Stem Cell Transplant for Inborn Errors of Metabolism

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. Identifier: NCT00176904
Recruitment Status : Completed
First Posted : September 15, 2005
Results First Posted : June 10, 2011
Last Update Posted : December 28, 2017
Information provided by (Responsible Party):
Masonic Cancer Center, University of Minnesota

Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions: Adrenoleukodystrophy
Metachromatic Leukodystrophy
Globoid Cell Leukodystrophy
Gaucher's Disease
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

  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
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]
[Units: Participants]
Count of Participants
<=18 years      117  86.7% 
Between 18 and 65 years      18  13.3% 
>=65 years      0   0.0% 
[Units: Years]
Mean (Standard Deviation)
 8.7  (8.9) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
Female      44  32.6% 
Male      91  67.4% 
Region of Enrollment 
[Units: Participants]
United States   135 

  Outcome Measures

1.  Primary:   Overall Survival   [ Time Frame: 100 Days, 1 Year and 3 Years ]

2.  Secondary:   Overall Donor Engraftment   [ Time Frame: Day 100 ]

3.  Secondary:   Number of Patients With Grade II-IV Acute Graft-Versus-Host Disease   [ Time Frame: Day 100 ]

4.  Secondary:   Number of Patients With Grade III-IV Acute Graft-Versus-Host Disease   [ Time Frame: Day 100 ]

5.  Secondary:   Number of Patients With Chronic Graft-Versus-Host Disease   [ Time Frame: 1 Year Post Transplant ]

  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
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

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

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

Responsible Party: Masonic Cancer Center, University of Minnesota Identifier: NCT00176904     History of Changes
Obsolete Identifiers: NCT00005894
Other Study ID Numbers: MT1995-01
First Submitted: September 12, 2005
First Posted: September 15, 2005
Results First Submitted: May 17, 2011
Results First Posted: June 10, 2011
Last Update Posted: December 28, 2017