Purpose

Tetralogy of Fallot (TOF) is the most common cyanotic congenital heart defect with the vast majority of survivors of corrective surgery left with some degree of right ventricular (RV) volume overload due to pulmonary regurgitation (PR) which cause RV enlargement with right heart failure, diminished biventricular function, ventricular arrhythmia, sudden death and decreased exercise performance over time. Pulmonary valve replacement has been thought to ameliorate these complications but the timing of replacement has yet to be determined with equipoise at the moment in this decision making process. As nearly all studies in this regard are retrospective with much less data in pediatric TOF than adults, this pilot trial sets the stage to create a prospective randomized trial in the teenage years.

Condition

Eligibility

Eligible Ages
Between 13 Years and 21 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  1. Males or females with repaired Tetralogy of Fallot (TOF), currently between 13 and 21 years of age.
  2. On clinical Cardiac Magnetic Resonance (CMR) : Right Ventricular End-Diastolic Volume Index (RVEDVi) between 140 and 180 cc/m2 inclusive with Right Ventricular End-Diastolic Function (RVEF) > 40% and Left Right Ventricular End-Diastolic (LVEF ) > 50%, RV outflow tract peak velocity < 3 meters/second; there will be no indexed Right Ventricular end-systolic volume (RVESVi) criteria; by defining RVEDVi and RVEF, Investigators will be inherently defining RVESVi
  3. On clinical echocardiogram: RV outflow tract peak velocity < 3 meters/second and tricuspid regurgitation with an RV pressure estimate < 1/2 systemic pressure.
  4. On Exercise Stress Test (EST), aerobic capacity > 70% of predicted.
  5. No QRS duration criteria on ECG.

Exclusion Criteria

  1. Any condition judged by the patient's physician that would cause this trial to be detrimental to the patient.
  2. Specific forms of TOF excluded are those with endocardial cushion defects, TOF with absent pulmonary valve and TOF with multiple aorto-pulmonary collaterals requiring unifocalization.
  3. Unilateral branch pulmonary artery stenosis (one lung receives < 25% of total flow)
  4. Contraindication to non-sedated exercise CMR (e.g. pacemaker/implanted cardioverter defibrillator); need for sedation
  5. Moderate or greater tricuspid regurgitation on echocardiogram or CMR or Qp/Qs > 1.5
  6. Significant strokes/hemiplegia or inability to exercise
  7. Genetic syndrome/developmental delay which would make QOL and EST date uninterpretable
  8. Pregnancy

Study Design

Phase
N/A
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Intervention Model Description
Subjects will be randomized to PVR or no PVR
Primary Purpose
Treatment
Masking
Single (Investigator)
Masking Description
Subjects will be randomly assigned to one of two groups. One will undergo PVR with catheter or surgery (whichever is most appropriate ). The other group will continue with medical management.

Arm Groups

ArmDescriptionAssigned Intervention
Other
PVR Arm
PVR arm will undergo PVR via catheter or surgery
  • Procedure: 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 Intervention
No PVR
No PVR group will continue with medical management

Recruiting Locations

Children's National and nearby locations

Children's National Medical Center
Washington, District of Columbia 20010
Contact:
Russel Cross, MD
202-476-2020
rcross@childrensnational.org

More Details

NCT ID
NCT03634072
Status
Recruiting
Sponsor
Children's Hospital of Philadelphia

Study Contact

Mark Fogel, MD
215-590-3534
fogel@email.chop.edu

Detailed Description

The purpose of this research study is to gather information on adolescents and young adults to help understand and improve the lives of patients with TOF.

Some patients diagnosed with TOF will have a procedure called pulmonary valve replacement (PVR) and some will not. PVR is done for valves that are too damaged to be repaired. This requires a surgeon or an expert in a procedure called cardiac catheterization to replace the damaged pulmonary valve with a valve made of tissue or amechanical valve. Multiple studies in adult TOF patients have suggested that PVR may lessen many clinical symptoms but no one is sure if it truly does. There is little information about PVR in adolescence but it is thought that lessening the amount of leakage of the pulmonary valve at a young age may avoid future complications such as right heart failure or abnormal beats of your heart. There is no agreement among cardiologists, surgeons or other healthcare providers as to whether PVR truly helps avoid complications in the future and if it does, when PVR should be done. Using the information in this study, we hope to find out if PVR in adolescents is helpful in both the short and long term.

The Investigators believe the results of this study will help provide doctors with enough information to support a future large scale research study to further evaluate the outcomes PVR. This study will involve randomization to either the PVR or no PVR cohort, medical records review, exercise test and Cardiac Magnetic Resonance (CMR) , and questionnaires.

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.