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 PSchaetzle BKomarlu REmani SGeva TPowell 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 SBeke DMThiagarajan 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 PGauvreau 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 SMKhairy 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 RSherwin EDKovach JMah DYAlexander MECecchin FWalsh EPTriedman JKAbrams DJ. Mechanism and ablation of arrhythmia following total cavopulmonary connection. Circulation Arrhythmia and Electrophysiology. 2015.

Curley MAQWypij 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 KPDearling JLSeto TDunning PFahey FPackard ABBriscoe DM. Use of [18F]FDG Positron emission tomography to monitor the development of cardiac allograft rejection. Transplantation. 2015.

DeWitt ESTriedman JKCecchin FMah DYAbrams DJWalsh EPGauvreau KAlexander 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 LJThiagarajan RRDarst JRMcElhinney DBLock JEMarshall 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 DCNewburger JW. Neurodevelopmental outcomes after cardiac surgery in infancy. Pediatrics. 2015;135(5):816-25.

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

Geng JPicker JZheng ZJZhang XQWang JHisama FBrown DWMullen MPHarris DStoler JSeman AMiller DTFu QHRoberts AEShen YP. Chromosome microarray testing for patients with congenital heart defects reveals novel disease causing loci and high diagnostic yield. Bmc Genomics. 2014;15.

Hallbergson AGauvreau KPowell AJGeva 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 ESLevine 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 MCGauvreau KConnor 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 JMHellinger ADionne RBrown LGalvin RGriggs SMittler KHarney KManzi SVanderPluym CBaker AO’Brien PO’Connell CAlmond CS. Utility of a dedicated pediatric cardiac anticoagulation program: the Boston Children’s Hospital experience. Pediatric Cardiol. 2015.

Quinonez LGBreitbart RTworetsky WLock JEMarshall ACEmani 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 TPBlume EDAlexander PMGauvreau 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 MCPereira LMScharp LAEmani SM, McGowan FX, Jr., DiNardo JA. Pharmacokinetics of tranexamic acid in neonates, infants, and children undergoing cardiac surgery with cardiopulmonary bypass. Anesthesiology. 2015.

Wu FMJonas MMOpotowsky AR, Harmon A, Raza R, Ukomadu C, Landzberg MJSingh MNValente AMEgidy Assenza GPerez-Atayde AR. Portal and centrilobular hepatic fibrosis in Fontan circulation and clinical outcomes. J Heart Lung Transplant. 2015.