Tetralogy of FallotTOFandPVRNCT03634072

Pediatric REPlAcement of the PulmonaRy ValvE in Tetralogy of Fallot -

A study testing PVR in people with Tetralogy of Fallot, run by Children's Hospital of Philadelphia at 6 sites in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

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Trial summary

Who is it for?: Ages 13 to 21 years
Conditions the study is for: Tetralogy of Fallot
What is being tested?: PVR
Type of treatment: Surgery or procedure
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 1 participants
Where: 6 sitesUnited States
Study dates: 6 Jul 2018to 16 Jan 2021
Phase: Interventional
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 13 to 21 years
All sexes
No healthy volunteers
Child, Adult

Who may be able to join

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.

Who cannot join

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

Show all 9Show fewer

Genetic syndrome/developmental delay which would make QOL and EST date uninterpretable
Pregnancy
Previous pulmonary valve replacement (PVR)

Trial locations and contacts

6 sites in 1 country.

City, postcode or hospital

6 sites in 1 country

United States6 sites

Children's Healthcare of Atlanta
Atlanta, Georgia, 30322
Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Northwestern University
Chicago, Illinois, 60611
Cincinnati Children's Hosptial Medical Center
Cincinnati, Ohio, 45229
The Childrens Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Children's National Medical Center
Washington D.C., District of Columbia, 20010

Other trials in United States

About this study

Official title: Pediatric REPlAcement of the PulmonaRy ValvE in Tetralogy of Fallot - The PREPARE-TOF Study

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.

Read the 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 a mechanical 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.

Conditions: Tetralogy of Fallot
Being tested: PVR
Medical subject headings: Tetralogy of Fallot

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

PVRProcedure or surgery
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

Study groups in NCT03634072
Study group	Role	What they receive
PVR Arm	Other	PVR arm will undergo PVR via catheter or surgery Receives: Procedure: PVR
No PVR	No intervention	No PVR group will continue with medical management

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: 2-3 years

Secondary outcomes (6)

Exercise Performances
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
Measured: 12-18 months
Prevalence of Arrhythmias
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
Measured: 12-18 months
Effects of Pulmonary Valve Replacement (PVR) on Diffuse Fibrosis
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).
Measured: 12-18 months
Effects of PVR on Exercise in the Magnetic Resonance (MR) Scanner
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).
Measured: 12-18 months
Effects of PVR on Biventricular Strain
Mechanisms of the effects of PVR in the definitive trial will be measured by obtaining preliminary data on biventricular strain.
Measured: 12-18 months
Quality of Life (QOL)
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
Measured: 12-18 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

6 Jul 201816 Jan 2021

First posted16 Aug 2018
Study start6 Jul 2018
Primary completion16 Jan 2021
Study completion16 Jan 2021

Why it stopped: Unable to meet enrollment goal

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Tetralogy of FallotStudies of the same condition, open and under way.
More trials run by Children's Hospital of PhiladelphiaThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase N/A
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Model description: Subjects will be randomized to PVR or no PVR
Masking: Single blind
Who is masked: Investigator
Enrolment: 1 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03634072
Sponsor's protocol ID: 18-015046
Other IDs: R34HL142142-01
Acronym: TOFandPVR
Status last verified: Sep 2023
First submitted: 2 Aug 2018
Last updated: 6 Oct 2023
Results posted: Yes, on ClinicalTrials.gov
FDA-regulated drug: No
FDA-regulated device: No
Individual data sharing: Yes: The Principal Investigator (PI) is responsible for data management and accuracy of records. The PI may assign designated qualified individuals to collect the information. Only investigators' on this protocol and assigned research staff on this protocol will have access to the data. Data will be entered into a REDcap database by a member of the study team. All data and records generated during this study will be kept confidential in accordance with Institutional and HIPAA policies on subject privacy. The study team will not use such data and records for any purpose other than conducting the study. In order to keep protected health information from disclosure, each patient will be given a unique identification number. The key to this code will be kept in a locked file in one of the study investigator's offices. Any data that is transmitted to any Data Coordinating Center (DCC) will be de-identified by the enrolling site

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingPhase 4
Sodium-Glucose Cotransporter 2 Inhibitors for Repaired Tetralogy of Fallot Patients for Enhancement of Cardio-Pulmonary Status Trial
Congenital Heart Disease, Repaired Tetralogy of Fallot (rTOF)
RecruitingPhase 1
Beta-blocker Administration for Cardiomyocyte Division
Tetralogy of Fallot, Double Outlet Right Ventricle
Active, not recruiting
ALTERRA: SAPIEN 3 THV With the Alterra Adaptive Prestent
Pulmonary Disease, Transcatheter Pulmonary Valve Replacement (TPVR)
Recruiting
Investigation of Tetralogy of Fallot in Neonates
Tetralogy of Fallot (TOF), Pulmonary Stenosis
RecruitingObservational study
Volution of Pulmonary Artery Dimensions in Tetralogy of Fallot
Tetralogy of Fallot (TOF), Congenital Heart Defects

All Tetralogy of Fallot trials

Study at a glance

From the registry record, last updated by the sponsor on 6 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Pediatric REPlAcement of the PulmonaRy ValvE in Tetralogy of Fallot - The PREPARE-TOF Study
ClinicalTrials.gov ID: NCT03634072
Acronym / sponsor ID: TOFandPVR / 18-015046
Conditions: Tetralogy of Fallot
Study type: Interventional
Recruitment status: TerminatedReason stopped: Unable to meet enrollment goal
Enrolment: 1 participants (actual)
Lead sponsor: Children's Hospital of PhiladelphiaUniversity, hospital or other
Collaborators: National Heart, Lung, and Blood Institute (NHLBI)
Study start: 6 Jul 2018 (actual)
Primary completion: 16 Jan 2021 (actual)
Study completion: 16 Jan 2021 (actual)
First posted: 16 Aug 2018 (actual)
Results: Posted 6 Oct 2023

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Single blind (investigator)
Number of arms: 2
Model description: Subjects will be randomized to PVR or no PVR

Arm	Type	Receives
PVR Arm	Other	Procedure: PVRPVR arm will undergo PVR via catheter or surgery
No PVR	No intervention	–No PVR group will continue with medical management

Who may qualify

Ages: 13 to 21 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult

Key inclusion criteria (5 listed)

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.

Key exclusion criteria (9 listed)

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

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
PVRSubjects 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	Procedure or surgery	PVR Arm

Primary outcome

Number of Participants Randomized to PVR Via Catheter or Surgery
Time frame: 2-3 years
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.

6 secondary outcomes

Exercise Performances
Time frame: 12-18 months
Prevalence of Arrhythmias
Time frame: 12-18 months
Effects of Pulmonary Valve Replacement (PVR) on Diffuse Fibrosis
Time frame: 12-18 months
Effects of PVR on Exercise in the Magnetic Resonance (MR) Scanner
Time frame: 12-18 months
Effects of PVR on Biventricular Strain
Time frame: 12-18 months
Quality of Life (QOL)
Time frame: 12-18 months

Results and safety

Results posted to the registry on 6 Oct 2023, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
PVR Arm	1	0
No PVR	0	0

Primary outcome: Number of Participants Randomized to PVR Via Catheter or Surgery

Time frame: 2-3 years. Number. Unit: PVR via catheter or surgery

Measure	PVR Arm n = 1	No PVR n = 0
Value	0	–

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

6 secondary outcomes are reported in the full results.

Adverse events

Time frame: 1 year. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
PVR Arm	0 of 1 (0%)	0 of 1 (0%)	0 of 1
No PVR	0 of 0	0 of 0	0

Limitations and caveats (from the sponsor): Study terminated early.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 6 Oct 2023.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Individual participant dataSharing planned: Yes. The Principal Investigator (PI) is responsible for data management and accuracy of records. The PI may assign designated qualified individuals to collect the information. Only investigators' on this protocol and assigned research staff on this protocol will have access to the data. Data will be entered into a REDcap database by a member of the study team. All data and records generated during this study will be kept confidential in accordance with Institutional and HIPAA policies on subject privacy. The study team will not use such data and records for any purpose other than conducting the study. In order to keep protected health information from disclosure, each patient will be given a unique identification number. The key to this code will be kept in a locked file in one of the study investigator's offices. Any data that is transmitted to any Data Coordinating Center (DCC) will be de-identified by the enrolling site

Related trials for Tetralogy of Fallot

RecruitingPhase 4
Sodium-Glucose Cotransporter 2 Inhibitors for Repaired Tetralogy of Fallot Patients for Enhancement of Cardio-Pulmonary Status Trial
Congenital Heart Disease, Repaired Tetralogy of Fallot (rTOF)
RecruitingPhase 1
Beta-blocker Administration for Cardiomyocyte Division
Tetralogy of Fallot, Double Outlet Right Ventricle
Active, not recruiting
ALTERRA: SAPIEN 3 THV With the Alterra Adaptive Prestent
Pulmonary Disease, Transcatheter Pulmonary Valve Replacement (TPVR)
Recruiting
Investigation of Tetralogy of Fallot in Neonates
Tetralogy of Fallot (TOF), Pulmonary Stenosis
RecruitingObservational study
Volution of Pulmonary Artery Dimensions in Tetralogy of Fallot
Tetralogy of Fallot (TOF), Congenital Heart Defects

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Mark Fogel, MD principal investigator

The Childrens Hospital of Philadelphia

6 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A study testing PVR in people with Tetralogy of Fallot, run by Children's Hospital of Philadelphia at 6 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 6 Oct 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 6 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

Yes. Results are posted in the registry (first posted 6 Oct 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Participants Randomized to PVR Via Catheter or Surgery (time frame: 2-3 years). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03634072.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Oct 2023.