- ICH GCP
- US Clinical Trials Registry
- Clinical Trial 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
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.
Study Overview
Status
Conditions
Intervention / Treatment
Study Type
Enrollment (Actual)
Phase
- Phase 2
Contacts and Locations
Study Locations
-
-
Maryland
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Baltimore, Maryland, United States, 21031
- The Sidney Kimmel Comprehensive Cancer Center
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-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
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
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
What is the study measuring?
Primary Outcome Measures
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
Time Frame: 1 year
|
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
Time Frame: 1 year
|
1 year
|
Collaborators and Investigators
Investigators
- Principal Investigator: Robert A Brodsky, MD, The Johns Hopkins University
- Principal Investigator: Amy Dezern, MD, The JOhns Hopkins Sydney Kimmel Comprehensive Cancer Center
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- J10117
- NA_00042991 (Other Identifier: The Johns Hopkins University)
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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