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Bone Marrow Transplant Trial for Patients With Refractory Severe Aplastic Anemia

Sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins (Other)
Overall Status
Terminated
CT.gov ID
NCT01383434
Collaborator
(none)
4
Enrollment
1
Location
38
Duration (Months)
0.1
Patients Per Site Per Month

Study Details

Study Description

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
  • Procedure: Bone Marrow Transplant
 Phase 2

Study Design

Study Type:
Interventional
Actual Enrollment :
4 participants
Allocation:
N/A
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 :
Jun 1, 2011
Actual Primary Completion Date :
Aug 1, 2014
Actual Study Completion Date :
Aug 1, 2014

Outcome Measures

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 [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 [1 year]

Eligibility Criteria

Criteria

Ages Eligible for Study:
6 Months to 70 Years
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
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

Locations

Site City State Country Postal Code
1 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

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
ClinicalTrials.gov Identifier:
NCT01383434
Other Study ID Numbers:
  • J10117
  • NA_00042991
First Posted:
Jun 28, 2011
Last Update Posted:
Oct 13, 2016
Last Verified:
Oct 1, 2016
Additional relevant MeSH terms:
Anemia
Anemia, Aplastic

Study Results

No Results Posted as of Oct 13, 2016