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Severe Thalassemia clinical trials

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NCT ID: NCT01005576 Completed - Severe Thalassemia Clinical Trials

Reduced Intensity Transplant Conditioning Regimen for Severe Thalassemia

URTH
Start date: January 2010
Phase: Phase 2
Study type: Interventional

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.