Mechanistic Clinical Trial of Beta-Blocker Administration For Reactivating Cardiomyocyte Division In Tetralogy of Fallot

Who is this study for? Pediatric patients up to 2 months old with congenital heart disease
What treatments are being studied? Propranolol Hydrochloride
Status: Recruiting
Location: See all (3) locations...
Intervention Type: Other, Procedure, Drug
Study Type: Interventional
Study Phase: Phase 1
SUMMARY

Heart failure is a common long-term complication in patients with congenital heart disease (CHD). Medical treatments to promote regeneration of new healthy heart muscle cells have the potential to provide new heart failure treatments for these patients. The development of such therapies is limited by the poor understanding of the ways in which heart muscles grow after birth. Investigators have learned that humans without heart disease generate new heart muscles cells up to the age of 20 years old and that this is decreased in patients with congenital heart disease like Tetralogy of Fallot. Investigators are trying to determine if treatment with a medicine called Propranolol can increase heart muscle cell proliferation and, with that, normalize heart growth. Investigators will examine discarded heart muscle tissue that is obtained during surgery for the presence of new heart muscle cells. Propranolol is approved by the Food and Drug Administration (FDA) to treat a certain kind of benign tumor in infants (hemangioma), but it is not currently approved by the FDA to increase heart muscle growth.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 1 month
Maximum Age: 2 months
Healthy Volunteers: f
View:

• Male and female infants \< 60 days of age with a diagnosis of tetralogy of Fallot (ToF) with pulmonary stenosis (PS) or double outlet right ventricle (DORV), tetralogy type by echocardiogram, who weigh greater than 2 kg at the time of consent and are tolerating enteral feeds.

• DORV variant

Locations
United States
New York
Weill Cornell Medicine
RECRUITING
New York
Pennsylvania
Children's Hospital of Philadelphia
NOT_YET_RECRUITING
Philadelphia
Upmc Children'S Hospital of Pittsburgh
RECRUITING
Pittsburgh
Contact Information
Primary
Rashida Blackwood, BS, MPH
rab4029@med.cornell.edu
646-962-9036
Time Frame
Start Date: 2022-12-16
Estimated Completion Date: 2030-12-31
Participants
Target number of participants: 40
Treatments
Active_comparator: Propranolol
The target dose of Propranolol hydrochloride for this study target dose for this study will be 4 mg/kg/day divided in 4 doses. The concentration of propranolol solution is 20 mg/5 mL. Additionally, labeled syringes will be provided to families for accurate weight-based dosing. Treatment with propranolol will begin at 1 month of age and continue until 4 months of age or 24hrs prior to surgical repair, whichever comes first.
Placebo_comparator: Placebo
Placebo will be given in a volume that corresponds to the patient's weight. Additionally, labeled syringes will be provided to families for accurate weight-based dosing. Treatment with placebo will begin at 1 month of age and continue until 4 months of age or 24hrs prior to surgical repair, whichever comes first. There is no current standard of care for pharmacologic therapy for infants with Tetralogy of Fallot (ToF). As such, there are no alternative treatments, and the placebo group is standard of care.
Authors
Anthony Fabio, Jessie Yester
Sponsors
Leads: Weill Medical College of Cornell University
Collaborators: National Heart, Lung, and Blood Institute (NHLBI)

This content was sourced from clinicaltrials.gov

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