NCT02162420

Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia

• Status: Completed
• Phase: N/A
• Study type: Interventional
• Enrollment: 61 (actual)
• Sponsor: Masonic Cancer Center, University of Minnesota · Academic / Other
• Sex: All
• Age: 0 Years – 70 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Dyskeratosis Congenita
• Aplastic Anemia

Interventions

Type	Name	Description
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.
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.
BIOLOGICAL	Stem Cell Transplant	Stem cell transplant on day 0.
DRUG	Anti-thymocyte globulin	ATG (Thymoglobulin - Rabbit ) 3 mg/kg IV on days -5 to -3 from stem cell transplant.

Timeline

• Start date: 2015-01-10
• Primary completion: 2024-08-08
• Completion: 2025-03-11
• First posted: 2014-06-12
• Last updated: 2025-05-08
• Results posted: 2025-05-08

Locations

1 site across 1 country: United States