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Allogeneic Stem Cell Transplantation Following Chemotherapy in Patients With Hemoglobinopathies

This study has been completed.
Sponsor:
Collaborators:
Beth Israel Deaconess Medical Center
Massachusetts General Hospital
Brigham and Women's Hospital
Emory University
Feist-Weiller Cancer Center at Louisiana State University Health Sciences
Ohio State University
Information provided by (Responsible Party):
Catherine Wu, MD, Dana-Farber Cancer Institute
ClinicalTrials.gov Identifier:
NCT00153985
First received: September 8, 2005
Last updated: July 24, 2013
Last verified: July 2013
Results First Received: December 5, 2012  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Conditions: Hemoglobinopathies
Sickle Cell Disease
Thalassemia
Interventions: Drug: Busulfex
Drug: Fludarabine
Drug: Alemtuzumab
Procedure: Stem Cell Transfusion

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Activated for enrollment 3/4/2004. Closed to enrollment 4/25/2008. Participating institutions included: Dana-Farber Cancer Institute, Boston, Massachusetts, Feist-Weiller Cancer Center, LSU Health Sciences Center, Shreveport, Louisiana and Winship Cancer Institute, Emory University, Atlanta, Georgia

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
All enrolled patients received a stem cell transplant.

Reporting Groups
  Description
Transplant for Severe Hemoglobinopathies Patients with severe hemoglobinopathies (eg. sickle cell disease, thalassemia major) with related donors who are identical at 6 HLA loci: (HLA-A, HLA-B, HLA-DRB1). The preparative regimen consisted of Busulfex, fludarabine and alemtuzumab.

Participant Flow:   Overall Study
    Transplant for Severe Hemoglobinopathies  
STARTED     2  
COMPLETED     2  
NOT COMPLETED     0  



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Transplant for Severe Hemoglobinopathies Patients with severe hemoglobinopathies (eg. sickle cell disease, thalassemia major) with related donors who are identical at 6 HLA loci: (HLA-A, HLA-B, HLA-DRB1). The preparative regimen consisted of Busulfex, fludarabine and alemtuzumab.

Baseline Measures
    Transplant for Severe Hemoglobinopathies  
Number of Participants  
[units: participants]
  2  
Age  
[units: participants]
 
<=18 years     0  
Between 18 and 65 years     2  
>=65 years     0  
Age  
[units: years]
Mean ± Standard Deviation
  25  ± 2  
Gender  
[units: participants]
 
Female     2  
Male     0  
Region of Enrollment  
[units: participants]
 
United States     2  



  Outcome Measures
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1.  Primary:   Stable Engraftment With Donor Stem Cells in Patients With Severe Hemoglobinopathy.   [ Time Frame: 3 years ]

2.  Secondary:   Solid Organ Toxicity Related to the Conditioning Regimen.   [ Time Frame: 3 years ]

3.  Secondary:   The Incidence of Grade II-IV Acute Graft vs. Host Disease.   [ Time Frame: 3 years ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
Early termination leading to small numbers of subjects analyzed.


  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Catherine Wu, MD
Organization: Dana-Farber Cancer Institute
phone: 617-632-5943
e-mail: cwu@partners.org


Publications of Results:

Responsible Party: Catherine Wu, MD, Dana-Farber Cancer Institute
ClinicalTrials.gov Identifier: NCT00153985     History of Changes
Other Study ID Numbers: 03-338
Study First Received: September 8, 2005
Results First Received: December 5, 2012
Last Updated: July 24, 2013
Health Authority: United States: Institutional Review Board