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Stem Cell Transplant for Inborn Errors of Metabolism

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ClinicalTrials.gov Identifier: NCT00176904
Recruitment Status : Completed
First Posted : September 15, 2005
Results First Posted : June 10, 2011
Last Update Posted : December 28, 2017
Sponsor:
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
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

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

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]
Count of Participants
 
<=18 years      117  86.7% 
Between 18 and 65 years      18  13.3% 
>=65 years      0   0.0% 
Age 
[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 ]

Measure Type Primary
Measure Title Overall Survival
Measure Description Number of patients alive at designated timepoints after transplant.
Time Frame 100 Days, 1 Year and 3 Years  

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).

Measured Values
   Patients Treated With Stem Cell Transplant 
Participants Analyzed   135 
Overall Survival 
[Units: Participants]
 
Day 100   120 
1 Year   92 
3 Years   81 

No statistical analysis provided for Overall Survival



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

Measure Type Secondary
Measure Title Overall Donor Engraftment
Measure Description Number of patients with full donor chimerism (state in bone marrow transplantation in which bone marrow and host cells exist compatibly without signs of graft-versus-host rejection disease) by Day 100 post-transplant of at least 90%.
Time Frame Day 100  

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.
1 Patient not included due to early death (before day 40).

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

Measured Values
   Patients Treated With Stem Cell Transplant 
Participants Analyzed   134 
Overall Donor Engraftment 
[Units: Participants]
 123 

No statistical analysis provided for Overall Donor Engraftment



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

Measure Type Secondary
Measure Title Number of Patients With Grade II-IV Acute Graft-Versus-Host Disease
Measure Description Number of patients who exhibited acute graft-versus-host disease by Day 100 post transplant. Graft-versus-host disease (GVHD) is a complication that can occur after a stem cell or bone marrow transplant in which the newly transplanted material attacks the transplant recipient's body. Grade I=mild, Grade II=moderate, Grade III=severe, Grade IV=life threatening.
Time Frame Day 100  

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).

Measured Values
   Patients Treated With Stem Cell Transplant 
Participants Analyzed   135 
Number of Patients With Grade II-IV Acute Graft-Versus-Host Disease 
[Units: Participants]
 34 

No statistical analysis provided for Number of Patients With Grade II-IV Acute Graft-Versus-Host Disease



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

Measure Type Secondary
Measure Title Number of Patients With Grade III-IV Acute Graft-Versus-Host Disease
Measure Description Number of patients who exhibited acute graft-versus-host disease by Day 100 post transplant. Graft-versus-host disease (GVHD) is a complication that can occur after a stem cell or bone marrow transplant in which the newly transplanted material attacks the transplant recipient's body. Grade I=mild, Grade II=moderate, Grade III=severe, Grade IV=life threatening.
Time Frame Day 100  

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).

Measured Values
   Patients Treated With Stem Cell Transplant 
Participants Analyzed   135 
Number of Patients With Grade III-IV Acute Graft-Versus-Host Disease 
[Units: Participants]
 13 

No statistical analysis provided for Number of Patients With Grade III-IV Acute Graft-Versus-Host Disease



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

Measure Type Secondary
Measure Title Number of Patients With Chronic Graft-Versus-Host Disease
Measure Description Number of patients who exhibited chronic graft-versus-host disease by 1 Year post transplant. Graft-versus-host disease (GVHD) is a complication that can occur after a stem cell or bone marrow transplant in which the newly transplanted material attacks the transplant recipient's body. Chronic GVHD is an extension of this syndrome.
Time Frame 1 Year Post Transplant  

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).

Measured Values
   Patients Treated With Stem Cell Transplant 
Participants Analyzed   135 
Number of Patients With Chronic Graft-Versus-Host Disease 
[Units: Participants]
 13 

No statistical analysis provided for Number of Patients With Chronic Graft-Versus-Host Disease




  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
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
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