Clinical Trials and Prospective Studies

Trial name: Fontan Imaging Biomarkers (FIB) Study
Description: This is a cross-sectional study analyzing the non-invasive measures of ventricular systolic function, diastolic function, and serum/urine biomarkers in patients after the Fontan operation.
Enrollment goal: 100
Contacts: Sunil Ghelani, MD, and Rahul Rathod, MD, rahul.rathod@cardio.chboston.orgTrial name: A Phase I Study of Dexmedetomidine Bolus and Infusion in Corrective Infant Cardiac Surgery: Safety and Pharmacokinetics
Description: To determine the safety of a DEX loading dose/infusion regimen as part of a balanced general anesthetic/sedative strategy for neonates/infants undergoing corrective congenital cardiac surgery using cardiopulmonary bypass.
Enrollment goal: total of 106 patients from 4 centers
Contact: Juan C. Ibla, MD,, 617-355-6225Trial name: PCGC (Pediatric Cardiac Genetic Consortium): The Genomic Basis of Congenital Heart Disease and Neurodevelopmental Outcomes
Description: Innovative genomic techniques will be used to uncover new genetic causes of congenital heart disease. The project will also explore how genetic mutations and variants affect child development and the central nervous system.
Enrollment goal: 2000 (consortium-wide)
Contact: Judith Geva, Research Coordinator,, 617-355-4979Trial name: Validation of the Residual Lesion Score for Congenital Heart Surgery. (A pediatric Heart Network Study)
Description: This multicenter prospective study will validate the RLS as a tool to predict early and mid-term outcomes for five common congenital cardiac operations. The RLS study will develop the first validated tool for measurement of residual lesions following congenital heart surgery.
Enrollment goal: total of 1250 subjects across 17 centers (250 subjects each in the following procedural categories: CAVSD repair, TOF/PS repair, ASO, Norwood, and Arch VSD repair)
Contacts: Meena Nathan, MD,, 617-355-4308 and Jane Newburger, MD, MPH,, 617-355-5427

Title: A Multicenter, Open-Label, 24-Week, Uncontrolled Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Oral Treprostinil Extended Release Tablets Following Transition from Remodulin or Inhaled Prostacyclin Therapy or as Add-on to Current PAH Therapy in De Novo Prostacyclin Pediatric Subjects Aged 7 to 17 Years with Pulmonary Arterial Hypertension
Description: This study offers oral treprostinil as an add-on therapy for pediatric PAH patients and will assess the safety, tolerability and pharmacokinetics of this drug. Eligible patients may be transitioned from IV Remodulin or inhaled prostacyclin or they may be de novo prostacyclin patients.
Enrollment goal: 40 nationwide, 4-8 at Boston Children’s
Contact: Mary Mullen, MD, PhD,, 617-355-7866

Title: Surgical Planning with 3D Printed Cardiac Models in Patients with Malformations that Require Biventricular Repair
Description: The primary aim of this prospective, randomized trial is to assess the correlation between pre-operative surgical planning via inspection of a 3D printed cardiac model with reduced OR time.
Enrollment goal: 30
Contacts: Bre Piekarski, RN, BSN,, 617-919-4457 and PI: Sitaram Emani, MD,, 617-355-7932

Title: Evaluation of Coagulation Testing in Patients Undergoing Cardiac Surgery
Description: The aim of this prospective study is to use discarded blood from routine laboratory testing in neonates to assess the efficacy of new technology for coagulation assessment that is faster and uses less blood than current available testing.
Enrollment goal: 100
Contacts: Bre Piekarski, RN, BSN,, 617-919-4457 and PI: Sitaram Emani, MD,, 617-355-7932

Title: Determining the Natural and “Unnatural” History of Anomalous Aortic Origin of a Coronary Artery with an Interarterial Course (AAOCA): Establishing a Multi-Institutional Registry
Description: The purpose of the study is to determine the outcome of surgical intervention versus observation in children and young adults with AAOCA. To do this we will create a risk stratification model utilizing a large multi-institutional registry with the Congenital Heart Surgeon Society (CHSS).
Enrollment goal: 200
Contacts: Bre Piekarski, RN, BSN,, 617-919-4457 and PI: Meena Nathan, MD,, 617-355-4308

Title:  COMMENCE TRIAL Prospective, Non‐randomized, Single Arm, Multicenter Clinical Evaluation of the Edwards Pericardial Bioprosthesis (Model 11000A) for Pulmonary Valve Replacement
Description: The COMMENCE pulmonary trial is to assess the safety and effectiveness of the Edwards Pericardial Bioprosthesis valve in the pulmonary position in patients 5 years or older requiring replacement of their native or prosthetic pulmonary valve.  Clinical data will be collected for 5 years after enrollment is completed.
Enrollment goal: 100 nationwide, 30-40 from Boston Children’s
Contact:  Christopher W. Baird, MD.  (, 617-355-1914)

Title:  HALO Clinical Study: A single arm, prospective, non-randomized, multi-center clinical investigation of the St. Jude’s Medical Masters HP 15mm Rotatable Mechanical Heart Valve (MHV)
Description: Currently, commercially available mechanical mitral valves range in sizes 16 mm to 37 mm. A portion of the pediatric population would benefit from the availability of a smaller valve.  This study plans to evaluate the safety and durability of 15 mm St. Jude Masters Heart Valve intended for mitral valve replacement in patients less than five years of age.
Enrollment goal:  20 nationwide
Contact:  Christopher W. Baird, MD.  (, 617-355-1914)

Title:  A prospective single center study of Aortic valve reconstruction using the Ozaki technique
Description: The Ozaki procedure is a technique in which aortic valve reconstruction replaces three aortic valve cusps by using gluteraldehyde treated autologous pericardium and can be applied to a wide spectrum of aortic valve disease. This is considered reconstruction rather than replacement as there are no foreign materials and limited need (ASA) for postoperative anticoagulation.
Enrollment goal:  50
Contact:  Christopher W. Baird, MD.  (, 617-355-1914)

Other Research Highlights

Banka P, Robinson JD, Uppu SC, Harris MA, Hasbani K, Lai WW, Richmond ME, Fratz S, Jain S, Johnson TR, Maskatia SA, Lu JC, Samyn MM, Patton D, Powell AJ. Cardiovascular magnetic resonance techniques and findings in children with myocarditis: a multicenter retrospective study. J Cardiovasc Magn Reson. 2015;17(1):96. PMID: 26576638

Beausejour Ladouceur V, Lawler PR, Gurvitz M, Pilote L, Eisenberg MJ, Ionescu-Ittu R, Guo L, Marelli AJ. Exposure to Low-Dose Ionizing Radiation From Cardiac Procedures in Patients With Congenital Heart Disease: 15-Year Data From a Population-Based Longitudinal Cohort. Circulation. 2015. PMID: 26538581

Ding JChen JWang YKataoka MMa LZhou PHu XLin ZNie M, Deng ZL, Pu WT, Wang DZ. Trbp regulates heart function through microRNA-mediated Sox6 repression. Nat Genet. 2015.

Dusenbery SM, Lunze FI, Jerosch-Herold M, Geva T, Newburger JW, Colan SD, Powell AJ. Left Ventricular Strain and Myocardial Fibrosis in Congenital Aortic Stenosis. Am J Cardiol. 2015.

Esch JJSalvin JMThiagarajan RRdel Nid PJRajagopal SK. Acute kidney injury after Fontan completion: Risk factors and outcomes. J Thorac Cardiovasc Surg. 2015;150(1):190-7.

Farias MFriedman KGLock JENewburger JWRathod RH. Differentiating Standardized Clinical Assessment and Management Plans From Clinical Practice Guidelines. Acad Med. 2015;90(8):1002.

Freud LR, Escobar-Diaz MC, Kalish BT, Komarlu R, Puchalski MD, Jaeggi ET, Szwast AL, Freire G, Levasseur SM, Kavanaugh-McHugh A, Michelfelder EC, Moon-Grady AJ, Donofrio MT, Howley LW, Tierney ESS, Cuneo BF, Morris SA, Pruetz JD, van der Velde ME, Kovalchin JP, Ikemba CM, Vernon MM, Samai C, Satou GM, Gotteiner NL, Phoon CK, Silverman NH, McElhinney DB, Tworetzky W. Outcomes and Predictors of Perinatal Mortality in Fetuses With Ebstein Anomaly or Tricuspid Valve Dysplasia in the Current Era A Multicenter Study. Circulation. 2015;132(6):481-9.

Ghelani SJHarrild DMGauvreau KGeva TRathod RH. Comparison Between Echocardiography and Cardiac Magnetic Resonance Imaging in Predicting Transplant-Free Survival After the Fontan Operation. Am J Cardiol. 2015.

Hofferberth SC, Alexander ME, Mah DY, Bautista-Hernandez VDel Nido PJFynn-Thompson F. Impact of pacing on systemic ventricular function in L-transposition of the great arteries. J Thorac Cardiovasc Surg. 2015. Epub 2015/09/28. PMID: 26410005

Lafranchi T, Lincoln P. Prenatal Counseling and Care for Single-Ventricle Heart Disease: One Center’s Model for Care. Crit Care Nurse. 2015;35(5):53-61. PMID: 26427975

Lin CH, Desai S, Nicolas R, Gauvreau K, Foerster S, Sharma A, Armsby L, Marshall ACOdegard K, DiNardo J, Vincent J, El-Said H, Spaeth J, Goldstein B, Holzer R, Kreutzer J, Balzer D, Bergersen L. Sedation and Anesthesia in Pediatric and Congenital Cardiac Catheterization: A Prospective Multicenter Experience. Pediatr Cardiol. 2015;36(7):1363-75.

Myers PO, Baird CW, Del Nido PJ, Pigula FA, Lang N, Marx GR, Emani SM. Neonatal Mitral Valve Repair in Biventricular Repair, Single Ventricle Palliation, and Secondary Left Ventricular Recruitment: Indications, Techniques, and Mid-Term Outcomes. Frontiers in surgery. 2015;2:59. PMID: 26618162

Pickard SSGeva AGauvreau KDel Nido PJGeva T. Long-term outcomes and risk factors for aortic regurgitation after discrete subvalvular aortic stenosis resection in children. Heart. 2015.

Porras D, Gurvitz M, Marshall AC, Emani SM. Hybrid Approach for Off-Pump Pulmonary Valve Replacement in Patients With a Dilated Right Ventricular Outflow Tract. Ann Thorac Surg. 2015;100(5):e99-e101. PMID: 26522579

Prakash A, Adlakha H, Rabideau N, Hass CJ, Morris SA, Geva TGauvreau K, Singh MN, Lacro RV. Segmental Aortic Stiffness in Children and Young Adults with Connective Tissue Disorders: Relationships with Age, Aortic Size, Rate of Dilation and Surgical Root Replacement. Circulation. 2015.

Quinonez LGGauvreau KBorisuk MIreland CMarshall AMMayer JEJenkins KJFynn-Thompson FEBaird CW. Outcomes of surgery for young children with multivessel pulmonary vein stenosis. J Thorac Cardiovasc Surg. 2015.

Stern KWD, White MT, Verghese GR, del Nido PJ, Geva T. Intraoperative Echocardiography for Congenital Aortic Valve Repair: Predictors of Early Reoperation. Ann Thorac Surg. 2015;100(2):678-85.