Trial Outcomes & Findings for Pediatric REPlAcement of the PulmonaRy ValvE in Tetralogy of Fallot - (NCT NCT03634072)

NCT ID: NCT03634072

Last Updated: 2023-10-06

Results Overview

Participants will be randomized by computer by the statistician at the Data Coordinating Center (DCC) at Northwestern University (NU) who will maintain the schedule; this will be a 2:1 randomization of PVR to no-PVR. There will be no blinding and the assignment will not be concealed from the investigators. There will be no stratifications within each group.

• Recruitment status: TERMINATED
• Study phase: NA
• Target enrollment: 1 participants
• Primary outcome timeframe: 2-3 years
• Results posted on: 2023-10-06

Participant Flow

Patient was enrolled in 2020 in clinic

No pre-assignment issues were found

Participant milestones

Measure	PVR Arm PVR arm will undergo PVR via catheter or surgery PVR: Subjects will undergo PVR via surgery or cardiac catheterization. PVR using cardiac catheterization may require a much shorter hospital stay than traditional heart surgery. If the valve is repaired by surgery, this will require open heart surgery to directly implant a pulmonary valve. 1 patient was enrolled in this arm from Emory University	No PVR No PVR group will continue with medical management No patients were enrolled in this group.
Overall Study STARTED	1	0
Overall Study COMPLETED	0	0
Overall Study NOT COMPLETED	1	0

Reasons for withdrawal

Measure	PVR Arm PVR arm will undergo PVR via catheter or surgery PVR: Subjects will undergo PVR via surgery or cardiac catheterization. PVR using cardiac catheterization may require a much shorter hospital stay than traditional heart surgery. If the valve is repaired by surgery, this will require open heart surgery to directly implant a pulmonary valve. 1 patient was enrolled in this arm from Emory University	No PVR No PVR group will continue with medical management No patients were enrolled in this group.
Overall Study Early termination of overall study	1	0

Baseline Characteristics

Pediatric REPlAcement of the PulmonaRy ValvE in Tetralogy of Fallot -

Baseline characteristics by cohort

Measure	PVR Arm n=1 Participants PVR arm will undergo PVR via catheter or surgery PVR: Subjects will undergo PVR via surgery or cardiac catheterization. PVR using cardiac catheterization may require a much shorter hospital stay than traditional heart surgery. If the valve is repaired by surgery, this will require open heart surgery to directly implant a pulmonary valve. 1 patient was enrolled in this arm from Emory University	No PVR No PVR group will continue with medical management No patients were enrolled in this group.	Total n=1 Participants Total of all reporting groups
Age, Categorical <=18 years	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Continuous	17 years STANDARD_DEVIATION 0 • n=5 Participants	—	16 years STANDARD_DEVIATION 0 • n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	—	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	—	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	—	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	—	0 Participants n=5 Participants
Race (NIH/OMB) White	1 Participants n=5 Participants	—	1 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	—	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	—	0 Participants n=5 Participants
Region of Enrollment United States	1 participants n=5 Participants	—	1 participants n=5 Participants

PRIMARY outcome

Timeframe: 2-3 years

Population: One subject was randomized to the PVR arm; however, no data was collected to analyze

Participants will be randomized by computer by the statistician at the Data Coordinating Center (DCC) at Northwestern University (NU) who will maintain the schedule; this will be a 2:1 randomization of PVR to no-PVR. There will be no blinding and the assignment will not be concealed from the investigators. There will be no stratifications within each group.

Outcome measures

Measure	PVR Arm n=1 Participants PVR arm will undergo PVR via catheter or surgery PVR: Subjects will undergo PVR via surgery or cardiac catheterization. PVR using cardiac catheterization may require a much shorter hospital stay than traditional heart surgery. If the valve is repaired by surgery, this will require open heart surgery to directly implant a pulmonary valve	No PVR No PVR group will continue with medical management
Number of Participants Randomized to PVR Via Catheter or Surgery	0 PVR via catheter or surgery	—

SECONDARY outcome

Timeframe: 12-18 months

Population: No data were collected and no intervention completed in the PVR arm.

This will be determined by exercise test performed to assess parameters such as oxygen consumption (VO2) at ventilatory anaerobic threshold (VAT) and normalized for age

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 12-18 months

Population: No data were collected and no intervention completed in the PVR arm.

This will be determined by reviewing Holter monitoring data to assess the prevalence of arrhythmias. This will also provide the endpoints for the larger, longer term trial

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 12-18 months

Population: No data were collected and no intervention completed in the PVR arm.

This will be measured by obtaining preliminary data on the difference between patients randomized to PVR and no PVR in regard to diffuse fibrosis (DF).

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 12-18 months

Population: No data were collected and no intervention completed in the PVR arm.

Mechanisms of the effects of PVR in the definitive trial will be measured by obtaining preliminary data on the difference between patients randomized to PVR and no PVR in regard to performing exercise cardiac magnetic resonance (CMR).

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 12-18 months

Population: No data were collected and no intervention completed in the PVR arm.

Mechanisms of the effects of PVR in the definitive trial will be measured by obtaining preliminary data on biventricular strain.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 12-18 months

Population: No data were collected and no intervention completed in the PVR arm.

Quality of life will be measured using the Pediatric Cardiac Quality of Life Inventory (PCQLI) - to measure quality of life. The PCQLI has been used for over 10 years and is a validated quality of life metric. The PCQLI measures disease-specific, pediatric health related quality of life and generates 3 scores, namely, total, disease impact subscale, and psychosocial impact subscale. Each subscale score has a maximum of 50 points, and their sum yields the total score. Higher scores represent better perceived pediatric health related quality of life

Outcome measures

Outcome data not reported

Adverse Events

PVR Arm

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

No PVR

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Dr Mark Fogel

Children's Hospital of Philadelphia

Phone: 215-590-7566

Email: fogel@chop.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place