At a glance
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Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia
In Brief
A clinical study evaluating Alemtuzumab, Fludarabine, and 4 other interventions for Dyskeratosis Congenita and Aplastic Anemia. Completed, enrolled 61 participants across 1 site.
Detailed Summary
Fludarabine-based preparative regimen followed by an allogeneic hematopoietic stem cell transplant using related or unrelated donor in persons 0-70 years of age diagnosed with dyskeratosis congenita or severe aplastic anemia who have bone marrow failure characterized by a requirement for red blood cell and platelet transfusions. Three different preparative regimens are included based on disease and donor type.
Study Details
Timeline
Interventions
Alemtuzumab 0.2 mg/kg IV over 2 hours on days -10 to -6 from transplant.
Fludarabine 40 mg/m2 IV over 1 hour on days -6 to -2 from transplant.
Cyclophosphamide 50 mg/kg IV over 2 hours on day -7 from transplant.
TBI 200 cGy as a single fraction on day -1 from transplant.
Stem cell transplant on day 0.
ATG (Thymoglobulin - Rabbit ) 3 mg/kg IV on days -5 to -3 from stem cell transplant.