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

Sponsors

Lead Sponsor: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Source Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Brief 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.

Overall Status Terminated
Start Date June 2011
Completion Date August 2014
Primary Completion Date August 2014
Phase Phase 2
Study Type Interventional
Primary Outcome
Measure Time Frame
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 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 1 year
Enrollment 4
Condition
Intervention

Intervention Type: Procedure

Intervention Name: Bone Marrow Transplant

Description: Matched sibling, haploidentical or matched unrelated donor bone marrow transplant following chemotherapy

Eligibility

Criteria:

Inclusion Criteria:

- Patients who have refractory or relapsed SAA following treatment with one or more immunosuppressive regimens.

- Age 6 months - 70 years

- Patients must meet medical criteria for myeloablative BMT

- Patients or their parents/guardians and donors must be able to sign consent forms.

- Patients must be geographically accessible and willing to participate in all stages of treatment.

Exclusion Criteria:

- Poor cardiac function: left ventricular ejection fraction <45% as determined by MUGA or ECHO.

- Poor pulmonary function: FEV1 and FVC <50% predicted.

- Poor renal function

- Positive leukocytotoxic crossmatch

- Women of childbearing potential who currently are pregnant (Β-HCG+) or who are not practicing adequate contraception

- Uncontrolled viral, bacterial, or fungal infections

Gender: All

Minimum Age: 6 Months

Maximum Age: 70 Years

Healthy Volunteers: No

Overall Official
Location
Facility: The Sidney Kimmel Comprehensive Cancer Center
Location Countries

United States

Verification Date

October 2016

Responsible Party

Type: Sponsor

Has Expanded Access No
Condition Browse
Study Design Info

Allocation: N/A

Intervention Model: Single Group Assignment

Primary Purpose: Treatment

Masking: None (Open Label)

Source: ClinicalTrials.gov