Clinical Trials and Prospective Studies

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The Heart Center continues its legacy of leading the field in research, and is currently enrolling patients for a wide variety of studies. Topics range from neurodevelopmental outcomes in patients with hypoplastic left heart syndrome (HLHS) to surgical planning with 3-D printed models.

Below is a sample list of our currently open studies:

Fontan Imaging Biomarkers (FIB) Study

This is a prospective study looking at the correlations of CMR, echocardiographic, blood, and urine biomarkers parameters of systolic and diastolic function in Fontan patients.  Patients will be approached for enrollment when scheduled for clinically indicated CMRs.

Enrollment goal: 100

Contact: Sunil Ghelani, MD, Sunil.Ghelani@cardio.chboston.org, and Rahul Rathod, MD, Rahul.rathod@cardio.chboston.org


A Prospective Adult Congenital Heart Disease Biorepository

This study involves prospectively collecting and storing blood and urine specimens for adults who have congenital heart disease. The ultimate aim is to facilitate research into underlying mechanisms of deterioration and development of diagnostic tools to better predict the incidence of specific adverse outcomes.

Enrollment goal: 1,200

Contact: Prince Owusu, MPH, prince.owusu@cardio.chboston.org, 617-355-4347 or

Alexander Opotowsky, MD, Alexander.Opotowsky@cardio.chboston.org, 617-355-6508


Preserved Umbilical Vein Use as Autologous Shunt Conduit in Neonatal Cardiac Surgery

This is a pilot study to evaluate the safety of the use of autologous umbilical vein as shunts or conduits in neonatal cardiac surgery.

Enrollment goal: 6 subjects

Contact: Bre Piekarski, RN, BSN, Breanna.Piekarski@cardio.chboston.org, 617-919-4457 (PI: Sitaram Emani, MD)
 


Determining the Natural and “Unnatural” History of Anomalous Aortic Origin of a Coronary Artery with an Interarterial Course (AAOCA): Establishing a Multi-Institutional Registry

This multi-institution prospective data registry will help determine the outcome of surgical intervention versus observation in children and young adults with AAOCA.

Enrollment goal: 200 subjects

Contact: Bre Piekarski, RN, BSN, Breanna.Piekarski@cardio.chboston.org, 617-919-4457 (PI: Meena Nathan, MD)


Evaluation of Coagulation Testing in Patients Undergoing Cardiac Surgery

This study, conducted in conjunction with Cardiac Anesthesia, uses discarded blood samples to evaluate the input of newer technologies to diagnose coagulopathies in children undergoing cardiac surgery.

Enrollment goal: 200 subjects

Contact: Bre Piekarski, RN, BSN, Breanna.Piekarski@cardio.chboston.org, 617-919-4457 (PI: Juan Ibla, MD)


Surgical Planning with 3D Printed Cardiac Models in Patients with Malformations that Require Biventricular Repair

This study will analyze the efficacy of using 3D printed cardiac models for pre-operative surgical planning in complex pediatric cardiac surgery patients.

Enrollment goal: 30 subjects

Contact: Bre Piekarski, RN, BSN, Breanna.Piekarski@cardio.chboston.org, 617-919-4457 (PI: Sitaram Emani, MD)


Cardiac Biomarkers and Echocardiographic Modalities for Rejection Surveillance after Pediatric Heart Transplantation

This study investigates non-invasive methods of monitoring pediatric heart transplant recipients for graft rejection. The primary goal is to determine if biomarker blood levels in conjunction with echocardiographic measurements can reliably rule out rejection in children after heart transplantation.

Enrollment goal: 160

Contact: Fatima Lunze, MD, PhD, Fatima.Lunze@cardio.chboston.org, 857-218-5434


Living with Congenital Aortic Stenosis: Exercise Restriction, Patterns of Adherence, and Quality of Life

This is a prospective study of patients with congenital aortic stenosis and their cardiologists using brief survey instruments including validated quality of life assessment.

Enrollment goal: 100 patient and physician pairs, including participating New England Congenital Cardiology Association (NECCA) sites.

Contact: Drs. Laura Mansfield, MD, laura.mansfield@cardio.chboston.org and David Brown, MD, david.brown@cardio.chboston.org


Normative values of cardiac MRI-derived values of aortic stiffness in Healthy Children and Young Adults 

This is a prospective trial to collect data on the aortic stiffness of healthy volunteers. Aortic stiffness is a novel parameter of vascular health and has been shown to be abnormal in patients with connective tissue disease as well as correlate with aortic dilation and aortic root replacement in those patients. We will begin by collecting normative values prospectively through the MRI lab. These measurements can be collected during a standard cardiac MRI and we plan to perform these short scans on healthy volunteers and will provide compensation for their time.

Enrollment goal: 30

Contact: Ashwin Prakash, MD, ashwin.prakash@cardio.chboston.org, 617-355-7366


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

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 Hospital

Contact: Mary Mullen, MD, PhD, mary.mullen@cardio.chboston.org, 617-355-7866


Tracking Outcomes and Practice in Pediatric Pulmonary Hypertension

This is a registry study that collects data from pediatric patients with newly diagnosed PH with the goals of comparing treatment strategies and goals, characterizing the clinical course of pediatric PH patients, and evaluating the outcomes in this patient population.

Enrollment goal: 200 nationwide, 5-10 at Boston Children's Hospital

Contact: Mary Mullen, MD, PhD, mary.mullen@cardio.chboston.org, 617-355-7866


Long-term Outcomes of Children with Hypoplastic Left Heart Syndrome (HLHS) and the Impact of Norwood Shunt Type Study (SVR III) and Brain Connectome and Neurodevelopmental Outcomes Study

The purpose of this study to determine which type of shunt placed at the time of the Norwood operation is most effective by school age. Specifically long-term differences in heart function, exercise tolerance, neurodevelopment or quality of life will be explored.  Participants will undergo cardiac MRI, echocardiogram, exercise testing, neurodevelopmental testing as well as brain MRI if also participating in Brain Connectome Study.

Enrollment goal: 35 at Boston Children's Hospital

Contact: Jane W. Newburger, MD, MPH (Jane.newburger@cardio.chboston.org) or Carolyn Dunbar-Masterson, RN (Carolyn.dunbar-masterson@cardio.chboston.org or 617-355-4201)


Pilot Study of Executive Function Intervention to Improve Neurodevelopmental Outcomes in Adolescents with CHD

This is a single center, single blinded 2-arm randomized controlled trial to test the immediate post-treatment and 3-month follow-up efficacy of Cogmed Working Memory intervention versus standard of care in 13-16 year-old adolescents with CHD. Study procedures include neurodevelopmental testing, parental questionnaires as well as medical and sociodemographic variables.

Enrollment goal: 60

Contact: Jane W. Newburger, MD, MPH (Jane.newburger@cardio.chboston.org), Johanna Calderon, PhD (Johanna.calderon@childrens.harvard.edu) or Carolyn Dunbar-Masterson, RN (Carolyn.dunbar-masterson@cardio.chboston.org or 617-355-4201)


Improving Neurodevelopmental Outcomes in Children with Congenital Heart Disease: An Intervention Study 

This is a single center, single blinded 2-arm randomized controlled trial to test the immediate post-treatment and 3-month follow-up efficacy of Cogmed Working Memory intervention versus standard of care in 7-12 year-old children with CHD. Study procedures include neurodevelopmental testing, parental questionnaires as well as medical and sociodemographic variables.

Enrollment goal: 100

Contact: Jane W. Newburger, MD, MPH (Jane.newburger@cardio.chboston.org), Johanna Calderon, PhD (Johanna.calderon@childrens.harvard.edu) or Carolyn Dunbar-Masterson, RN (Carolyn.dunbar-masterson@cardio.chboston.org or 617-355-4201)


Validation of the Residual Lesion Score (RLS) for Congenital Heart Surgery

The purpose of the study is to learn if a scoring system, called the Residual Lesion Score, can predict how children do after cardiac surgery. Participants will be less than one of year of age and will have undergone one of the following cardiac surgeries: ASO, CAVC repair, TOF repair, aortic arch with VSD repair and stage 1 Norwood.

Enrollment goal: 250

Contact: Meena Nathan, MD, MPH (meena.nathan@cardio.chboston.org) or Carolyn Dunbar-Masterson, RN (Carolyn.dunbar-masterson@cardio.chboston.org or 617-355-4201)


A Phase I Study of Dexmedetomidine Bolus and Infusion in Corrective Infant Cardiac Surgery: Safety and Pharmacokinetics.

The purpose of the study is determine safe doses of dexmedetomidine for infants undergoing corrective heart surgery. Eligible subjects will undergo one of the following one surgeries: VSD repair, TOF repair or ASO. Participants will receive open-label dexmedetomidine while in the operating room and up to twelve hours after surgery in the CICU.

Enrollment goal: 20

Contact: Juan Ibla, MD (Juan.ibla@childrens.harvard.edu) or Carolyn Dunbar-Masterson, RN (Carolyn.dunbar-masterson@cardio.chboston.org or 617-355-4201)


Fontan Udenafil Exercise Longitudinal Assessment Trial (FUEL)

The purpose of this study is to find out if udenafil can improve or reduce decline in exercise ability of Fontan patients ages 12 to 19 years. Participants will be randomized to take udenafil or placebo twice daily for 6 months.  Study procedures include vascular function tests, echocardiograms, exercise testing, safety labs and questionnaires.

Enrollment goal: 50

Contact: Jonathan Rhodes, MD (Jonathan.rhodes@childrens.harvard.edu) or Carolyn Dunbar-Masterson, RN (Carolyn.dunbar-masterson@cardio.chboston.org or 617-355-4201)

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- Sandra L. Fenwick, President and CEO

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