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Trial record 16 of 20 for:    "Tay-Sachs Disease"

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
Enrollment 135
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Patients Treated With Stem Cell Transplant
Hide Arm/Group Description All patients treated with protocol regimen (chemotherapy and stem cell transplant).
Period Title: Overall Study
Started 135
Completed 135
Not Completed 0
Arm/Group Title Patients Treated With Stem Cell Transplant
Hide Arm/Group Description All patients treated with protocol regimen (chemotherapy and stem cell transplant).
Overall Number of Baseline Participants 135
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 135 participants
<=18 years
117
  86.7%
Between 18 and 65 years
18
  13.3%
>=65 years
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 135 participants
8.7  (8.9)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 135 participants
Female
44
  32.6%
Male
91
  67.4%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 135 participants
135
1.Primary Outcome
Title Overall Survival
Hide Description Number of patients alive at designated timepoints after transplant.
Time Frame 100 Days, 1 Year and 3 Years
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Patients Treated With Stem Cell Transplant
Hide Arm/Group Description:
All patients treated with protocol regimen (chemotherapy and stem cell transplant).
Overall Number of Participants Analyzed 135
Measure Type: Number
Unit of Measure: Participants
Day 100 120
1 Year 92
3 Years 81
2.Secondary Outcome
Title Overall Donor Engraftment
Hide 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
1 Patient not included due to early death (before day 40).
Arm/Group Title Patients Treated With Stem Cell Transplant
Hide Arm/Group Description:
All patients treated with protocol regimen (chemotherapy and stem cell transplant).
Overall Number of Participants Analyzed 134
Measure Type: Number
Unit of Measure: Participants
123
3.Secondary Outcome
Title Number of Patients With Grade II-IV Acute Graft-Versus-Host Disease
Hide 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Patients Treated With Stem Cell Transplant
Hide Arm/Group Description:
All patients treated with protocol regimen (chemotherapy and stem cell transplant).
Overall Number of Participants Analyzed 135
Measure Type: Number
Unit of Measure: Participants
34
4.Secondary Outcome
Title Number of Patients With Grade III-IV Acute Graft-Versus-Host Disease
Hide 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Patients Treated With Stem Cell Transplant
Hide Arm/Group Description:
All patients treated with protocol regimen (chemotherapy and stem cell transplant).
Overall Number of Participants Analyzed 135
Measure Type: Number
Unit of Measure: Participants
13
5.Secondary Outcome
Title Number of Patients With Chronic Graft-Versus-Host Disease
Hide 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Patients Treated With Stem Cell Transplant
Hide Arm/Group Description:
All patients treated with protocol regimen (chemotherapy and stem cell transplant).
Overall Number of Participants Analyzed 135
Measure Type: Number
Unit of Measure: Participants
13
Time Frame Serious adverse experiences were collected during the first 100 days after transplant then 6 months and annually for 3 years.
Adverse Event Reporting Description Selected serious adverse experiences (graft failure/autologous recovery, severe acute GVHD (grades III and IV) and death were collected during the first 100 days after transplant then 6 months and annually for 3 years. After day 100, only death or an unexpected adverse event will be reported. Adverse events were not collected in this study.
 
Arm/Group Title Patients Treated With Stem Cell Transplant
Hide Arm/Group Description All patients treated with protocol regimen (chemotherapy and stem cell transplant).
All-Cause Mortality
Patients Treated With Stem Cell Transplant
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Patients Treated With Stem Cell Transplant
Affected / at Risk (%) # Events
Total   62/135 (45.93%)    
Blood and lymphatic system disorders   
Primary graft failure  1  2/135 (1.48%)  2
General disorders   
Auto recovery  1 [1]  9/135 (6.67%)  9
Death  1  54/135 (40.00%)  54
Indicates events were collected by systematic assessment
1
Term from vocabulary, CTCAE (3.0)
[1]
Delayed side effects that includes, loss of appetite, nausea and vomiting, changes in vision, mouth and throat soreness, diarrhea, difficulty sleeping and fatigue, infection, hair loss, skin reactions and graft-versus-host disease.
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Patients Treated With Stem Cell Transplant
Affected / at Risk (%) # Events
Total   0/135 (0.00%)    
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.
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
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