ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00176826
Recruitment Status : Terminated (Replaced by another protocol)
First Posted : September 15, 2005
Results First Posted : April 28, 2014
Last Update Posted : January 23, 2018
Sponsor:
Information provided by (Responsible Party):
Masonic Cancer Center, University of Minnesota

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: None (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

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

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 
Overall Participants Analyzed 
[Units: Participants]
 22 
Age 
[Units: Participants]
Count of Participants
 
<=18 years      22 100.0% 
Between 18 and 65 years      0   0.0% 
>=65 years      0   0.0% 
Age 
[Units: Years]
Mean (Standard Deviation)
 1.7  (0.9) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
 
Female      6  27.3% 
Male      16  72.7% 
Region of Enrollment 
[Units: Participants]
 
United States   22 


  Outcome Measures

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

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
No text entered.


  More Information

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



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 ( Other Identifier: Institutional Review Board, University of Minnesota )
First Submitted: September 12, 2005
First Posted: September 15, 2005
Results First Submitted: March 27, 2014
Results First Posted: April 28, 2014
Last Update Posted: January 23, 2018