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Bone Marrow Transplant Trial for Patients With Refractory Severe Aplastic Anemia

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ClinicalTrials.gov Identifier: NCT01383434
Recruitment Status : Terminated (New revised SAA BMT study opened to take over this study)
First Posted : June 28, 2011
Last Update Posted : October 13, 2016
Sponsor:
Information provided by (Responsible Party):
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Study Description
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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.


Condition or disease Intervention/treatment Phase
Severe Aplastic Anemia Procedure: Bone Marrow Transplant Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 4 participants
Intervention Model: Single Group Assignment
Masking: None (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
Study Start Date : June 2011
Actual Primary Completion Date : August 2014
Actual Study Completion Date : August 2014

Resource links provided by the National Library of Medicine


Arms and Interventions
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Intervention Details:
  • Procedure: Bone Marrow Transplant
    Matched sibling, haploidentical or matched unrelated donor bone marrow transplant following chemotherapy

Outcome Measures
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Primary Outcome Measures :
  1. 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 ]
  2. 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 ]

Eligibility Criteria
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Ages Eligible for Study:   6 Months to 70 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
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
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01383434


Locations
United States, Maryland
The Sidney Kimmel Comprehensive Cancer Center
Baltimore, Maryland, United States, 21031
Sponsors and Collaborators
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
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
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Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Responsible Party: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
ClinicalTrials.gov Identifier: NCT01383434     History of Changes
Other Study ID Numbers: J10117
NA_00042991 ( Other Identifier: The Johns Hopkins University )
First Posted: June 28, 2011    Key Record Dates
Last Update Posted: October 13, 2016
Last Verified: October 2016

Additional relevant MeSH terms:
Anemia
Anemia, Aplastic
Hematologic Diseases
Bone Marrow Diseases