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Cyclophosphamide and Anti-thymocyte Globulin Followed By Methotrexate and Cyclosporine in Preventing Chronic Graft-Versus-Host Disease in Patients With Severe Aplastic Anemia Undergoing Donor Bone Marrow Transplant

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ClinicalTrials.gov Identifier: NCT00343785
Recruitment Status : Completed
First Posted : June 23, 2006
Results First Posted : March 16, 2017
Last Update Posted : April 13, 2017
Sponsor:
Collaborator:
National Heart, Lung, and Blood Institute (NHLBI)
Information provided by (Responsible Party):
Rainer Storb, Fred Hutchinson Cancer Research Center

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Aplastic Anemia
Interventions Drug: cyclophosphamide
Biological: anti-thymocyte globulin
Drug: cyclosporine
Procedure: allogeneic bone marrow transplantation
Drug: methotrexate
Genetic: DNA analysis
Other: flow cytometry
Genetic: polymorphism analysis
Other: laboratory biomarker analysis
Enrollment 21
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Treatment (Conditioning Regimen, Transplant, GVHD Prophylaxis)
Hide Arm/Group Description Patients receive a conditioning regimen comprising cyclophosphamide IV on days -5 to -2 and anti-thymocyte globulin IV over 4-10 hours on days -4 to -2. Patients undergo allogeneic bone marrow transplantation on day 0. Patients then receive GVHD prophylaxis comprising methotrexate IV on days 1, 3, 6, and 11 and cyclosporine IV over 1 hour or PO twice daily on days -1 to 50, followed by a taper until 6 months after grafting.
Period Title: Overall Study
Started 21
Completed 21
Not Completed 0
Arm/Group Title Treatment (Conditioning Regimen, Transplant, GVHD Prophylaxis)
Hide Arm/Group Description Patients receive a conditioning regimen comprising cyclophosphamide IV on days -5 to -2 and anti-thymocyte globulin IV over 4-10 hours on days -4 to -2. Patients undergo allogeneic bone marrow transplantation on day 0. Patients then receive GVHD prophylaxis comprising methotrexate IV on days 1, 3, 6, and 11 and cyclosporine IV over 1 hour or PO twice daily on days -1 to 50, followed by a taper until 6 months after grafting.
Overall Number of Baseline Participants 21
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 21 participants
15
(3 to 52)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 21 participants
Female
5
  23.8%
Male
16
  76.2%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 21 participants
American Indian or Alaska Native
0
   0.0%
Asian
5
  23.8%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
0
   0.0%
White
9
  42.9%
More than one race
3
  14.3%
Unknown or Not Reported
4
  19.0%
1.Primary Outcome
Title Incidence of Chronic GVHD
Hide Description Analyzed using cumulative incidence estimates, treating death or rejection as competing risk events.
Time Frame 2 years
Hide Outcome Measure Data
Hide Analysis Population Description
All Patients
Arm/Group Title Patients Receive a Conditioning Regimen Comprising Cyclophosph
Hide Arm/Group Description:
Patients receive a conditioning regimen comprising cyclophosphamide IV on days -5 to -2 and anti-thymocyte globulin IV over 4-10 hours on days -4 to -2. Patients undergo allogeneic bone marrow transplantation on day 0. Patients then receive GVHD prophylaxis comprising methotrexate IV on days 1, 3, 6, and 11 and cyclosporine IV over 1 hour or PO twice daily on days -1 to 50, followed by a taper until 6 months after grafting.
Overall Number of Participants Analyzed 21
Measure Type: Number
Unit of Measure: number participants with chronic GVHD
5
2.Secondary Outcome
Title Number of Days to Neutrophil Recovery to >500/uL
Hide Description First of 3 consecutive days of neutrophils >500/uL
Time Frame 100 days post-transplant
Hide Outcome Measure Data
Hide Analysis Population Description
All patients
Arm/Group Title Treatment (Conditioning Regimen, Transplant, GVHD Prophylaxis)
Hide Arm/Group Description:
Patients receive a conditioning regimen comprising cyclophosphamide IV on days -5 to -2 and anti-thymocyte globulin IV over 4-10 hours on days -4 to -2. Patients undergo allogeneic bone marrow transplantation on day 0. Patients then receive GVHD prophylaxis comprising methotrexate IV on days 1, 3, 6, and 11 and cyclosporine IV over 1 hour or PO twice daily on days -1 to 50, followed by a taper until 6 months after grafting.
Overall Number of Participants Analyzed 21
Median (Full Range)
Unit of Measure: days
26
(21 to 36)
3.Secondary Outcome
Title Overall Survival
Hide Description Number of patients alive at one year
Time Frame From the time of enrollment until death from any cause up to one year
Hide Outcome Measure Data
Hide Analysis Population Description
All Patients
Arm/Group Title Treatment (Conditioning Regimen, Transplant, GVHD Prophylaxis)
Hide Arm/Group Description:
Patients receive a conditioning regimen comprising cyclophosphamide IV on days -5 to -2 and anti-thymocyte globulin IV over 4-10 hours on days -4 to -2. Patients undergo allogeneic bone marrow transplantation on day 0. Patients then receive GVHD prophylaxis comprising methotrexate IV on days 1, 3, 6, and 11 and cyclosporine IV over 1 hour or PO twice daily on days -1 to 50, followed by a taper until 6 months after grafting.
Overall Number of Participants Analyzed 21
Measure Type: Count of Participants
Unit of Measure: Participants
21
 100.0%
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Treatment (Conditioning Regimen, Transplant, GVHD Prophylaxis)
Hide Arm/Group Description Patients receive a conditioning regimen comprising cyclophosphamide IV on days -5 to -2 and anti-thymocyte globulin IV over 4-10 hours on days -4 to -2. Patients undergo allogeneic bone marrow transplantation on day 0. Patients then receive GVHD prophylaxis comprising methotrexate IV on days 1, 3, 6, and 11 and cyclosporine IV over 1 hour or PO twice daily on days -1 to 50, followed by a taper until 6 months after grafting.
All-Cause Mortality
Treatment (Conditioning Regimen, Transplant, GVHD Prophylaxis)
Affected / at Risk (%)
Total   0/21 (0.00%)    
Show Serious Adverse Events Hide Serious Adverse Events
Treatment (Conditioning Regimen, Transplant, GVHD Prophylaxis)
Affected / at Risk (%) # Events
Total   5/21 (23.81%)    
Cardiac disorders   
hypertension  2/21 (9.52%)  2
Ear and labyrinth disorders   
Tinnitus [1]  1/21 (4.76%)  1
Gastrointestinal disorders   
parental nutrition [2]  3/21 (14.29%)  3
Hepatobiliary disorders   
hyperbilirubinemia [3]  1/21 (4.76%)  1
Nervous system disorders   
hypoxia  1/21 (4.76%)  1
[1]
significant tinnitus in both ears with hearing loss, correctable with hearing aids
[2]
mucositis requiring parental nutrition
[3]
bilirubin > 3x ULN, cholangitis lenta
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Treatment (Conditioning Regimen, Transplant, GVHD Prophylaxis)
Affected / at Risk (%) # Events
Total   21/21 (100.00%)    
Gastrointestinal disorders   
nausea  21/21 (100.00%)  21
vomiting  21/21 (100.00%)  21
mucositits  21/21 (100.00%)  21
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Dr. Rainer Storb, Director Transplantation Biology
Organization: Fred Hutchinson Cancer Research Center
Phone: 2066676839
Responsible Party: Rainer Storb, Fred Hutchinson Cancer Research Center
ClinicalTrials.gov Identifier: NCT00343785     History of Changes
Other Study ID Numbers: 2054.00
NCI-2010-01781 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) )
P01HL036444 ( U.S. NIH Grant/Contract )
First Submitted: June 22, 2006
First Posted: June 23, 2006
Results First Submitted: January 26, 2017
Results First Posted: March 16, 2017
Last Update Posted: April 13, 2017