Pediatric REPlAcement of the PulmonaRy ValvE in Tetralogy of Fallot -

NCT03634072 | Terminated Results

• 2 other identifiers: 18-015046R34HL142142-01
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 6

Brief Summary

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 (PVR) 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.

Conditions

Tetralogy of Fallot

Outcome Measures

Primary Outcomes (1)

• Number of Participants Randomized to PVR Via Catheter or Surgery

  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.

  2-3 years

Secondary Outcomes (6)

• Exercise Performances

  12-18 months

• Prevalence of Arrhythmias

  12-18 months

• Effects of Pulmonary Valve Replacement (PVR) on Diffuse Fibrosis

  12-18 months

• Effects of PVR on Exercise in the Magnetic Resonance (MR) Scanner

  12-18 months

• Effects of PVR on Biventricular Strain

  12-18 months

Study Arms (2)

PVR Arm

OTHER

PVR arm will undergo PVR via catheter or surgery

Procedure: PVR

No PVR

NO INTERVENTION

No PVR group will continue with medical management

Interventions

PVR PROCEDURE

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

PVR Arm

Eligibility Criteria

• Age: 13 Years - 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Males or females with repaired Tetralogy of Fallot (TOF), currently between 13 and 21 years of age.
• 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 (if not available this will be skipped); there will be no indexed Right Ventricular end-systolic volume (RVESVi) criteria; by defining RVEDVi and RVEF, Investigators will be inherently defining RVESVi
• On clinical echocardiogram: RV outflow tract peak velocity \< 3 meters/second (if not available this will be skipped), at least mild pulmonary insufficiency and tricuspid regurgitation with an RV pressure estimate \< 1/2 systemic pressure.
• On Exercise Stress Test (EST), aerobic capacity \> 60% of predicted.
• No Q-wave, R-wave, S-wave (QRS) duration criteria on ECG.

You may not qualify if:

• Any condition judged by the patient's physician that would cause this trial to be detrimental to the patient.
• 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.
• Unilateral branch pulmonary artery stenosis (one lung receives \< 25% of total flow)
• Contraindication to non-sedated exercise CMR (e.g. pacemaker/implanted cardioverter defibrillator); need for sedation
• If data available, moderate or greater tricuspid regurgitation on echocardiogram or CMR or Qp/Qs \> 1.5
• Significant strokes/hemiplegia or inability to exercise
• Genetic syndrome/developmental delay which would make QOL and EST date uninterpretable
• Pregnancy
• Previous pulmonary valve replacement (PVR)

Sponsors & Collaborators

• Children's Hospital of Philadelphia: lead
• National Heart, Lung, and Blood Institute (NHLBI): collaborator

Study Sites (6)

Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States

Location

Children's Healthcare of Atlanta

Atlanta, Georgia, 30322, United States

Location

Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

Northwestern University

Chicago, Illinois, 60611, United States

Location

Cincinnati Children's Hosptial Medical Center

Cincinnati, Ohio, 45229, United States

Location

The Childrens Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

MeSH Terms

Conditions

Tetralogy of Fallot

Condition Hierarchy (Ancestors)

Heart Defects, Congenital; Cardiovascular Abnormalities; Cardiovascular Diseases; Heart Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Limitations and Caveats

Study terminated early.

Results Point of Contact

• Title: Dr Mark Fogel
• Organization: Children's Hospital of Philadelphia

fogel@chop.edu

215-590-7566

Study Officials

• Mark Fogel, MD

  The Childrens Hospital of Philadelphia

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: INVESTIGATOR
• Masking Details: 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.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Subjects will be randomized to PVR or no PVR

Study Record Dates

• First Submitted: August 2, 2018
• First Posted: August 16, 2018
• Study Start: July 6, 2018
• Primary Completion: January 16, 2021
• Study Completion: January 16, 2021
• Last Updated: October 6, 2023
• Results First Posted: October 6, 2023

Record last verified: 2023-09

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Time Frame: 2-3 years
• Access Criteria: All future studies using these patients will require separate Institutional Review Board (IRB) approved protocols and consent forms. Only investigators' on this protocol and assigned research staff on this protocol will have access to the data

Locations

US (6)