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Study Results
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Stem Cell Transplant for Bone Marrow Failures
This study has been completed.
Study NCT00176878   Information provided by Masonic Cancer Center, University of Minnesota
First Received: September 12, 2005   Last Updated: August 28, 2009   History of Changes
Study Type: Interventional
Study Design: Non-Randomized, Open Label, Uncontrolled, Parallel Assignment
Conditions: Diamond-Blackfan Anemia
Kostmann's Neutropenia
Shwachman-Diamond Syndrome
Interventions: Procedure: Stem cell transplant
Drug: Busulfan, fludarabine and anti-thymocyte globulin (ATG)
Procedure: Total lymphoid irradiation

  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
Intent-To-Treat All patients with non-malignant, congenital bone marrow failure disorders and treated with stem cell transplant, study medications and irradiation.

Participant Flow:   Overall Study
  Intent-To-Treat
STARTED   10  
COMPLETED   10  
NOT COMPLETED   0  



  Baseline Characteristics
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Reporting Groups
  Description
Intent-To-Treat All patients with non-malignant, congenital bone marrow failure disorders and treated with stem cell transplant, study medications and irradiation.

Baseline Measures
  Intent-To-Treat
Number of Participants  
[units: participants]
 		10
Age  
[units: participants]
 		 
<=18 years 10
Between 18 and 65 years 0
>=65 years 0
Age  
[units: years]
Mean ± Standard Deviation 		4.8 ± 7.6
Gender  
[units: participants]
 		 
Female 3
Male 7
Region of Enrollment  
[units: participants]
 		 
United States 10



  Outcome Measures
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1.  Primary:   Number of Patients Alive (Survival)at 2 Years   [ 2 years ]

Measure Type Primary
Measure Title Number of Patients Alive (Survival)at 2 Years
Measure Description Calculated from day 1 of transplant to last contact.
Time Frame 2 years  
Safety Issue No  

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
Intent-To-Treat All patients with non-malignant, congenital bone marrow failure disorders and treated with stem cell transplant, study medications and irradiation.

Measured Values
  Intent-To-Treat
Number of Participants Analyzed
[units: participants] 		10
Number of Patients Alive (Survival)at 2 Years
[units: Participants]
 		6

No statistical analysis provided for Number of Patients Alive (Survival)at 2 Years



2.  Secondary:   Number of Patients Alive at Three Years (Survival)   [ 3 years ]

Measure Type Secondary
Measure Title Number of Patients Alive at Three Years (Survival)
Measure Description Number of subjects who survived 3 years post-transplant.
Time Frame 3 years  
Safety Issue No  

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
Intent-To-Treat All patients with non-malignant, congenital bone marrow failure disorders and treated with stem cell transplant, study medications and irradiation.

Measured Values
  Intent-To-Treat
Number of Participants Analyzed
[units: participants] 		10
Number of Patients Alive at Three Years (Survival)
[units: Participants]
 		6

No statistical analysis provided for Number of Patients Alive at Three Years (Survival)



3.  Secondary:   Number of Patients With Succcessful Engraftment After Transplantation   [ 42 Days ]

Measure Type Secondary
Measure Title Number of Patients With Succcessful Engraftment After Transplantation
Measure Description Number of patients who received non-genotypic identical marrow or cord blood cells using a "non-myeloablative" preparative regimen and exhibited engraftment at Day 42.
Time Frame 42 Days  
Safety Issue No  

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
Intent-To-Treat All patients with non-malignant, congenital bone marrow failure disorders and treated with stem cell transplant, study medications and irradiation.

Measured Values
  Intent-To-Treat
Number of Participants Analyzed
[units: participants] 		10
Number of Patients With Succcessful Engraftment After Transplantation
[units: Participants]
 		10

No statistical analysis provided for Number of Patients With Succcessful Engraftment After Transplantation



4.  Secondary:   Number of Patients With Grade 2-4 Acute Graft Versus Host Disease   [ 100 Days ]

Measure Type Secondary
Measure Title Number of Patients With Grade 2-4 Acute Graft Versus Host Disease
Measure Description Number of patients with Grade 2, 3 and 4 Acute (normally observed within the first 100 days) Graft Versus Host Disease. Acute GVHD is staged as follows: overall grade (skin-liver-gut) with each organ staged individually from a low of 1 to a high of 4. Patients with grade IV GVHD usually have a poor prognosis. Grade 2 = moderate, Grade 3 = severe, Grade 4 = life threatening.
Time Frame 100 Days  
Safety Issue Yes  

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
Intent-To-Treat All patients with non-malignant, congenital bone marrow failure disorders and treated with stem cell transplant, study medications and irradiation.

Measured Values
  Intent-To-Treat
Number of Participants Analyzed
[units: participants] 		10
Number of Patients With Grade 2-4 Acute Graft Versus Host Disease
[units: Participants]
 		5

No statistical analysis provided for Number of Patients With Grade 2-4 Acute Graft Versus Host Disease



5.  Secondary:   Number of Patients With Chronic Graft Versus Host Disease   [ 2 years ]

Measure Type Secondary
Measure Title Number of Patients With Chronic Graft Versus Host Disease
Measure Description Number of patients who exhibited chronic (normally occurs after 100 days) Graft Versus Host Disease at 2 years post transplant. Chronic graft-versus-host-disease, over its long-term course, can also cause damage to the connective tissue and exocrine glands.
Time Frame 2 years  
Safety Issue Yes  

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
Intent-To-Treat All patients with non-malignant, congenital bone marrow failure disorders and treated with stem cell transplant, study medications and irradiation.

Measured Values
  Intent-To-Treat
Number of Participants Analyzed
[units: participants] 		10
Number of Patients With Chronic Graft Versus Host Disease
[units: Participants]
 		3

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



6.  Secondary:   Number of Patients With Disease Recurrence   [ 2 years ]

Measure Type Secondary
Measure Title Number of Patients With Disease Recurrence
Measure Description Number of patients who exhibited disease recurrence at 2 years.
Time Frame 2 years  
Safety Issue No  

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
Intent-To-Treat All patients with non-malignant, congenital bone marrow failure disorders and treated with stem cell transplant, study medications and irradiation.

Measured Values
  Intent-To-Treat
Number of Participants Analyzed
[units: participants] 		10
Number of Patients With Disease Recurrence
[units: Participants]
 		0

No statistical analysis provided for Number of Patients With Disease Recurrence




  Serious Adverse Events
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  Other Adverse Events
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  More Information
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Certain Agreements:  
All Principal Investigators ARE employed by the organization sponsoring the study.


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
Pharmacokinetic parameters in patients receiving 2 mg/kg/dose of busulfan twice daily was not performed. No data is available.  


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


No publications provided


Responsible Party: Masonic Cancer Center, University of Minnesota ( Paul Orchard, M.D. )
Study ID Numbers: 9504M09637, MT2000-18
Study First Received: September 12, 2005
Results First Received: June 18, 2009
Last Updated: August 28, 2009
ClinicalTrials.gov Identifier: NCT00176878     History of Changes
Health Authority: United States: Institutional Review Board





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