drugset / Trial / NCT01005576
Reduced Intensity Transplant Conditioning Regimen for Severe Thalassemia
Phase 2
Completed
21 enrolled
Washington University School of Medicine
Carelon Research · collabPediatric Blood and Marrow Transplant Consortium · collab
NaSingle-groupOpen-labelTreatment
Summary
This study is being done to determine if blood cell transplants, with either bone marrow or cord blood from unrelated donors, are effective in children with severe thalassemia and if this treatment approach has acceptable risks and side effects. This study includes a preparative regimen with Hydroxyurea, Alemtuzumab, Fludarabine, Thiotepa and Melphalan that provides intense host immunosuppression without myeloablation. The primary hypothesis is that this regimen will promote stable engraftment of unrelated donor hematopoietic cells, support normal erythropoiesis, and result in an event free survival of \> 75% of children with thalassemia major.
Timeline
- Start
- 2010-01
- Primary completion
- 2014-04
- Completion
- 2014-07
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Alemtuzumab | Monoclonal antibody | 20 mg | Intravenous |
| Subject | Fludarabine | Small molecule | 30 mg/m2 | Intravenous |
| Subject | Hydroxyurea | Other / unclassified | 30 mg/kg | Oral |
| Subject | Melphalan | Small molecule | 140 mg/m2 | Intravenous |
| Subject | Thiotepa | Other / unclassified | 8 mg/kg | Intravenous |