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

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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
Enrollment 22
Recruitment Details 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 Patients had to have a suitable donor identified prior to the subject starting the conditioning regimen.
Arm/Group Title Intent-To-Treat
Hide Arm/Group Description 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.
Period Title: Overall Study
Started 22
Completed 22
Not Completed 0
Arm/Group Title Intent-To-Treat
Hide Arm/Group Description 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.
Overall Number of Baseline Participants 22
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 22 participants
<=18 years
22
 100.0%
Between 18 and 65 years
0
   0.0%
>=65 years
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 22 participants
1.7  (0.9)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 22 participants
Female
6
  27.3%
Male
16
  72.7%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 22 participants
22
1.Primary Outcome
Title Time to Transplant Engraftment
Hide Description [Not Specified]
Time Frame Day 100 Post Transplant
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Intent-To-Treat
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 22
Mean (Standard Deviation)
Unit of Measure: days
19.8  (5.2)
2.Secondary Outcome
Title Number of Patients With Treatment Related Mortality.
Hide Description [Not Specified]
Time Frame Day 100 Post Transplant
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Intent-To-Treat
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 22
Measure Type: Number
Unit of Measure: participants
6
3.Secondary Outcome
Title Number of Patients Surviving (Disease-free)
Hide Description [Not Specified]
Time Frame 1 year
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Intent-To-Treat
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 22
Measure Type: Number
Unit of Measure: participants
14
4.Secondary Outcome
Title Number of Patients With Grade II-IV Graft-Versus-Host Disease (GVHD)
Hide Description [Not Specified]
Time Frame Day 100 Post Transplant
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Intent-To-Treat
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 22
Measure Type: Number
Unit of Measure: participants
4
5.Secondary Outcome
Title Number of Patients With Graft Failure
Hide Description [Not Specified]
Time Frame Day 100 Post transplant
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Intent-To-Treat
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 22
Measure Type: Number
Unit of Measure: participants
2
6.Secondary Outcome
Title Number of Patients With III-IV Graft-Versus-Host Disease (GVHD)
Hide Description [Not Specified]
Time Frame Day 100 Post Transplant
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Intent-To-Treat
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 22
Measure Type: Number
Unit of Measure: participants
1
7.Secondary Outcome
Title Number of Patients Surviving (Disease-free)
Hide Description [Not Specified]
Time Frame 3 years
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Intent-To-Treat
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 22
Measure Type: Number
Unit of Measure: participants
10
Time Frame From the time consent was signed to the end of follow-up, which was 3 years.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Intent-To-Treat
Hide Arm/Group Description 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.
All-Cause Mortality
Intent-To-Treat
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Intent-To-Treat
Affected / at Risk (%) # Events
Total   2/22 (9.09%)    
General disorders   
Death * 1 [1]  1/22 (4.55%)  1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
Secondary Malignancy * 1  1/22 (4.55%)  1
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, CTCAE (3.0)
[1]
Multi-organ failure
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Intent-To-Treat
Affected / at Risk (%) # Events
Total   0/22 (0.00%)    
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Dr. Angela Smith
Organization: University of Minnesota
Phone: 612-626-2778
EMail: smith719@umn.edu
Layout table for additonal information
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