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Trial record 2 of 16 for:    "Congenital Hypoplastic Anemia" | "Fludarabine"

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
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: 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