drugset / Trial / NCT00176852
Stem Cell Transplant for Hemoglobinopathy
Phase 2/3
Completed
22 enrolled
Masonic Cancer Center, University of Minnesota
National Marrow Donor Program · collab
Non-randomizedParallel-groupOpen-labelTreatment
Summary
This study tests the clinical outcomes of one of two preparative regimens (determined by available donor source) in patients with non-malignant hemoglobinopathies. The researchers hypothesize that these regimens will have a positive effect on post transplant engraftment and the incidence of graft-versus-host-disease. Regimen A2 has replaced Regimen A in this study. Two patients were treated on Regimen A but did not have evidence of initial engraftment thus triggering the stopping rule for that arm of this study.
Timeline
- Start
- 2002-06
- Primary completion
- 2014-03
- Completion
- 2020-01
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | ANTILYMPHOCYTE IMMUNOGLOBULIN (HORSE) | Protein / enzyme biologic | 30 mg/kg | Intravenous |
| Subject | Alemtuzumab | Monoclonal antibody | 0.2 mg/kg | — |
| Subject | Busulfan | Small molecule | 0.8 mg/kg | Intravenous |
| Subject | Cyclophosphamide | Other / unclassified | 50 mg/kg | Intravenous |
| Subject | Fludarabine | Small molecule | 35 mg/m2 | Intravenous |
| Background | GCSF | Unknown | 5 ug/kg | — |