Bone Marrow Transplant Trial for Patients With Refractory Severe Aplastic Anemia

Severe Aplastic Anemia Clinical Trial

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT01383434
• Other study ID #: J10117
• Secondary ID: NA_00042991
• Status: Terminated
• Phase: Phase 2
• First received: June 22, 2011
• Last updated: October 12, 2016
• Start date: June 2011
• Est. completion date: August 2014

Study information

• Verified date: October 2016
• Source: Sidney Kimmel Comprehensive Cancer Center
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Institutional Review Board
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 4
• Est. completion date: August 2014
• Est. primary completion date: August 2014
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 6 Months to 70 Years

Eligibility

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 (?-HCG+) or who are not practicing adequate contraception

• Uncontrolled viral, bacterial, or fungal infections

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Anemia
• Anemia, Aplastic
• Severe Aplastic Anemia

Intervention

Procedure:
• Bone Marrow Transplant
Matched sibling, haploidentical or matched unrelated donor bone marrow transplant following chemotherapy

Locations

Country	Name	City	State
United States	The Sidney Kimmel Comprehensive Cancer Center	Baltimore	Maryland

Sponsors (1)

Lead Sponsor	Collaborator
Sidney Kimmel Comprehensive Cancer Center

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Yes
Primary	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	Yes