Bone Marrow Transplant Trial for Patients With Refractory Severe Aplastic Anemia
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT01383434|
Recruitment Status : Terminated (New revised SAA BMT study opened to take over this study)
First Posted : June 28, 2011
Last Update Posted : October 13, 2016
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.
|Condition or disease||Intervention/treatment||Phase|
|Severe Aplastic Anemia||Procedure: Bone Marrow Transplant||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||4 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
|Study Start Date :||June 2011|
|Actual Primary Completion Date :||August 2014|
|Actual Study Completion Date :||August 2014|
- Procedure: Bone Marrow Transplant
Matched sibling, haploidentical or matched unrelated donor bone marrow transplant following chemotherapy
- 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 ]
- 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 ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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|