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

Terminated

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

4

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

    • Maryland
      • Baltimore, Maryland, United States, 21031
        • The Sidney Kimmel Comprehensive Cancer Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 months to 70 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

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

This section provides details of the study plan, including how the study is designed and what the study is measuring.

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

This is where you will find people and organizations involved with this study.

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

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

June 1, 2011

Primary Completion (Actual)

August 1, 2014

Study Completion (Actual)

August 1, 2014

Study Registration Dates

First Submitted

June 22, 2011

First Submitted That Met QC Criteria

June 27, 2011

First Posted (Estimate)

June 28, 2011

Study Record Updates

Last Update Posted (Estimate)

October 13, 2016

Last Update Submitted That Met QC Criteria

October 12, 2016

Last Verified

October 1, 2016

More Information

Terms related to this study

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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