The Heart Institute of UPMC Children’s Hospital of Pittsburgh is recognized nationally and internationally for its innovative clinical and laboratory research initiatives. Our researchers and clinicians work side-by-side to study clinically relevant cardiovascular problems in search of innovative solutions that will improve the lives of patients.
Congenital heart disease (CHD) is one of the Heart Institute’s primary areas of research focus. The most common of all birth defects, CHD affects 0.8% of newborns – nearly 40,000 babies born in the United States each year. As a result of improved diagnoses and advanced surgical techniques, there are now 2.4 million people living with CHD in the U.S., and our research programs seek to improve their lives.
From Lab to Bedside
Our research studies, many of which are sponsored by the National Institutes of Health and industry partners, are focused on delivering the best patient outcomes for our patients, families, and communities. Our most successful research initiatives begin with basic science discoveries in the laboratory. Then, in partnership with our clinical researchers, we bring our innovation to the bedside.
An example can be found in our investigations of congenital cardiovascular malformations. These represent one of the leading causes of morbidity and mortality in children and adults, yet it remains unclear how many congenital cardiovascular malformations occur. Our research laboratories investigate the physiology and biomechanics of developing cardiovascular systems in animal models and stem cells to better understand the structural and functional maturation of the normal heart and blood vessels and the causes of CHD. This knowledge is then further explored through our numerous clinical studies focused on CHD.
Unique Collaborations
Through a unique collaboration, the Pediatric Institute for Heart Regeneration and Therapeutics, scientists and physicians from UPMC Children’s Hospital of Pittsburgh are pursuing groundbreaking investigations into how to make heart cells, called cardiomyocytes, replicate and proliferate to help the heart to heal itself in cases of heart failure or congenital defects.
Interactions between mother and fetus may also contribute to altered heart development. To better understand these interactions, we investigate maternal and embryonic/fetal cardiovascular function in animal models using high-resolution ultrasound and other advanced techniques.
In our research labs we also study cardiovascular structure and function in animal models as part of our evaluation of novel therapies using cells, advanced materials, medical devices, and software to help in the treatment of congenital and acquired heart disease.
Pediatric Cardiology Clinical Research Studies
UPMC has long been a leader in heart care, with a rich history in clinical research and innovation. Our clinicians, surgeons, and physicians conduct ongoing pediatric cardiology clinical research studies to:
- Explore and employ new technologies to improve treatments for heart diseases and congenital heart defects (CHDs).
- Delve into basic science questions to better understand pediatric heart disease and CHDs.
- Try to understand the genetics of heart disease and CHDs, which may help us develop therapies to prevent or limit the severity of these conditions.
GDMT Study
An observational study evaluating serum biomarkers, hemodynamic, and imaging criteria in CHD patients ages 13+. Study involves blood tests and neurocognitive testing. Participants are compensated $25 per blood draw.
PI: Anita Saraf, MD | Coordinator: Nik Gerhart gerhartne5@upmc.edu
Minima Stent PAS (Renata Medical)
An open-label post approval study to collect post-market, real-world safety and effectiveness data of the Minima Stent System when used to treat native and post-operative congenital vascular stenoses within the aortic and pulmonary circulations in neonates, infants, and small children.
PI: Lauren Carlozzi, MD | Coordinator: Nik Gerhart gerhartne5@upmc.edu
Uncertainty Aware Virtual Treatment Planning for PPS (Stanford)
This study is looking to characterize ex vivo mechanical properties and histological characteristics of health and diseased human PA tissue to support computer modeling and basic science studies. Enrolling patients with Alagille Syndrome and TOF and single PA focal stenosis undergoing cardiac cath with angioplasty/stent.
PI: Jaqueline Kreutzer, MD | Coordinator: Nik Gerhart gerhartne5@upmc.edu
OPTIMA (AHA)
This study is looking to form the basis for a non-invasive, genomic blood test that can be used to quantify net immune state of pediatric heart transplant recipients and ID patients at high risk for immunosuppression-related complications.
PI: Brian Feingold, MD | Coordinator: Kyla Thorn thornk2@upmc.edu
FUEL 2 (Mezzion)
A randomized controlled trial studying the safety and efficacy of udenafil to improve exercise capacity in adolescent participants with Fontan physiology. Study activities include two CPETs over a 26-week period, randomization, labs, and phone calls.
PI: Lauren Carlozzi, MD | Coordinator: Taylor Casteel casteeltl@upmc.edu
VALOR (Merck)
A randomized controlled trial to evaluate vericiguat in pediatric patients with heart failure due to left ventricular systolic dysfunction. Eligible participants will receive oral vericiguat or placebo. Study activities include knee x-ray, periodic safety labs, audiometry, and ECG. Study duration is one year.
PI: Brian Feingold, MD | Coordinator: Maggie Abraham abrahammc@upmc.edu
SCOUT-HCM (Bristol Myers Squib)
A randomized controlled trial to evaluate mavacamten in adolescents with obstructive HCM. Oral mavacamten or placebo will be administered to eligible participants. Study activities include echocardiograms every 2-4 weeks and monthly safety labs. Study duration is 79 weeks and includes a placebo-controlled period followed by an active treatment period.
PI: Brian Feingold, MD | Coordinator: Maggie Abraham abrahammc@upmc.edu
COMPASSION S3 – THV (Edwards)
A post approval study in patients undergoing a Sapien 3 THV implant with a dysfunctional RVOT or previously implanted surgical valve in the pulmonic position. Study activities include ECG, TTEs and fluoroscopy. Patients will be followed over a 5-year period.
PI: Jacqueline Kreutzer, MD | Coordinator: Nik Gerhart gerhartne5@upmc.edu
Neurocognitive Function Study
A prospective longitudinal muti-institutional pilot study of patients ages 13-30 with repaired congenital heart disease, who required cardiac surgery during infancy and are scheduled to undergo RVOT re-intervention, to determine the impact of re-intervention on neurocognitive function. Study activities include QoL surveys. Patients are followed for one year.
PI: Bryan Goldstein, MD | Coordinator: Nik Gerhart gerhartne5@upmc.edu
RCT for Jardiance
A randomized controlled trial studying the safety and clinical effectiveness of Jardiance therapy in high complexity ACHD patients in preclinical heart failure. Study activities include 1 year of study drug or placebo, imaging, labs, and neurocognitive testing.
PI: Anita Saraf, MD, PhD | Coordinator: Kyla Thorn thornk2@upmc.edu
ASCENT ASD (atHeart)
A clinical trial evaluating the safety and efficacy of the reSept ASD Occluder to treat patients with clinically significant secundum atrial septal defect. This trial involves the use of an investigational device intended for use in patients who require closure of the ASD and in whom a transcatheter approach is deemed appropriate. Study activities include ECGs and echocardiograms. Patients will be followed over a 5-year period.
PI: Bryan Goldstein, MD | Coordinator: Nik Gerhart gerhartne5@upmc.edu – Enrollment temporarily paused
Propranolol RCT (WCM)
A randomized controlled trial studying the effects of propranolol on cardiomyocyte regeneration in infants with tetralogy of Fallot. Participants will be randomized 1:1 to propranolol or placebo. Other research activities include cardiac imaging (MRI, echo), urine collection, and collection of myocardium on day of TOF repair surgery.
PI: Adam Christopher, MD | Coordinator: Shannon Janzef janzefsl@upmc.edu
Genetic Investigations in Atrial Arrhythmias
This study is investigating atrial fibrillation in patients younger than 35 years of age. Study activities involve blood and saliva collection.
PI: Gaurav Arora, MD | Coordinator: Taylor Casteel casteeltl@upmc.edu
