Reduced Intensity Transplant Conditioning Regimen for Severe Thalassemia
Status:
Completed
Trial end date:
2014-07-01
Target enrollment:
Participant gender:
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.
Phase:
Phase 2
Details
Lead Sponsor:
Washington University School of Medicine
Collaborators:
HealthCore-NERI New England Research Institutes Pediatric Blood and Marrow Transplant Consortium