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
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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[1] 279 randomized to MBTS, but 3 did not have a Norwood surgery; one was withdrawn-with limited data.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 ]
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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 ]

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 ]

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 ]

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

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 ]

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 ]

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

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

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 ]

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

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

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

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

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

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 ]

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 ]


  Serious Adverse Events


  Other Adverse Events


  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.


  More Information
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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.


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