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Trial record 3 of 18 for:    "Severe Congenital Neutropenia"

Stem Cell Transplant for Bone Marrow Failure Syndromes

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ClinicalTrials.gov Identifier: NCT00176878
Recruitment Status : Completed
First Posted : September 15, 2005
Results First Posted : August 6, 2009
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: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Diamond-Blackfan Anemia
Kostmann's Neutropenia
Shwachman-Diamond Syndrome
Interventions Procedure: Stem cell transplant
Drug: Fludarabine monophosphate
Procedure: Total lymphoid irradiation
Drug: Busulfan
Biological: anti-thymocyte globulin
Enrollment 10
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Bone Marrow Failure Patients
Hide Arm/Group Description All patients with non-malignant, congenital bone marrow failure disorders and treated with stem cell transplant, chemotherapy (Busulfan, ATG, Fludarabine) and irradiation.
Period Title: Overall Study
Started 10
Completed 10
Not Completed 0
Arm/Group Title Bone Marrow Failure Patients
Hide Arm/Group Description All patients with non-malignant, congenital bone marrow failure disorders and treated with stem cell transplant, chemotherapy (Busulfan, ATG, Fludarabine) and irradiation.
Overall Number of Baseline Participants 10
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants
<=18 years
10
 100.0%
Between 18 and 65 years
0
   0.0%
>=65 years
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 10 participants
4.8  (7.6)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants
Female
3
  30.0%
Male
7
  70.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 10 participants
10
1.Primary Outcome
Title Number of Patients Alive (Survival) at 2 Years
Hide Description Calculated from day 1 of transplant to last contact.
Time Frame 2 years
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Bone Marrow Failure Patients
Hide Arm/Group Description:
All patients with non-malignant, congenital bone marrow failure disorders and treated with stem cell transplant, chemotherapy (Busulfan, ATG, Fludarabine) and irradiation.
Overall Number of Participants Analyzed 10
Measure Type: Number
Unit of Measure: Participants
6
2.Secondary Outcome
Title Number of Patients Alive at Three Years (Survival)
Hide Description Number of subjects who survived 3 years post-transplant.
Time Frame 3 years
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Bone Marrow Failure Patients
Hide Arm/Group Description:
All patients with non-malignant, congenital bone marrow failure disorders and treated with stem cell transplant, chemotherapy (Busulfan, ATG, Fludarabine) and irradiation.
Overall Number of Participants Analyzed 10
Measure Type: Number
Unit of Measure: Participants
6
3.Secondary Outcome
Title Number of Patients With Succcessful Engraftment After Transplantation
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Bone Marrow Failure Patients
Hide Arm/Group Description:
All patients with non-malignant, congenital bone marrow failure disorders and treated with stem cell transplant, chemotherapy (Busulfan, ATG, Fludarabine) and irradiation.
Overall Number of Participants Analyzed 10
Measure Type: Number
Unit of Measure: Participants
10
4.Secondary Outcome
Title Number of Patients With Grade 2-4 Acute Graft Versus Host Disease
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Bone Marrow Failure Patients
Hide Arm/Group Description:
All patients with non-malignant, congenital bone marrow failure disorders and treated with stem cell transplant, chemotherapy (Busulfan, ATG, Fludarabine) and irradiation.
Overall Number of Participants Analyzed 10
Measure Type: Number
Unit of Measure: Participants
5
5.Secondary Outcome
Title Number of Patients With Chronic Graft Versus Host Disease
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Bone Marrow Failure Patients
Hide Arm/Group Description:
All patients with non-malignant, congenital bone marrow failure disorders and treated with stem cell transplant, chemotherapy (Busulfan, ATG, Fludarabine) and irradiation.
Overall Number of Participants Analyzed 10
Measure Type: Number
Unit of Measure: Participants
3
6.Secondary Outcome
Title Number of Patients With Disease Recurrence
Hide Description Number of patients who exhibited disease recurrence at 2 years.
Time Frame 2 years
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Bone Marrow Failure Patients
Hide Arm/Group Description:
All patients with non-malignant, congenital bone marrow failure disorders and treated with stem cell transplant, chemotherapy (Busulfan, ATG, Fludarabine) and irradiation.
Overall Number of Participants Analyzed 10
Measure Type: Number
Unit of Measure: Participants
0
Time Frame Day 1 of study up through 1 year
Adverse Event Reporting Description Only serious adverse events are reported. Other adverse events were not collected.
 
Arm/Group Title Bone Marrow Failure Patients
Hide Arm/Group Description All patients with non-malignant, congenital bone marrow failure disorders and treated with stem cell transplant, chemotherapy (Busulfan, ATG, Fludarabine) and irradiation.
All-Cause Mortality
Bone Marrow Failure Patients
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Bone Marrow Failure Patients
Affected / at Risk (%) # Events
Total   5/10 (50.00%)    
Blood and lymphatic system disorders   
Bone marrow graft failure  1 [1]  3/10 (30.00%)  3
Graft-versus-host disease (acute)  1 [2]  1/10 (10.00%)  1
General disorders   
Death  1  3/10 (30.00%)  3
Indicates events were collected by systematic assessment
1
Term from vocabulary, CTCAE (3.0)
[1]
Autologous recovery, Chimerism (secondary)
[2]
Grade III-IV
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Bone Marrow Failure Patients
Affected / at Risk (%) # Events
Total   0/10 (0.00%)    
Pharmacokinetic parameters in patients receiving 2 mg/kg/dose of busulfan twice daily was not performed. No data is available.
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Paul Orchard, M.D.
Organization: Masonic Cancer Center, University of Minnesota
Phone: 612-626-2313
EMail: orcha001@umn.edu
Layout table for additonal information
Responsible Party: Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier: NCT00176878     History of Changes
Obsolete Identifiers: NCT00005895
Other Study ID Numbers: MT2000-18
9504M09637 ( Other Identifier: IRB, University of Minnesota )
First Submitted: September 12, 2005
First Posted: September 15, 2005
Results First Submitted: June 18, 2009
Results First Posted: August 6, 2009
Last Update Posted: December 28, 2017