Bone Marrow Transplant Trial for Patients With Refractory Severe Aplastic Anemia

This study is currently recruiting participants. (see Contacts and Locations)
Verified March 2014 by Sidney Kimmel Comprehensive Cancer Center
Sponsor:
Information provided by (Responsible Party):
Sidney Kimmel Comprehensive Cancer Center
ClinicalTrials.gov Identifier:
NCT01383434
First received: June 22, 2011
Last updated: March 23, 2014
Last verified: March 2014
  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 ]

Estimated Enrollment: 25
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

Contacts
Contact: Robert A Brodsky, MD 410-502-2546 brodsro@jhmi.edu
Contact: Amy Dezern, MD

Locations
United States, Maryland
The Sidney Kimmel Comprehensive Cancer Center Recruiting
Baltimore, Maryland, United States, 21031
Contact: Amy Dezern, MD    410-502-7208    adezern1@jhmi.edu   
Contact: Robert Brodsky, MD    410-502-2546    brodsro@jhmi.edu   
Principal Investigator: Robert A. Brodsky, MD         
Principal Investigator: Amy Dezern, MD         
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: March 23, 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 July 31, 2014