Severe Thalassemia Clinical Trial
Official title:
A Pilot Trial of Unrelated Donor Hematopoietic Cell Transplantation for Children With Severe Thalassemia Using a Reduced Intensity Conditioning Regimen (The URTH Trial)
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.
n/a