Design and rationale of a clinical trial to increase cardiomyocyte division in infants with tetralogy of Fallot.
Samar R El Khoudary, Anthony Fabio, Jessie W Yester...
https://pubmed.ncbi.nlm.nih.gov/34265312Actively Recruiting
Phase 1
Age: 30Days - 60Days
All Genders
ID04713657
Mechanistic Clinical Trial of Beta-Blocker Administration For Reactivating Cardiomyocyte Division In Tetralogy of Fallot
Led by Weill Medical College of Cornell University · Updated on 2025-11-18
40
Participants Needed
3
Research Sites
N/A
Total Duration
On this page
Sponsors
W
Weill Medical College of Cornell University
Lead Sponsor
N
National Heart, Lung, and Blood Institute (NHLBI)
Collaborating Sponsor
AI-Summary
What this Trial Is About
Tetralogy of Fallot and double outlet right ventricle are congenital heart conditions that can lead to heart failure. Researchers are studying whether the medicine Propranolol can help increase the growth of new heart muscle cells in infants with these conditions. This study is a phase 1 randomized trial aiming to understand if Propranolol can reactivate heart muscle cell division and support normal heart growth in infants up to about 4 months old undergoing surgical repair. Participants will be randomly assigned to receive either Propranolol or a placebo starting at 1 month of age until 4 months or just before surgery. Propranolol will be given by parents in four daily doses based on the infant's weight. Additionally, patients will receive labeled N-thymidine by mouth for five days to help measure heart muscle cell growth. The study includes heart imaging using echocardiogram and cardiac MRI before and at surgery, as well as tissue and blood sample collection during surgery for detailed analysis. During the study, infants will have regular physical exams including vital signs and feeding and diaper diaries to monitor medication processing. Researchers will analyze heart tissue removed during surgery to measure new heart muscle cell growth using isotope mass spectrometry. The main outcome is cardiomyocyte division at surgery. Secondary outcomes include heart muscle size changes assessed by imaging and microscopy. Participants will be monitored from 1 month of age until surgery, with close follow-up and safety assessments during this time.
CONDITIONS
Brief Title
Beta-blocker Administration for Cardiomyocyte Division
Who Can Participate
Age: 30Days - 60Days
All Genders
Eligibility Criteria
You may qualify if you...
- Male and female infants younger than 60 days old with Tetralogy of Fallot with pulmonary stenosis or double outlet right ventricle diagnosed by echocardiogram
- Infants must weigh more than 2 kilograms at the time of consent
- Infants must be tolerating feeding by mouth
- DORV variant included
You will not qualify if you...
- Congenital atrio-ventricular block on EKG with PR interval greater than 120 milliseconds
- Use of medications that interact with propranolol
- Families unable to comply with study requirements or deemed unsuitable by investigators
- Gestational age less than 35 weeks
- Infants of diabetic mothers or with asthma or underlying respiratory disease
- Presence of metal implants in infants
AI-Screening
AI-Powered Screening
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2
3
Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Treatment
Duration - Approximately 3 months or until 24 hours before surgery
Participants receive either propranolol or placebo starting at 1 month of age until 4 months of age or 24 hours prior to surgical repair, whichever comes first. During treatment, participants will also take 15N-thymidine by mouth once daily for 5 consecutive days beginning one week after starting the study drug. Parents or caregivers administer medications and keep dosing diaries. Urine samples are collected from diapers during thymidine administration to monitor processing of the labeled thymidine.
Monthly visits until surgery
Surgery and Immediate Post-operative Care
Duration - Day of surgery
Participants undergo surgical repair of Tetralogy of Fallot. Before surgery, blood samples and heart tissue removed during surgery are collected for analysis.
1 visit (in-person, surgical procedure)
Trial Site Locations
Total: 3 locations
1
Weill Cornell Medicine
New York, New York, United States, 10065
Actively Recruiting
2
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
3
Upmc Children'S Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15224
Actively Recruiting
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Research Team
R
Rashida Blackwood, BS, MPH
How is the study designed?
Study Type
INTERVENTIONAL
Masking
TRIPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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