Comparison of Two Types of Shunts in Infants With Single Ventricle Defect Undergoing Staged Reconstruction--Pediatric Heart Network

This study has been completed.
Sponsor:
Collaborators:
Pediatric Heart Network
Information provided by:
New England Research Institutes
ClinicalTrials.gov Identifier:
NCT00115934
First received: June 26, 2005
Last updated: August 2, 2013
Last verified: August 2013
Results First Received: September 14, 2010  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Heart Defects, Congenital
Interventions: Procedure: Blalock-Taussig pulmonary artery shunt
Procedure: Right ventricular to pulmonary artery shunt

  Participant Flow
  Hide Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Subjects undergoing a Norwood were randomized to MBTS or RVPAS at 15 North American centers.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
There were no pre-assignment requirements beyond the initial screening criteria.

Reporting Groups
  Description
MBTS Blalock-Taussig pulmonary artery shunt
RVPAS Right ventricular to pulmonary artery shunt

Participant Flow:   Overall Study
    MBTS     RVPAS  
STARTED     279 [1]   276 [2]
COMPLETED     275     274  
NOT COMPLETED     4     2  
Lost to Follow-up                 1                 0  
Did not receive intervention                 3                 2  
[1] 279 randomized to MBTS, but 3 did not have a Norwood surgery; one was withdrawn-with limited data.
[2] 276 randomized to RVPAS, but 2 did not have a Norwood surgery and were excluded from analyses.



  Baseline Characteristics
  Hide Baseline Characteristics

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
MBTS Blalock-Taussig pulmonary artery shunt
RVPAS Right ventricular to pulmonary artery shunt
Total Total of all reporting groups

Baseline Measures
    MBTS     RVPAS     Total  
Number of Participants  
[units: participants]
  275     274     549  
Age  
[units: participants]
     
<=18 years     275     274     549  
Between 18 and 65 years     0     0     0  
>=65 years     0     0     0  
Age  
[units: days]
Mean ± Standard Deviation
  5.1  ± 4.1     5.0  ± 4.0     5.1  ± 4.0  
Gender  
[units: participants]
     
Female     107     102     209  
Male     168     172     340  
Region of Enrollment  
[units: participants]
     
United States     268     266     534  
Canada     7     8     15  



  Outcome Measures
  Hide All Outcome Measures

1.  Primary:   Proportion of Patients Who Died or Received a Heart Transplant   [ Time Frame: Measured at 12 months ]

Measure Type Primary
Measure Title Proportion of Patients Who Died or Received a Heart Transplant
Measure Description The primary outcome was the proportion of patients who died or had cardiac transplantation 12 months after randomization.
Time Frame Measured at 12 months  
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.
555 subjects were enrolled in the study; 5 of these subjects were excluded from analyses because a Norwood procedure was not performed; 1 subject was excluded because the subject withdrew before the 12-month follow-up, 12-month status was unknown. The data were analyzed on an intention to treat basis, subjects were analyzed as randomized.

Reporting Groups
  Description
MBTS Blalock-Taussig pulmonary artery shunt
RVPAS Right ventricular to pulmonary artery shunt

Measured Values
    MBTS     RVPAS  
Number of Participants Analyzed  
[units: participants]
  275     274  
Proportion of Patients Who Died or Received a Heart Transplant  
[units: Participants]
  100     72  


Statistical Analysis 1 for Proportion of Patients Who Died or Received a Heart Transplant
Groups [1] All groups
Method [2] Fisher Exact
P Value [3] 0.013
Risk Difference (RD) [4] -10.1
95% Confidence Interval ( -17.8 to -2.4 )
[1] Additional details about the analysis, such as null hypothesis and power calculation:
  The original sample size of 456 was based on 85% power, with a two-sided, two sample test of proportions (anticipating 28% MBTS subjects with events, 16% RVPAS subjects with events), and an alpha of 0.05. The critical p-value was 0.044 because four interim analyses were performed. The target trial size was increased from 466 to 554 to account for crossovers. The stopping boundary was crossed at the 4th interim look; however, the trial was not halted, because all subjects were enrolled.
[2] Other relevant method information, such as adjustments or degrees of freedom:
  No text entered.
[3] Additional information, such as whether or not the p-value is adjusted for multiple comparisons and the a priori threshold for statistical significance:
  No text entered.
[4] Other relevant estimation information:
  The risk difference is defined as the percent of subjects with events in the RVPAS group minus the percent of subjects with events in the MBTS group.



2.  Secondary:   Proportion of Deaths or Heart Transplants Over Time From Randomization to the End of the Trial   [ Time Frame: From Randomization to the End of the Trial, an average of 32 months ]

Measure Type Secondary
Measure Title Proportion of Deaths or Heart Transplants Over Time From Randomization to the End of the Trial
Measure Description This secondary outcome was the proportion of deaths or cardiac transplantation over time from randomization to the end of the trial.
Time Frame From Randomization to the End of the Trial, an average of 32 months  
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
MBTS Blalock-Taussig pulmonary artery shunt
RVPAS Right ventricular to pulmonary artery shunt

Measured Values
    MBTS     RVPAS  
Number of Participants Analyzed  
[units: participants]
  275     274  
Proportion of Deaths or Heart Transplants Over Time From Randomization to the End of the Trial  
[units: Participants]
  107     88  


Statistical Analysis 1 for Proportion of Deaths or Heart Transplants Over Time From Randomization to the End of the Trial
Groups [1] All groups
Method [2] Log Rank
P Value [3] 0.06
[1] Additional details about the analysis, such as null hypothesis and power calculation:
  No text entered.
[2] Other relevant method information, such as adjustments or degrees of freedom:
  No text entered.
[3] Additional information, such as whether or not the p-value is adjusted for multiple comparisons and the a priori threshold for statistical significance:
  No text entered.



3.  Secondary:   Echocardiographic Measures of Heart Size and Function: Right Ventricle (RV) End-diastolic Volume Indexed to Body Surface Area (BSA)   [ Time Frame: Measured post-Norwood, an average of 17 days post-Norwood ]

Measure Type Secondary
Measure Title Echocardiographic Measures of Heart Size and Function: Right Ventricle (RV) End-diastolic Volume Indexed to Body Surface Area (BSA)
Measure Description Right ventricular end-diastolic volume indexed to BSA^1.3. Echocardiograms were performed at time points relative to the two palliative surgeries (post-Norwood and pre-Stage II) as well as at the specific time point of 14 months of age.
Time Frame Measured post-Norwood, an average of 17 days post-Norwood  
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.
241 and 239 patients remained in the MBTS and RVPAS groups at the time of the post-Norwood echo. Of these, some patients did not have acceptable echos or measures were not able to be obtained to calculate the volumes.

Reporting Groups
  Description
MBTS Blalock-Taussig pulmonary artery shunt
RVPAS Right ventricular to pulmonary artery shunt

Measured Values
    MBTS     RVPAS  
Number of Participants Analyzed  
[units: participants]
  173     194  
Echocardiographic Measures of Heart Size and Function: Right Ventricle (RV) End-diastolic Volume Indexed to Body Surface Area (BSA)  
[units: ml/m^2.6]
Median ( Inter-Quartile Range )
  91.2  
  ( 76.3 to 108.1 )  
  86.5  
  ( 73.8 to 104.3 )  


Statistical Analysis 1 for Echocardiographic Measures of Heart Size and Function: Right Ventricle (RV) End-diastolic Volume Indexed to Body Surface Area (BSA)
Groups [1] All groups
Method [2] Wilcoxon (Mann-Whitney)
P Value [3] 0.09
[1] Additional details about the analysis, such as null hypothesis and power calculation:
  No text entered.
[2] Other relevant method information, such as adjustments or degrees of freedom:
  No text entered.
[3] Additional information, such as whether or not the p-value is adjusted for multiple comparisons and the a priori threshold for statistical significance:
  No text entered.



4.  Secondary:   Echocardiographic Measures of Heart Size and Function: RV End-diastolic Volume Indexed to BSA   [ Time Frame: Measured pre-stage II surgery, an average of 15 days pre-stage II surgery ]

Measure Type Secondary
Measure Title Echocardiographic Measures of Heart Size and Function: RV End-diastolic Volume Indexed to BSA
Measure Description Right ventricular end-diastolic volume indexed to BSA^1.3. Echocardiograms were performed at time points relative to the two palliative surgeries (post-Norwood and pre-Stage II) as well as at the specific time point of 14 months of age.
Time Frame Measured pre-stage II surgery, an average of 15 days pre-stage II surgery  
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.
181 and 214 patients remained in the MBTS and RVPAS groups at the time of the pre-Stage II echo. Of these, some patients did not have acceptable echos or not all measures were able to be obtained to calculate the volumes.

Reporting Groups
  Description
MBTS Blalock-Taussig pulmonary artery shunt
RVPAS Right ventricular to pulmonary artery shunt

Measured Values
    MBTS     RVPAS  
Number of Participants Analyzed  
[units: participants]
  127     151  
Echocardiographic Measures of Heart Size and Function: RV End-diastolic Volume Indexed to BSA  
[units: ml/m^2.6]
Median ( Inter-Quartile Range )
  113.4  
  ( 94.1 to 138.6 )  
  105.2  
  ( 86.8 to 125.3 )  


Statistical Analysis 1 for Echocardiographic Measures of Heart Size and Function: RV End-diastolic Volume Indexed to BSA
Groups [1] All groups
Method [2] Wilcoxon (Mann-Whitney)
P Value [3] 0.009
[1] Additional details about the analysis, such as null hypothesis and power calculation:
  No text entered.
[2] Other relevant method information, such as adjustments or degrees of freedom:
  No text entered.
[3] Additional information, such as whether or not the p-value is adjusted for multiple comparisons and the a priori threshold for statistical significance:
  No text entered.



5.  Secondary:   Echocardiographic Measures of Heart Size and Function: RV End-diastolic Volume Indexed to BSA   [ Time Frame: Measured at 14 months of age ]

Measure Type Secondary
Measure Title Echocardiographic Measures of Heart Size and Function: RV End-diastolic Volume Indexed to BSA
Measure Description Right ventricular end-diastolic volume indexed to BSA^1.3. Echocardiograms were performed at time points relative to the two palliative surgeries (post-Norwood and pre-Stage II) as well as at the specific time point of 14 months of age.
Time Frame Measured at 14 months of age  
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.
184 and 179 patients remained in the MBTS and RVPAS groups at the 14-months of age echo. Of these, some patients did not have acceptable echos or not all measures were able to be obtained to calculate the volumes.

Reporting Groups
  Description
MBTS Blalock-Taussig pulmonary artery shunt
RVPAS Right ventricular to pulmonary artery shunt

Measured Values
    MBTS     RVPAS  
Number of Participants Analyzed  
[units: participants]
  107     129  
Echocardiographic Measures of Heart Size and Function: RV End-diastolic Volume Indexed to BSA  
[units: ml/m^2.6]
Median ( Inter-Quartile Range )
  83.3  
  ( 69.4 to 100.3 )  
  88.9  
  ( 72.6 to 104.7 )  


Statistical Analysis 1 for Echocardiographic Measures of Heart Size and Function: RV End-diastolic Volume Indexed to BSA
Groups [1] All groups
Method [2] Wilcoxon (Mann-Whitney)
P Value [3] 0.07
[1] Additional details about the analysis, such as null hypothesis and power calculation:
  No text entered.
[2] Other relevant method information, such as adjustments or degrees of freedom:
  No text entered.
[3] Additional information, such as whether or not the p-value is adjusted for multiple comparisons and the a priori threshold for statistical significance:
  No text entered.



6.  Secondary:   Echocardiographic Measures of Heart Size and Function: RV End-systolic Volume Indexed to BSA   [ Time Frame: Measured post-Norwood, an average of 17 days post-Norwood ]

Measure Type Secondary
Measure Title Echocardiographic Measures of Heart Size and Function: RV End-systolic Volume Indexed to BSA
Measure Description Right ventricular end-systolic volume indexed to BSA. Echocardiograms were performed at time points relative to the two palliative surgeries (post-Norwood and pre-Stage II) as well as at the specific time point of 14 months of age.
Time Frame Measured post-Norwood, an average of 17 days post-Norwood  
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.
241 and 239 patients remained in the MBTS and RVPAS groups at the time of the post-Norwood echo. Of these, some patients did not have acceptable echos or not all measures were able to be obtained to calculate the volumes.

Reporting Groups
  Description
MBTS Blalock-Taussig pulmonary artery shunt
RVPAS Right ventricular to pulmonary artery shunt

Measured Values
    MBTS     RVPAS  
Number of Participants Analyzed  
[units: participants]
  173     194  
Echocardiographic Measures of Heart Size and Function: RV End-systolic Volume Indexed to BSA  
[units: ml/m^2.6]
Median ( Inter-Quartile Range )
  50.1  
  ( 40.2 to 61.9 )  
  44.2  
  ( 36.6 to 57.1 )  


Statistical Analysis 1 for Echocardiographic Measures of Heart Size and Function: RV End-systolic Volume Indexed to BSA
Groups [1] All groups
Method [2] Wilcoxon (Mann-Whitney)
P Value [3] 0.001
[1] Additional details about the analysis, such as null hypothesis and power calculation:
  No text entered.
[2] Other relevant method information, such as adjustments or degrees of freedom:
  No text entered.
[3] Additional information, such as whether or not the p-value is adjusted for multiple comparisons and the a priori threshold for statistical significance:
  No text entered.



7.  Secondary:   Echocardiographic Measures of Heart Size and Function: RV End-systolic Volume Indexed to BSA   [ Time Frame: Measured pre-stage II surgery, an average of 15 days pre-stage II surgery ]

Measure Type Secondary
Measure Title Echocardiographic Measures of Heart Size and Function: RV End-systolic Volume Indexed to BSA
Measure Description Right ventricular end-systolic volume indexed to BSA^1.3. Echocardiograms were performed at time points relative to the two palliative surgeries (post-Norwood and pre-Stage II) as well as at the specific time point of 14 months of age.
Time Frame Measured pre-stage II surgery, an average of 15 days pre-stage II surgery  
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.
181 and 214 patients remained in the MBTS and RVPAS groups at the time of the pre-Stage II echo. Of these, some patients did not have acceptable echos or not all measures were able to be obtained to calculate the volumes.

Reporting Groups
  Description
MBTS Blalock-Taussig pulmonary artery shunt
RVPAS Right ventricular to pulmonary artery shunt

Measured Values
    MBTS     RVPAS  
Number of Participants Analyzed  
[units: participants]
  127     151  
Echocardiographic Measures of Heart Size and Function: RV End-systolic Volume Indexed to BSA  
[units: ml/m^2.6]
Median ( Inter-Quartile Range )
  63.2  
  ( 49.9 to 83.0 )  
  57.9  
  ( 44.6 to 69.9 )  


Statistical Analysis 1 for Echocardiographic Measures of Heart Size and Function: RV End-systolic Volume Indexed to BSA
Groups [1] All groups
Method [2] Wilcoxon (Mann-Whitney)
P Value [3] 0.004
[1] Additional details about the analysis, such as null hypothesis and power calculation:
  Wilcoxon rank-sum test
[2] Other relevant method information, such as adjustments or degrees of freedom:
  No text entered.
[3] Additional information, such as whether or not the p-value is adjusted for multiple comparisons and the a priori threshold for statistical significance:
  No text entered.



8.  Secondary:   Echocardiographic Measures of Heart Size and Function: RV End-systolic Volume Indexed to BSA   [ Time Frame: Measured at 14 months of age ]

Measure Type Secondary
Measure Title Echocardiographic Measures of Heart Size and Function: RV End-systolic Volume Indexed to BSA
Measure Description Right ventricular end-systolic volume indexed to BSA^1.3. Echocardiograms were performed at time points relative to the two palliative surgeries (post-Norwood and pre-Stage II) as well as at the specific time point of 14 months of age.
Time Frame Measured at 14 months of age  
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.
184 and 179 patients remained in the MBTS and RVPAS groups at the 14-months of age echo. Of these, some patients did not have acceptable echos or not all measures were able to be obtained to calculate the volumes.

Reporting Groups
  Description
MBTS Blalock-Taussig pulmonary artery shunt
RVPAS Right ventricular to pulmonary artery shunt

Measured Values
    MBTS     RVPAS  
Number of Participants Analyzed  
[units: participants]
  107     129  
Echocardiographic Measures of Heart Size and Function: RV End-systolic Volume Indexed to BSA  
[units: ml/m^2.6]
Median ( Inter-Quartile Range )
  45.4  
  ( 38.5 to 56.6 )  
  50.5  
  ( 39.7 to 60.8 )  


Statistical Analysis 1 for Echocardiographic Measures of Heart Size and Function: RV End-systolic Volume Indexed to BSA
Groups [1] All groups
Method [2] Wilcoxon (Mann-Whitney)
P Value [3] 0.10
[1] Additional details about the analysis, such as null hypothesis and power calculation:
  No text entered.
[2] Other relevant method information, such as adjustments or degrees of freedom:
  No text entered.
[3] Additional information, such as whether or not the p-value is adjusted for multiple comparisons and the a priori threshold for statistical significance:
  No text entered.



9.  Secondary:   Echocardiographic Measures of Heart Size and Function: RV Ejection Fraction   [ Time Frame: Measured post-Norwood, an average of 17 days post-Norwood ]

Measure Type Secondary
Measure Title Echocardiographic Measures of Heart Size and Function: RV Ejection Fraction
Measure Description Right ventricular ejection fraction: 100 x (RV end-diastolic volume - RV end-systolic volume)/RV end-diastolic volume. Echocardiograms were performed at time points relative to the two palliative surgeries (post-Norwood and pre-Stage II) as well as at the specific time point of 14 months of age.
Time Frame Measured post-Norwood, an average of 17 days post-Norwood  
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.
241 and 239 patients remained in the MBTS and RVPAS groups at the time of the post-Norwood echo. Of these, some patients did not have acceptable echos or not all measures were able to be obtained to calculate the volumes used to calculate the ejection fraction.

Reporting Groups
  Description
MBTS Blalock-Taussig pulmonary artery shunt
RVPAS Right ventricular to pulmonary artery shunt

Measured Values
    MBTS     RVPAS  
Number of Participants Analyzed  
[units: participants]
  173     194  
Echocardiographic Measures of Heart Size and Function: RV Ejection Fraction  
[units: Percentage of RV end-diastolic volume]
Mean ± Standard Deviation
  44.5  ± 7.6     48.5  ± 7.6  


Statistical Analysis 1 for Echocardiographic Measures of Heart Size and Function: RV Ejection Fraction
Groups [1] All groups
Method [2] t-test, 2 sided
P Value [3] <0.001
[1] Additional details about the analysis, such as null hypothesis and power calculation:
  No text entered.
[2] Other relevant method information, such as adjustments or degrees of freedom:
  No text entered.
[3] Additional information, such as whether or not the p-value is adjusted for multiple comparisons and the a priori threshold for statistical significance:
  No text entered.



10.  Secondary:   Echocardiographic Measures of Heart Size and Function: RV Ejection Fraction   [ Time Frame: Measured pre-stage II surgery, an average of 15 days pre-stage II surgery ]

Measure Type Secondary
Measure Title Echocardiographic Measures of Heart Size and Function: RV Ejection Fraction
Measure Description Right ventricular ejection fraction: 100 x (RV end-diastolic volume - RV end-systolic volume)/RV end-diastolic volume. Echocardiograms were performed at time points relative to the two palliative surgeries (post-Norwood and pre-Stage II) as well as at the specific time point of 14 months of age.
Time Frame Measured pre-stage II surgery, an average of 15 days pre-stage II surgery  
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.
181 and 214 patients remained in the MBTS and RVPAS groups at the time of the pre-Stage II echo. Of these, some patients did not have acceptable echos or not all measures were able to be obtained to calculate the volumes.

Reporting Groups
  Description
MBTS Blalock-Taussig pulmonary artery shunt
RVPAS Right ventricular to pulmonary artery shunt

Measured Values
    MBTS     RVPAS  
Number of Participants Analyzed  
[units: participants]
  128     151  
Echocardiographic Measures of Heart Size and Function: RV Ejection Fraction  
[units: Percentage of RV end-diastolic volume]
Mean ± Standard Deviation
  42.9  ± 7.9     44.7  ± 8.3  


Statistical Analysis 1 for Echocardiographic Measures of Heart Size and Function: RV Ejection Fraction
Groups [1] All groups
Method [2] t-test, 2 sided
P Value [3] 0.07
[1] Additional details about the analysis, such as null hypothesis and power calculation:
  No text entered.
[2] Other relevant method information, such as adjustments or degrees of freedom:
  No text entered.
[3] Additional information, such as whether or not the p-value is adjusted for multiple comparisons and the a priori threshold for statistical significance:
  No text entered.



11.  Secondary:   Echocardiographic Measures of Heart Size and Function: RV Ejection Fraction   [ Time Frame: Measured at 14 months of age ]

Measure Type Secondary
Measure Title Echocardiographic Measures of Heart Size and Function: RV Ejection Fraction
Measure Description Right ventricular ejection fraction: 100 x (RV end-diastolic volume - RV end-systolic volume)/RV end-diastolic volume. Echocardiograms were performed at time points relative to the two palliative surgeries (post-Norwood and pre-Stage II) as well as at the specific time point of 14 months of age.
Time Frame Measured at 14 months of age  
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.
184 and 179 patients remained in the MBTS and RVPAS groups at the 14-months of age echo. Of these, some patients did not have acceptable echos or not all measures were able to be obtained to calculate the volumes.

Reporting Groups
  Description
MBTS Blalock-Taussig pulmonary artery shunt
RVPAS Right ventricular to pulmonary artery shunt

Measured Values
    MBTS     RVPAS  
Number of Participants Analyzed  
[units: participants]
  107     129  
Echocardiographic Measures of Heart Size and Function: RV Ejection Fraction  
[units: Percentage of RV end-diastolic volume]
Mean ± Standard Deviation
  42.7  ± 7.4     42.7  ± 8.0  


Statistical Analysis 1 for Echocardiographic Measures of Heart Size and Function: RV Ejection Fraction
Groups [1] All groups
Method [2] t-test, 2 sided
P Value [3] 0.97
[1] Additional details about the analysis, such as null hypothesis and power calculation:
  No text entered.
[2] Other relevant method information, such as adjustments or degrees of freedom:
  No text entered.
[3] Additional information, such as whether or not the p-value is adjusted for multiple comparisons and the a priori threshold for statistical significance:
  No text entered.



12.  Secondary:   Angiographic Findings: Left Pulmonary Artery Size   [ Time Frame: Measured pre-stage II surgery, on average 26 days prior to stage II palliation ]

Measure Type Secondary
Measure Title Angiographic Findings: Left Pulmonary Artery Size
Measure Description Diameter of distal left pulmonary artery. Angiograms were performed at the time points relative to the second palliative surgery (pre-Stage II).
Time Frame Measured pre-stage II surgery, on average 26 days prior to stage II palliation  
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.
These patients had pre-stage II palliation visits with acceptable cardiac catheterizations.

Reporting Groups
  Description
MBTS Blalock-Taussig pulmonary artery shunt
RVPAS Right ventricular to pulmonary artery shunt

Measured Values
    MBTS     RVPAS  
Number of Participants Analyzed  
[units: participants]
  159     191  
Angiographic Findings: Left Pulmonary Artery Size  
[units: mm]
Mean ± Standard Deviation
  5.2  ± 1.5     5.4  ± 1.7  


Statistical Analysis 1 for Angiographic Findings: Left Pulmonary Artery Size
Groups [1] All groups
Method [2] t-test, 2 sided
P Value [3] 0.54
[1] Additional details about the analysis, such as null hypothesis and power calculation:
  No text entered.
[2] Other relevant method information, such as adjustments or degrees of freedom:
  No text entered.
[3] Additional information, such as whether or not the p-value is adjusted for multiple comparisons and the a priori threshold for statistical significance:
  No text entered.



13.  Secondary:   Angiographic Findings: Right Pulmonary Artery Size   [ Time Frame: Measured pre-stage II surgery, on average 26 days prior to stage II palliation ]

Measure Type Secondary
Measure Title Angiographic Findings: Right Pulmonary Artery Size
Measure Description Diameter of distal right pulmonary artery. Angiograms were performed at the time points relative to the second palliative surgery (pre-Stage II).
Time Frame Measured pre-stage II surgery, on average 26 days prior to stage II palliation  
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.
These patients had pre-stage II palliation visits with acceptable cardiac catheterizations.

Reporting Groups
  Description
MBTS Blalock-Taussig pulmonary artery shunt
RVPAS Right ventricular to pulmonary artery shunt

Measured Values
    MBTS     RVPAS  
Number of Participants Analyzed  
[units: participants]
  159     191  
Angiographic Findings: Right Pulmonary Artery Size  
[units: mm]
Mean ± Standard Deviation
  6.1  ± 2.0     5.4  ± 1.7  


Statistical Analysis 1 for Angiographic Findings: Right Pulmonary Artery Size
Groups [1] All groups
Method [2] t-test, 2 sided
P Value [3] <0.001
[1] Additional details about the analysis, such as null hypothesis and power calculation:
  No text entered.
[2] Other relevant method information, such as adjustments or degrees of freedom:
  No text entered.
[3] Additional information, such as whether or not the p-value is adjusted for multiple comparisons and the a priori threshold for statistical significance:
  No text entered.



14.  Secondary:   Unintended Cardiovascular Interventional Procedures   [ Time Frame: From Randomization to 12 months ]

Measure Type Secondary
Measure Title Unintended Cardiovascular Interventional Procedures
Measure Description Unintended cardiovascular procedures included balloon dilation of the shunt or branch pulmonary arteries, stent placement in the shunt or branch pulmonary arteries, shunt revision, crossover between MBTS and RVPAS shunt, balloon dilation, stent placement or surgical revisions of the neo-aorta, and pulmonary artery reconstructions, other than those undertaken as a standard component of the stage II procedure. The number of cardiovascular procedures was analyzed; trial participants may have had more than one unintended cardiovascular. procedure.
Time Frame From Randomization to 12 months  
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
MBTS Blalock-Taussig pulmonary artery shunt
RVPAS Right ventricular to pulmonary artery shunt

Measured Values
    MBTS     RVPAS  
Number of Participants Analyzed  
[units: participants]
  275     274  
Unintended Cardiovascular Interventional Procedures  
[units: procedures]
  192     252  


Statistical Analysis 1 for Unintended Cardiovascular Interventional Procedures
Groups [1] All groups
Method [2] Poisson regression
P Value [3] 0.003
[1] Additional details about the analysis, such as null hypothesis and power calculation:
  No text entered.
[2] Other relevant method information, such as adjustments or degrees of freedom:
  The offset parameter used in the poisson regression was the log of the number of patients in each treatment arm.
[3] Additional information, such as whether or not the p-value is adjusted for multiple comparisons and the a priori threshold for statistical significance:
  No text entered.



15.  Secondary:   Complications: Total Number Experienced During Norwood Hospitalization   [ Time Frame: Norwood Hospitalization, an average of 36 days ]

Measure Type Secondary
Measure Title Complications: Total Number Experienced During Norwood Hospitalization
Measure Description Complications, reported as 'Other Adverse Events' in the safety section, are those adverse events that were not considered serious. Many normal peri-operative occurrences meet the standard criteria of an adverse event; therefore, for this trial a serious adverse event was (a) death, (b) acute shunt failure requiring intervention, (c) cardiac arrest requiring CPR and medications, (d) cardiopulmonary insufficiency requiring ECMO, (e) cardiovascular re-operation (unplanned), (f) necrotizing enterocolitis requiring laparotomy, and (g) any event considered by the study investigator as serious.
Time Frame Norwood Hospitalization, an average of 36 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
MBTS Blalock-Taussig pulmonary artery shunt
RVPAS Right ventricular to pulmonary artery shunt

Measured Values
    MBTS     RVPAS  
Number of Participants Analyzed  
[units: participants]
  275     274  
Complications: Total Number Experienced During Norwood Hospitalization  
[units: complications]
  850     792  


Statistical Analysis 1 for Complications: Total Number Experienced During Norwood Hospitalization
Groups [1] All groups
Method [2] Poisson Regression
P Value [3] 0.20
[1] Additional details about the analysis, such as null hypothesis and power calculation:
  No text entered.
[2] Other relevant method information, such as adjustments or degrees of freedom:
  The offset parameter used in this analysis was the log of the number of patients per treatment arm.
[3] Additional information, such as whether or not the p-value is adjusted for multiple comparisons and the a priori threshold for statistical significance:
  No text entered.



16.  Secondary:   Complications: Total Number Experienced From Norwood Discharge to Stage II Discharge   [ Time Frame: From Norwood Discharge to Stage II discharge, an average of 4.2 months ]

Measure Type Secondary
Measure Title Complications: Total Number Experienced From Norwood Discharge to Stage II Discharge
Measure Description Complications, reported as 'Other Adverse Events' in the safety section, are those adverse events that were not considered serious. Many normal peri-operative occurrences meet the standard criteria of an adverse event; therefore, for this trial a serious adverse event was (a) death, (b) acute shunt failure requiring intervention, (c) cardiac arrest requiring CPR and medications, (d) cardiopulmonary insufficiency requiring ECMO, (e) cardiovascular re-operation (unplanned), (f) necrotizing enterocolitis requiring laparotomy, and (g) any event considered by the study investigator as serious.
Time Frame From Norwood Discharge to Stage II discharge, an average of 4.2 months  
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.
These numbers reflect those patients who were discharged from the hospital after the Norwood procedure and were transplant free.

Reporting Groups
  Description
MBTS Blalock-Taussig pulmonary artery shunt
RVPAS Right ventricular to pulmonary artery shunt

Measured Values
    MBTS     RVPAS  
Number of Participants Analyzed  
[units: participants]
  219     233  
Complications: Total Number Experienced From Norwood Discharge to Stage II Discharge  
[units: complications]
  367     515  


Statistical Analysis 1 for Complications: Total Number Experienced From Norwood Discharge to Stage II Discharge
Groups [1] All groups
Method [2] Poisson Regression
P Value [3] 0.002
[1] Additional details about the analysis, such as null hypothesis and power calculation:
  No text entered.
[2] Other relevant method information, such as adjustments or degrees of freedom:
  The offset parameter used in this analysis was the log of the number of patients per treatment arm.
[3] Additional information, such as whether or not the p-value is adjusted for multiple comparisons and the a priori threshold for statistical significance:
  No text entered.



17.  Secondary:   Complications: Total Number Experienced From Stage II Discharge to 14 Months of Age   [ Time Frame: From Stage II Discharge to 14 Months of Age, an average of 8.9 months ]

Measure Type Secondary
Measure Title Complications: Total Number Experienced From Stage II Discharge to 14 Months of Age
Measure Description Complications, reported as 'Other Adverse Events' in the safety section, are those adverse events that were not considered serious. Many normal peri-operative occurrences meet the standard criteria of an adverse event; therefore, for this trial a serious adverse event was (a) death, (b) acute shunt failure requiring intervention, (c) cardiac arrest requiring CPR and medications, (d) cardiopulmonary insufficiency requiring ECMO, (e) cardiovascular re-operation (unplanned), (f) necrotizing enterocolitis requiring laparotomy, and (g) any event considered by the study investigator as serious.
Time Frame From Stage II Discharge to 14 Months of Age, an average of 8.9 months  
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.
These numbers reflect the number of patients who were discharged from the hospital after the Stage 2 procedure and were transplant-free.

Reporting Groups
  Description
MBTS Blalock-Taussig pulmonary artery shunt
RVPAS Right ventricular to pulmonary artery shunt

Measured Values
    MBTS     RVPAS  
Number of Participants Analyzed  
[units: participants]
  176     202  
Complications: Total Number Experienced From Stage II Discharge to 14 Months of Age  
[units: complications]
  81     145  


Statistical Analysis 1 for Complications: Total Number Experienced From Stage II Discharge to 14 Months of Age
Groups [1] All groups
Method [2] Poisson regression
P Value [3] 0.03
[1] Additional details about the analysis, such as null hypothesis and power calculation:
  No text entered.
[2] Other relevant method information, such as adjustments or degrees of freedom:
  The offset parameter used in this analysis was the log of the number of patients per treatment arm.
[3] Additional information, such as whether or not the p-value is adjusted for multiple comparisons and the a priori threshold for statistical significance:
  No text entered.




  Serious Adverse Events


  Other Adverse Events


  More Information
  Hide More Information

Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.


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
As in all surgical trials comparing two operations, it was not possible to blind the personnel caring for the subjects to the intervention. However, the components of the primary endpoint, death and transplantation, are discrete objective events.  


Results Point of Contact:  
Name/Title: Lynn Sleeper, ScD
Organization: New England Research Institutes
phone: 617-972-3235
e-mail: lsleeper@neriscience.com


Publications of Results:

Other Publications:
Publications automatically indexed to this study:


Responsible Party: Lynn Sleeper, ScD, PI, New England Research Institutes, Watertown, MA
ClinicalTrials.gov Identifier: NCT00115934     History of Changes
Other Study ID Numbers: 194, U01HL068270, U01HL068269, U01HL068279, U01HL068281, U01HL068285, U01HL068288, U01HL068290, U01HL068292
Study First Received: June 26, 2005
Results First Received: September 14, 2010
Last Updated: August 2, 2013
Health Authority: United States: Federal Government