Bone Marrow Transplant Trial for Patients With Refractory Severe Aplastic Anemia

This study has been terminated.

(New revised SAA BMT study opened to take over this study)

Sponsor:

Information provided by (Responsible Party):

Sidney Kimmel Comprehensive Cancer Center

ClinicalTrials.gov Identifier:

NCT01383434

First received: June 22, 2011

Last updated: October 1, 2014

Last verified: October 2014

History of Changes

Purpose

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	Intervention	Phase
Severe Aplastic Anemia	Procedure: Bone Marrow Transplant	Phase 2


Study Type:	Interventional
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

Resource links provided by NLM:

MedlinePlus related topics: Anemia Aplastic Anemia Bone Marrow Transplantation

Genetic and Rare Diseases Information Center resources: Aplastic Anemia

U.S. FDA Resources

Further study details as provided by Sidney Kimmel Comprehensive Cancer Center:

Primary Outcome Measures:

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 ]


Enrollment:	4
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)

Intervention Details:

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

Eligibility


Ages Eligible for Study:	6 Months to 70 Years (Child, Adult, Senior)
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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

Contacts and Locations

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01383434

Locations


United States, Maryland
The Sidney Kimmel Comprehensive Cancer Center
Baltimore, Maryland, United States, 21031

Sponsors and Collaborators

Sidney Kimmel Comprehensive Cancer Center

Investigators


Principal Investigator:	Robert A Brodsky, MD	The Johns Hopkins University
Principal Investigator:	Amy Dezern, MD	The JOhns Hopkins Sydney Kimmel Comprehensive Cancer Center

More Information


Responsible Party:	Sidney Kimmel Comprehensive Cancer Center
ClinicalTrials.gov Identifier:	NCT01383434 History of Changes
Other Study ID Numbers:	J10117 NA_00042991
Study First Received:	June 22, 2011
Last Updated:	October 1, 2014
Health Authority:	United States: Institutional Review Board

Additional relevant MeSH terms:


Anemia Anemia, Aplastic Hematologic Diseases Bone Marrow Diseases

ClinicalTrials.gov processed this record on September 27, 2016

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