Pediatric REPlAcement of the PulmonaRy ValvE in Tetralogy of Fallot - (TOFandPVR)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03634072|
Recruitment Status : Active, not recruiting
First Posted : August 16, 2018
Last Update Posted : January 6, 2021
|Condition or disease||Intervention/treatment||Phase|
|Tetralogy of Fallot||Procedure: PVR||Not Applicable|
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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||80 participants|
|Intervention Model:||Parallel Assignment|
|Intervention Model Description:||Subjects will be randomized to PVR or no PVR|
|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.|
|Official Title:||Pediatric REPlAcement of the PulmonaRy ValvE in Tetralogy of Fallot - The PREPARE-TOF Study|
|Actual Study Start Date :||July 6, 2018|
|Estimated Primary Completion Date :||August 1, 2021|
|Estimated Study Completion Date :||August 1, 2021|
PVR arm will undergo PVR via catheter 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
No Intervention: No PVR
No PVR group will continue with medical management
- Operational feasibility of a randomized multicenter trial [ Time Frame: 2-3 years ]The operational feasibility will be assessed by the ability to recruit the required number of patients in the time given (recruiting the full complement of 90 patients over 18 months across 5 centers (ie 1 patient per month per center) to achieve 500 patients across 8 centers over the course of 2-3 years in the larger definitive trial
- Exercise performances [ Time Frame: 12-18 months ]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
- Prevalence of arrhythmias [ Time Frame: 12-18 months ]This will be determined by reviewing Holter monitoring data the assess the prevalence of arrhythmias. This will also provide the endpoints for the larger, longer term trial
- Effects of PVR on Diffuse Fibrosis [ Time Frame: 12-18 months ]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).
- Effects of PVR on exercise in the Magnetic Resonance (MR) scanner [ Time Frame: 12-18 months ]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 CMR.
- Effects of PVR on Biventricular Strain [ Time Frame: 12-18 months ]Mechanisms of the effects of PVR in the definitive trial will be measured by obtaining preliminary data on biventricular strain.
- Quality of Life (QOL) [ Time Frame: 12-18 months ]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 (Marino BS et al. Pediatrics. 2010;126:498-508). 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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03634072
|United States, District of Columbia|
|Children's National Medical Center|
|Washington, District of Columbia, United States, 20010|
|United States, Georgia|
|Children's Healthcare of Atlanta|
|Atlanta, Georgia, United States, 30322|
|United States, Illinois|
|Lurie Children's Hospital of Chicago|
|Chicago, Illinois, United States, 60611|
|Chicago, Illinois, United States, 60611|
|United States, Ohio|
|Cincinnati Children's Hosptial Medical Center|
|Cincinnati, Ohio, United States, 45229|
|United States, Pennsylvania|
|The Childrens Hospital of Philadelphia|
|Philadelphia, Pennsylvania, United States, 19104|
|Principal Investigator:||Mark Fogel, MD||The Childrens Hospital of Philadelphia|