T-Cell Depletion and Stem Cell Transplant for Immune Deficiencies and Histiocytic Disorders

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier:
NCT00176826
First received: September 12, 2005
Last updated: March 27, 2014
Last verified: March 2014
Results First Received: March 27, 2014  
Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Conditions: Hemophagocytic Lymphohistiocytosis
X-Linked Lymphoproliferative Disorders
Chediak-Higashi Syndrome
Griscelli Syndrome
Immunologic Diseases
Langerhans-Cell Histiocytosis
Hematologic Diseases
Interventions: Procedure: Stem Cell Transplant
Drug: Myeloablative conditioning regimen

  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
Subjects were recruited from the clinic or hospital where they were being seen for their disease. The study was discussed with them at the time that treatment options were being presented.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Patients had to have a suitable donor identified prior to the subject starting the conditioning regimen.

Reporting Groups
  Description
Intent-To-Treat Patients who were treated with chemotherapies (myeloablative conditioning regimen) and stem cell transplant. Busulfan intravenously for 4 days followed by cyclophosphamide intravenously for 4 days. Rabbit ATG is given intravenously for 4 doses pre-transplant.

Participant Flow:   Overall Study
    Intent-To-Treat  
STARTED     22  
COMPLETED     22  
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
Intent-To-Treat Patients who were treated with chemotherapies (myeloablative conditioning regimen) and stem cell transplant. Busulfan intravenously for 4 days followed by cyclophosphamide intravenously for 4 days. Rabbit ATG is given intravenously for 4 doses pre-transplant.

Baseline Measures
    Intent-To-Treat  
Number of Participants  
[units: participants]
  22  
Age  
[units: participants]
 
<=18 years     22  
Between 18 and 65 years     0  
>=65 years     0  
Age  
[units: years]
Mean ± Standard Deviation
  1.7  ± 0.9  
Gender  
[units: participants]
 
Female     6  
Male     16  
Region of Enrollment  
[units: participants]
 
United States     22  



  Outcome Measures
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1.  Primary:   Time to Transplant Engraftment   [ Time Frame: Day 100 Post Transplant ]

2.  Secondary:   Number of Patients With Treatment Related Mortality.   [ Time Frame: Day 100 Post Transplant ]

3.  Secondary:   Number of Patients Surviving (Disease-free)   [ Time Frame: 1 year ]

4.  Secondary:   Number of Patients With Grade II-IV Graft-Versus-Host Disease (GVHD)   [ Time Frame: Day 100 Post Transplant ]

5.  Secondary:   Number of Patients With Graft Failure   [ Time Frame: Day 100 Post transplant ]

6.  Secondary:   Number of Patients With III-IV Graft-Versus-Host Disease (GVHD)   [ Time Frame: Day 100 Post Transplant ]

7.  Secondary:   Number of Patients Surviving (Disease-free)   [ 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
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  More Information
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Certain Agreements:  
All Principal Investigators ARE employed by the organization sponsoring the study.


Results Point of Contact:  
Name/Title: Dr. Angela Smith
Organization: University of Minnesota
phone: 612-626-2778
e-mail: smith719@umn.edu


No publications provided


Responsible Party: Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier: NCT00176826     History of Changes
Obsolete Identifiers: NCT00973843
Other Study ID Numbers: UMN-MT2000-21, 0010M66781
Study First Received: September 12, 2005
Results First Received: March 27, 2014
Last Updated: March 27, 2014
Health Authority: United States: Institutional Review Board