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Trial record 5 of 9 for:    "Fucosidosis"
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Stem Cell Transplant for Inborn Errors of Metabolism

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier:
NCT00176904
First received: September 12, 2005
Last updated: November 6, 2012
Last verified: November 2012
History of Changes
Results First Received: May 17, 2011  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Conditions: Adrenoleukodystrophy
Metachromatic Leukodystrophy
Globoid Cell Leukodystrophy
Gaucher's Disease
Fucosidosis
Wolman Disease
Niemann-Pick Disease
Batten Disease
GM1 Gangliosidosis
Tay Sachs Disease
Sandhoff Disease
Interventions: Procedure: Stem Cell Transplant
Drug: Busulfan, Cyclophosphamide, Antithymocyte Globulin

  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
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Patients Treated With Stem Cell Transplant All patients treated with protocol regimen (chemotherapy and stem cell transplant).

Participant Flow:   Overall Study
    Patients Treated With Stem Cell Transplant
STARTED   135 
COMPLETED   135 
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
Patients Treated With Stem Cell Transplant All patients treated with protocol regimen (chemotherapy and stem cell transplant).

Baseline Measures
   Patients Treated With Stem Cell Transplant 
Overall Participants Analyzed 
[Units: Participants]
 		 135 
Age 
[Units: Participants]
 		 
<=18 years   117 
Between 18 and 65 years   18 
>=65 years   0 
Age 
[Units: Years]
Mean (Standard Deviation) 		 8.7  (8.9) 
Gender 
[Units: Participants]
 		 
Female   44 
Male   91 
Region of Enrollment 
[Units: Participants]
 		 
United States   135 


  Outcome Measures
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1.  Primary:   Overall Survival   [ Time Frame: 100 Days, 1 Year and 3 Years ]
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2.  Secondary:   Overall Donor Engraftment   [ Time Frame: Day 100 ]
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3.  Secondary:   Number of Patients With Grade II-IV Acute Graft-Versus-Host Disease   [ Time Frame: Day 100 ]
  Show Outcome Measure 3

4.  Secondary:   Number of Patients With Grade III-IV Acute Graft-Versus-Host Disease   [ Time Frame: Day 100 ]
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5.  Secondary:   Number of Patients With Chronic Graft-Versus-Host Disease   [ Time Frame: 1 Year Post Transplant ]
  Show Outcome Measure 5


  Serious Adverse Events
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  Other Adverse Events
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  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
Change in Neuropsychometric function data is not available. Data would have been noted as descriptive and patient specific, as well as disease specific; not table format. No formal analysis was intended.


  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: Paul Orchard, M.D.
Organization: Masonic Cancer Center, University of Minnesota
phone: 612-626-2313
e-mail: orcha001@umn.edu


Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):


Responsible Party: Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier: NCT00176904     History of Changes
Obsolete Identifiers: NCT00005894
Other Study ID Numbers: MT1995-01
Study First Received: September 12, 2005
Results First Received: May 17, 2011
Last Updated: November 6, 2012