Bone Marrow Transplant Trial for Patients With Refractory Severe Aplastic Anemia
Patients with severe, refractory aplastic anemia have a severe, life threatening disease in their bone marrow. Refractory disease means that disease has come back or not responded after receiving one or more immunosuppressive treatments. High dose chemotherapy followed by bone marrow transplantation (BMT) has been used to treat blood diseases like aplastic anemia but complications from Graft vs Host disease (GVHD) and graft failure have limited the survival for those patients.
Another study done here at Johns Hopkins has shown that in patients with other diseases (blood cancers) some immunosuppressive drugs given after the BMT has decreased how often patients had complications of GVHD and engraftment failure.
This research is being done to find if this approach will help patients with aplastic anemia who have failed other treatments will have better outcomes.
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Phase II Trial of Myeloablative Conditioning and Transplantation of HLA-matched, Partially HLA-mismatched, HLA-haploidentical or Matched Unrelated (MUD) Bone Marrow for Patients With Refractory Severe Aplastic Anemia|
- To estimate the overall survival (OS) at 1 year following HLA-matched, Partially HLA-mismatched, HLA-haploidentical or matched unrelated Bone Marrow for Patients with Refractory Severe Aplastic Anemia [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]
- To estimate the event-free survival (EFS) at 1 year following HLA-matched, Partially HLA-mismatched, HLA-haploidentical or matched unrelated Bone Marrow for Patients with Refractory Severe Aplastic Anemia [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]
|Study Start Date:||June 2011|
|Estimated Study Completion Date:||June 2015|
|Estimated Primary Completion Date:||June 2015 (Final data collection date for primary outcome measure)|
Procedure: Bone Marrow Transplant
Please refer to this study by its ClinicalTrials.gov identifier: NCT01383434
|United States, Maryland|
|The Sidney Kimmel Comprehensive Cancer Center|
|Baltimore, Maryland, United States, 21031|
|Principal Investigator:||Robert A Brodsky, MD||The Johns Hopkins University|
|Principal Investigator:||Amy Dezern, MD||The JOhns Hopkins Sydney Kimmel Comprehensive Cancer Center|