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:
Sidney Kimmel Comprehensive Cancer Center
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
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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:
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:
-
Procedure: Bone Marrow Transplant
Matched sibling, haploidentical or matched unrelated donor bone marrow transplant following chemotherapy
Eligibility| Ages Eligible for Study: | 6 Months to 70 Years |
| 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
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
No publications provided
| 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 Bone Marrow Diseases Hematologic Diseases |
ClinicalTrials.gov processed this record on February 27, 2015