Clinical Trial: Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia

Brief 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.

Detailed Summary:
Sponsor: Masonic Cancer Center, University of Minnesota

Current Primary Outcome: Incidence of neutrophil engraftment [ Time Frame: day 42 ]

Incidence of neutrophil engraftment by day 42.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Incidence of regimen related mortality [ Time Frame: Day 100 ]
    Incidence of regimen related mortality by day 100.
  • Incidence of acute graft-versus-host disease [ Time Frame: Day 100 ]
    Incidence of acute graft-versus-host disease by day 100.
  • Incidence of chronic graft-versus-host disease [ Time Frame: 6 months ]
    Incidence of chronic graft-versus-host disease by 6 months and 1 year


Original Secondary Outcome: Same as current

Information By: Masonic Cancer Center, University of Minnesota

Dates:
Date Received: June 10, 2014
Date Started: January 2015
Date Completion: July 2024
Last Updated: August 15, 2016
Last Verified: August 2016