NCT01383434

Bone Marrow Transplant Trial for Patients With Refractory Severe Aplastic Anemia

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

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 4 (actual)
• Sponsor: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins · Academic / Other
• Sex: All
• Age: 6 Months – 70 Years
• Healthy volunteers: Not accepted

Summary

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.

Conditions

• Severe Aplastic Anemia

Interventions

Type	Name	Description
PROCEDURE	Bone Marrow Transplant	Matched sibling, haploidentical or matched unrelated donor bone marrow transplant following chemotherapy

Timeline

• Start date: 2011-06-01
• Primary completion: 2014-08-01
• Completion: 2014-08-01
• First posted: 2011-06-28
• Last updated: 2016-10-13

Locations

1 site across 1 country: United States