Trials & Studies

Clinical Trials and Prospective Studies

Trial Name: Oral Treprostinil in Pediatric Patients with Pulmonary Arterial Hypertension (TDE-PH-206)

This study will evaluate the safety, tolerability and pharmacokinetics of oral treprostinil following transition from intravenous remodulin or inhaled prostacyclin or as add-on to current PAH therapy in de novo prostacyclin pediatric patients (age 7-17 years) with pulmonary arterial hypertension.

Enrollment goal: (4-7 patients)

Contact: Mary Mullen, MD, PhD (, 617-355-7866

Trial Name:  Phase Ii Randomized, Placebo-Controlled, Double Blind Clinical Trial of Valsartan for Attenuating Disease Evolution in Early Sarcomeric Hcm (Vanish)

This is a multicenter, double-blind placebo controlled phase II randomized clinical trial to assess the safety and efficacy of valsartan in attenuating disease evolution in early sarcomeric hypertrophic cardiomyopathy (HCM).Enrollment goal: 15 – 20 at Boston Children’s Hospital, 150 in total.

Contact: Jane Messere, Research Nurse ( 857-218-3628

Trial Name: A Novel Non-invasive Approach to Rejection Surveillance in Pediatric Heart Transplant Recipients

We propose to develop an innovative, non-invasive approach based on blood biomarker and echocardiography for non-rejection screening rather than rejection in pediatric heart transplant recipients, using patients’ own baseline biomarker and echocardiographic data as internal control. This approach has the potential to reduce invasive biopsies and health care costs after heart transplantation.

Enrollment goal: We had enrolled about 120 patients with  100% enrollment rate (each approach patient/family agreed to participant in the study. Since summer 2013 we have been enrolling patients until we have the biopsy proven  9 rejection event collected echo-biomarker   data (as study was powered).
Contact: Steve Colan, MD ( CHBOSTON.ORG)  617-355-6429

Other Research Highlights

A sample of recently-published research by Heart Center faculty. Boston Children’s Heart Center authors are in bold

Baird CWMyers POBorisuk MPigula FAEmani SM. Ring-reinforced Sano conduit at Norwood stage I reduces proximal conduit obstruction. Ann Thorac Surg. 2015;99(1):171-9.

Banka P, Schaetzle B, Komarlu R, Emani S, Geva T, Powell AJ. Cardiovascular magnetic resonance parameters associated with early transplant-free survival in children with small left hearts following conversion from a univentricular to biventricular circulation. Journal of Cardiovascular Magnetic Resonance. 2014;16.

Blume ED, Balkin EM, Aiyagari R, Ziniel S, Beke DM, Thiagarajan R, Taylor L, Kulik T, Pituch K, Wolfe J. Parental perspectives on suffering and quality of life at end-of-life in children with advanced heart disease: an exploratory study*. Pediatric Critical Care Medicine. 2014;15(4):336-42.

Brown DW, Cohen KE, O'Brien P, Gauvreau K, Klitzner TS, Beekman RH, Kugler JD, Martin GR, Neish SR, Rosenthal GL, Lannon C, Jenkins KJ. Impact of prenatal diagnosis in survivors of initial palliation of single ventricle heart disease. Pediatr Cardiol. 2015;36(2):314-21.

Clair M, Fernandes SM, Khairy P, Graham DA, Krieger EV, Opotowsky AR, Singh MN, Colan SD, Meijboom EJ, Landzberg MJ. Aortic valve dysfunction and aortic dilation in adults with coarctation of the aorta. Congenital Heart Disease. 2014;9(3):235-43.

Correa R, Sherwin ED, Kovach J, Mah DY, Alexander ME, Cecchin F, Walsh EP, Triedman JK, Abrams DJ. Mechanism and ablation of arrhythmia following total cavopulmonary connection. Circulation Arrhythmia and Electrophysiology. 2015.

Curley MAQ, Wypij D, Watson RS, Grant MJC, Asaro LA, Cheifetz IM, Dodson BL, Franck LS, Gedeit RG, Angus DC, Matthay MA, Network. Protocolized sedation vs. usual care in pediatric patients mechanically ventilated for acute respiratory failure a randomized clinical trial. Jama-Journal of the American Medical Association. 2015;313(4):379-89.

Daly KP, Dearling JL, Seto T, Dunning P, Fahey F, Packard AB, Briscoe DM. Use of [18F]FDG Positron emission tomography to monitor the development of cardiac allograft rejection. Transplantation. 2015.

DeWitt ES, Triedman JK, Cecchin F, Mah DY, Abrams DJ, Walsh EP, Gauvreau K, Alexander ME. Time dependence of risks and benefits in pediatric primary prevention implantable cardioverter-defibrillator therapy. Circulation Arrhythmia and Electrophysiology. 2014;7(6):1057-U137.

Eastaugh LJ, Thiagarajan RR, Darst JR, McElhinney DB, Lock JE, Marshall AC. Percutaneous Left Atrial decompression in patients supported with extracorporeal membrane oxygenation for cardiac disease. Pediatric Critical Care Medicine. 2015;16(1):59-65.

Gaynor JW, Stopp C, Wypij D, Andropoulos DB, Atallah J, Atz AM, Beca J, Donofrio MT, Duncan K, Ghanayem NS, Goldberg CS, Hovels-Gurich H, Ichida F, Jacobs JP, Justo R, Latal B, Li JS, Mahle WT, McQuillen PS, Menon SC, Pemberton VL, Pike NA, Pizarro C, Shekerdemian LS, Synnes A, Williams I, Bellinger DC, Newburger JW. Neurodevelopmental outcomes after cardiac surgery in infancy. Pediatrics. 2015;135(5):816-25.

Gellis L, Banka P, Marshall A, Emani S, Porras D. Transcatheter balloon dilation for recurrent right ventricular outflow tract obstruction following valve-sparing repair of tetralogy of Fallot. Catheter Cardiovasc Interv. 2015.

Geng J, Picker J, Zheng ZJ, Zhang XQ, Wang J, Hisama F, Brown DW, Mullen MP, Harris D, Stoler J, Seman A, Miller DT, Fu QH, Roberts AE, Shen YP. Chromosome microarray testing for patients with congenital heart defects reveals novel disease causing loci and high diagnostic yield. Bmc Genomics. 2014;15.

Hallbergson A, Gauvreau K, Powell AJ, Geva T. Right ventricular remodeling after pulmonary valve replacement: early gains, late losses. Ann Thorac Surg. 2014.

Harvey RP, Graham RM, Pu WT. Introduction to the special issue on heart regeneration and rejuvenation. Stem Cell Research. 2014;13(3PB):521-2.

Lacro RV, Dietz HC, Sleeper LA, Yetman AT, Bradley TJ, Colan SD, Pearson GD, Selamet Tierney ES, Levine JC, Atz AM, Benson DW, Braverman AC, Chen S, De Backer J, Gelb BD, Grossfeld PD, Klein GL, Lai WW, Liou A, Loeys BL, Markham LW, Olson AK, Paridon SM, Pemberton VL, Pierpont ME, Pyeritz RE, Radojewski E, Roman MJ, Sharkey AM, Stylianou MP, Wechsler SB, Young LT, Mahony L. Atenolol versus losartan in children and young adults with Marfan's syndrome. N Engl J Med. 2014;371(22):2061-71.

McLellan MC, Gauvreau K, Connor JA. Validation of the Cardiac Children's Hospital Early Warning Score: an early warning scoring tool to prevent cardiopulmonary arrests in children with heart disease. Congenital Heart Disease. 2014;9(3):194-202.

Murray JM, Hellinger A, Dionne R, Brown L, Galvin R, Griggs S, Mittler K, Harney K, Manzi S, VanderPluym C, Baker A, O'Brien P, O'Connell C, Almond CS. Utility of a dedicated pediatric cardiac anticoagulation program: the Boston Children's Hospital experience. Pediatric Cardiol. 2015.

Quinonez LG, Breitbart R, Tworetsky W, Lock JE, Marshall AC, Emani SM. Stented bovine jugular vein graft (Melody valve) for surgical mitral valve replacement in infants and children. J Thorac Cardiovasc Surg. 2014;148(4):1443-9.

Singh TP, Blume ED, Alexander PM, Gauvreau K. Association of hemodynamic profiles with wait-list mortality in children listed for heart transplantation with idiopathic dilated cardiomyopathy. Am J Cardiol. 2015;115(2):243-8.

Wesley MC, Pereira LM, Scharp LA, Emani SM, McGowan FX, Jr., DiNardo JA. Pharmacokinetics of tranexamic acid in neonates, infants, and children undergoing cardiac surgery with cardiopulmonary bypass. Anesthesiology. 2015.

Wu FM, Jonas MM, Opotowsky AR, Harmon A, Raza R, Ukomadu C, Landzberg MJ, Singh MN, Valente AM, Egidy Assenza G, Perez-Atayde AR. Portal and centrilobular hepatic fibrosis in Fontan circulation and clinical outcomes. J Heart Lung Transplant. 2015.