood forming) cell transplant for Hurler syndrome, Maroteaux Lamy syndrome, Mannosidosis, or I-cell disease.
Timeline
- Start
- 1999-05
- Primary completion
- 2008-05
- Completion
- 2010-05
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Background | ATG | Unknown | — | Intravenous |
| Background | Busulfan | Small molecule | — | Intravenous |
| Background | Cyclophosphamide | Other / unclassified | — | Intravenous |