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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:
National Heart, Lung, and Blood Institute (NHLBI)
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
History of Changes
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
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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
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[1] 279 randomized to MBTS, but 3 did not have a Norwood surgery; one was withdrawn-with limited data.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
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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
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1.  Primary:   Proportion of Patients Who Died or Received a Heart Transplant   [ Time Frame: Measured at 12 months ]
  Show Outcome Measure 1

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 ]
  Show Outcome Measure 2

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 ]
  Show Outcome Measure 3

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 ]
  Show Outcome Measure 4

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

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 ]
  Show Outcome Measure 6

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 ]
  Hide Outcome Measure 7

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 ]
  Show Outcome Measure 8

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

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 ]
  Show Outcome Measure 10

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

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

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

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

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

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 ]
  Show Outcome Measure 16

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 ]
  Show Outcome Measure 17


  Serious Adverse Events
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  Other Adverse Events
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  Limitations and Caveats
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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.


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