Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia | Aplastic Anemia and MDS International Foundation

Clinical Trial: NCT02162420

Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia
For more details on this clinical trial, including contact information, please see this trial’s listing on clinicaltrials.gov:
Purpose: 

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.

Status: 
Recruiting
Study Date: 
Tue, 06/10/2014
Bone Marrow Disease(s): 
aplastic anemia
Intervention: 
Drug: Alemtuzumab Alemtuzumab 0.2 mg/kg IV over 2 hours on days -10 to -6 from transplant. Drug: Fludarabine Fludarabine 40 mg/m2 IV over 1 hour on days -6 to -2 from transplant. Other Name: Fludara Drug: Cyclophosphamide Cyclophosphamide 50 mg/kg IV over 2 hours on day -7 from transplant. Radiation: Total Body Irradiation TBI 200 cGy as a single fraction on day -1 from transplant. Other Name: TBI Biological: Stem Cell Transplant Stem cell transplant on day 0. Other Name: HSCT Drug: Anti-thymocyte globulin ATG (Thymoglobulin - Rabbit ) 3 mg/kg IV on days -5 to -3 from stem cell transplant. Other Name: Rabbit ATG