Title: Intravenous Remodulin (Treprostinil) as Add-on Therapy for the Treatment of Persistent Pulmonary Hypertension of the Newborn: A Randomized, Placebo-Controlled, Safety and Efficacy Study

Description: This study investigates whether the addition of IV Remodulin will reduce the rate of clinical worsening as compared to standard of care in PPHN subjects who do not show an adequate response to iNO. Additionally, the study will evaluate the safety and treatment effects of IV Remodulin in the neonatal population.

Enrollment goal: 1-4 subjects

Primary investigator: Mary Mullen MD, PhD

Contact: Alessandra Russo: Alessandra.Russo@cardio.chboston.org or 857-218-3847


Title: Investigation of Actigraphy, an Exercise Measurement Device, as a Novel, Well-Defined, Reliable, Feasible, Easy to Use and Non-Invasive Study Endpoint to Facilitate Pediatric Pulmonary Arterial Hypertension Trials and Drug Development

Description: This prospective, multi-center study investigates the utility of actigraphy as a clinical measure of physical activity in children with PAH. More specifically, the study will focus on describing the use of actigraphy in children and determining if correlations exist between actigraphy data and disease severity, progression, clinical worsening and survival in children with PAH.

Enrollment goal: 10-15 subjects

Primary investigator: Mary Mullen MD, PhD

Contact: Alessandra Russo: Alessandra.Russo@cardio.chboston.org or 857-218-3847


Title: TOMORROW: Pediatric Use of Macitentan to Delay Disease Progression in PAH Worldwide

Description: The purpose of this study is to evaluate macitentan in comparison to standard of care (SoC) with regard to delaying disease progression in children with PAH. In addition, this study aims to assess the safety, tolerability and pharmacokinetics of macitentan in children ages 2-17 with PAH.

Enrollment goal: 2-4 subjects

Primary investigator: Mary Mullen MD, PhD

Contact: Alessandra Russo: Alessandra.Russo@cardio.chboston.orgor 857-218-3847


Title: Open-Label, Individual Dose Titration Study to Evaluate Safety, Tolerability, and Pharmacokinetics of Riociguat in Children From 6 to Less Than 18 Years of Age With Pulmonary Arterial Hypertension (PAH)

Description: This open-label, individual dose titration study aims to evaluate the safety, tolerability and pharmacokinetics of oral riociguat treatment in children (ages 6-18) with PAH. The study will also explore the efficacy of the drug in this pediatric population.

Enrollment goal: 2-4 subjects

Primary investigator: Mary Mullen MD, PhD

Contact: Alessandra Russo: Alessandra.Russo@cardio.chboston.org or 857-218-3847


Title: CAscade SCreening for Awareness and DEtection of Familial Hypercholesterolemia (CASCADE) Registry

Description: The purpose of this project is to add a regional supplement to a registry collecting and maintaining de-identified clinical information about patients with familial hypercholesterolemia (FH). The information will be used to track trends in therapy, events and outcomes in individuals diagnosed with FH. This information will further provide knowledge and understanding of the disease, and improve the care, quality of life and survival of those with FH.

Enrollment goal: 5,000 diagnosed individuals nationwide

Primary investigator: Sarah de Ferranti, MD, MPH

Contact: Heather Harker Ryan, MS, RN: heather.harker@cardio.chboston.org or 617-355-0955


Title: Awareness of Women’s Health Issues in Adolescent and Younger Adult Women (FUTURE)

Description: A collaboration with Adolescent Medicine at Boston Children’s, this study aims to assess young women’s knowledge of cardiovascular disease and its risk factors.

Enrollment goal: 70 patients in Preventive Cardiology. 304 previously enrolled in Adolescent Medicine.

Primary investigator: Holly Gooding, MD, (Adolescent Medicine) and Sarah de Ferranti, MD, MPH

Contact: Courtney Brown (Adolescent Medicine): Courtney.Brown@childrens.harvard.edu; or Hannah Palfrey (Cardiology): Hannah.Palfrey@cardio.chboston.org


Title: Children with Homozygous Familial Hypercholesterolemia on Apheresis: An International Registry (CHAIN)

Description: A multi-center study out of Amsterdam Medical Center to gain insight on the current and possible future role of apheresis in the treatment of children with HoFH worldwide, as well as the current status of detection and follow up of these children.

Enrollment goal: 25-40 subjects

Primary investigator: Sarah de Ferranti, MD, MPH

Contact: Carolyn Dunbar-Masterson, BSN, RN: Carolyn.Dunbar-Masterson@cardio.chboston.org


Title: Dyslipidemia of Obesity Intervention in Teens (DO IT!)

Description: A Pediatric Heart Network trial aimed to determine if treatment of combined dyslipidemia of obesity (CDO) in adolescents with oral pitavastatin will improve vascular measures of early atherosclerosis with acceptable safety.

Enrollment goal: 50 subjects worldwide

Primary investigator: Sarah de Ferranti, MD, MPH

Contact: Austin Liou: Austin.Liou@cardio.chboston.org; or Heather Harker Ryan, MS, RN, PhD(c): heather.harker@cardio.chboston.org


Title: Immune Cell Metabolism with Elevated Low-Density Lipoprotein Cholesterol (ICE-LDL)

Description: This study will analyze blood samples collected from patients with familial hypercholesterolemia (FH) to examine how the cells in the immune system use energy in response to exposure to various levels of low-density lipoprotein cholesterol (LDL-C).

Enrollment goal: 15 subjects

Primary investigator: Michael Mendelson, MD, ScM, and Ivan Zanoni, PhD, (GI/Immunobiology)

Contact: Hannah Palfrey: Hannah.Palfrey@cardio.chboston.org


Title: Cerebral Microemboli During Pediatric Cardiac Catheterization: A Pilot Study Using Transcranial Doppler Sonography

Description: The primary aim of this study is to use transcranial Doppler sonography to evaluate the burden of cerebral microemboli during pediatric cardiac catheterization and the circumstances under which they occur. Recently, patients on mechanical circulatory support (ECMO or VAD) in the CICU have been added to the protocol. This study also investigates signal processing algorithms to improve the accuracy of automated emboli detection by TCD.

Enrollment goal: 40 subjects

Primary investigator: Barry Kussman, MBBCh, and Kerri LaRovere, MD

Contact: Rachel Bernier, MPH: rachel.bernier@childrens.harvard.edu or 857-218-5348


Title: GORE CARDIOFORM ASD Occluder Clinical Study: A Study to Evaluate Safety and Efficacy in the Treatment of Transcatheter Closure of Ostium Secundum Atrial Septal Defects (ASDs) – The Gore ASSURED Clinical Study

Description: The goal of this study is to evaluate the safety and efficacy of a new ASD occluder device as part of an FDA-approved IDE multicenter trial.

Enrollment goal: 20 subjects

Primary investigator: Lisa Bergersen, MD, MPH

Contact: Cardio Outcomes Clinical Research Staff: CardioOutcomes@childrens.harvard.edu or 617-355-8228


Title: Neurocognitive Function of Adolescents and Young Adults With Repaired Complex Congenital Heart Disease After Recent Cardiac Re-Interventions: A Pilot Study

Description: The goal of this study is to determine the impact of right ventricular outflow tract re-intervention on neurocognitive function for patients with repaired complex congenital heart disease who are scheduled for surgical or transcatheter pulmonary valve replacement.

Enrollment goal: 20 subjects

Primary investigator: Lisa Bergersen, MD, MPH

Contact: Cardio Outcomes Clinical Research Staff: CardioOutcomes@childrens.harvard.edu or 617-355-8228


Title: Stress Ulcer Prophylaxis versus Placebo – A Blinded Randomized Control Trial to Evaluate the Safety of Two Strategies in Critically Ill Infants With Congenital Heart Disease (SUPPRESS-CHD)

Description: A single-center, randomized, placebo-controlled pilot study examining whether withholding stress ulcer prophylaxis from critically ill infants with congenital heart disease in the CICU is feasible, safe (no increased rate of upper gastrointestinal bleeds), and results in a favorably diverse aerodigestive microbiome potentially decreasing the rate of hospital-acquired infections.

Enrollment goal: 100 infants (< 12 months)

Primary investigator: Kimberly I. Mills, MD; Ben D. Albert, MD; and Nilesh M. Mehta, MD

Contact: Kimberly I. Mills, MD: kimberly.mills@cardio.chboston.org or 716-949-4944


Title: Boston Circulatory Arrest Study (BCAS) – Adult: Antecedents and Correlates of Well-Being in Adults with Transposition of the Great Arteries

Description: This study evaluates long-term neuropsychological, mental health and well-being outcomes in adults with d-loop transposition of the great arteries who were enrolled as part of the initial BCAS cohort from 1988-1992. Study participants will complete full neuropsychological and mental health evaluations as well as a brain MRI.

Enrollment goal: 100 subjects (out of an original cohort of 171) and 50 controls

Primary investigator: Michelle Gurvitz, MD, MS

Contact: Donna Duva: donna.duva@cardio.chboston.org or 617-355-5422


Title: Phase III Multi-Center Open Label Randomized Clinical Trial Comparing Everolimus and Low Dose Tacrolimus to Tacrolimus and Mycophenolate Mofetil at 6 Months Post Heart Transplant to Prevent Long-Term Complications after Pediatric Heart Transplantation

Description: This study is a randomized trial with children who have undergone recent heart transplant, to determine whether a new rejection treatment (everolimus and low-dose tacrolimus) can reduce or prevent complications of transplant, including rejection, coronary artery disease and kidney disease, when compared to usual care (tacrolimus and mycophenolate mofetil).

Enrollment goal: 14 subjects

Primary investigator: Kevin P. Daly, MD

Contact: Matthew MacLean: Matthew.MacLean@cardio.chboston.org or 617-355-4213


Title: Parent-Physician Communication, Prognostic Awareness and Symptom Burden in Children with Advanced Heart Disease

Description: This study prospectively evaluates quality of life, parent understanding of prognosis and goals of care, and parent-caregiver communication related to the care of children with advanced heart disease. Parent, physician and bedside nurse enrollment will occur simultaneously.

Enrollment goal: 20-patient pilot, followed by 160 parent/physician/nurse triads

Primary investigator: Betsy Blume, MD

Contact: Rocky Reichman: Jeffrey.Reichman@cardio.chboston.org


Title: Evaluation of the Safety and Tolerability of Oral ENTRESTO for the Treatment of Pediatric Heart Failure Patients

Description: Multi-center, dose-escalation study to define the pharmacokinetics, pharmacodynamics, safety and tolerability of ENTRESTO (valsartan/sacubitril) in pediatric patients with heart failure. Patients less than 18 years of age with two ventricle heart disease and systolic heart failure (LVEF<40%) are eligible to participate.

Enrollment goal: 3 subjects

Primary investigator: Kevin P. Daly, MD

Contact: Kevin Daly, MD: kevin.daly@cardio.chboston.org; Jane Messere, BSN, RN: jane.messere@cardio.chboston.org or 857-719-6391


Title: A Multicenter, Randomized Controlled Trial Comparing Standard of Care Enalapril to ENTRESTO for the Treatment of Pediatric Heart Failure

Description: This is a 52-week randomized, double-blind, parallel group, active-controlled study to evaluate the efficacy and safety of ENTRESTO (valsartan/sacubitril) compared with enalapril in pediatric heart failure patients. Patients must be less than 18 years of age with two ventricle heart disease and systolic heart failure (LVEF < 40%) to be eligible to enroll.

Enrollment goal: 18 subjects

Primary investigator: Kevin P. Daly, MD

Contact: Kevin Daly, MD: kevin.daly@cardio.chboston.org; Jane Messere, BSN, RN: jane.messere@cardio.chboston.org or 857-719-6391


Title: A Prospective Adult Congenital Heart Disease Biorepository

Description: 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: Ongoing

Primary investigator: Alexander Opotowsky, MD, MPH

Contact: Catherine Gray, BS: Catherine.gray@cardio.chboston.org or 617-919-4458


Title: Prospective, Non‐Randomized, Single-Arm, Multi-Center Clinical Evaluation of the Edwards Pericardial Bioprosthesis (Model 11000A) for Pulmonary Valve Replacement

Description: The Edwards Pericardial Bioprosthesis, Model 11000A, is indicated for patients 5 years or older requiring replacement of their native or prosthetic pulmonary valve. The objective of this trial is to assess the safety and effectiveness of the Edwards Pericardial Bioprosthesis Model 11000A in the pulmonary position. This is a prospective, non-randomized, single-arm, multi-center study. Up to 100 pulmonary valve replacement (PVR) subjects at up to 10 clinical sites will be enrolled. Clinical data will be collected from patients enrolled for five years after the 100th patient is enrolled. Data will be collected from at least three centers with data available on a minimum of 15 patients who have completed the one-year follow-up visit.

Enrollment goal: 30-50 subjects per site

Primary investigator: Christopher W. Baird, MD

Contact: Michele J. Borisuk, MSN, CPNP: Michele.borisuk@cardio.chboston.org or 617-355-3515


Title: Prospective, Non-Randomized, Open-Label Clinical Study to Assess the Feasibility of the Bioabsorbable Pulmonary Valved Conduit in Subjects Undergoing Right Ventricular Outflow Tract (RVOT) Reconstruction

Description: The bioabsorbable pulmonary valved (PV) conduit is a gamma sterilized, bioabsorbable, polymer-based medical device. The valve itself is formed by three leaflets, which are incorporated within the conduit wall. This is a prospective, multi-center, non-randomized, single-arm, open label, clinical study. The bioabsorbable pulmonary valved conduit is used for the reconstruction of the right ventricular outflow tract (RVOT). There is no specific device-related surgical procedure necessary for the implantation. Pulmonary valve replacement is the only treatment proven to reduce right ventricular size and improve right ventricular function in the long term. The PV conduit is used for correction or reconstruction of the right ventricular outflow tract (RVOT) in patients less than 22 years with any of the following congenital heart malformations: tetralogy of Fallot, truncus arteriosus, pulmonary atresia, transposition of great arteries with ventricular septal defect, pulmonary stenosis in combination with other defects in congenital heart defect (CHD) syndromes. In addition, the PV conduit can be used for the replacement of previously implanted, but dysfunctional, pulmonary homografts or valved conduits. The PV conduit is surgically implanted using the same surgical technique as would be used for any standard homograft or off-the-shelf PV conduit. No special medication is required other than standard practice. The patient will be followed and data collected to evaluate the growth of the conduit at 6, 36, 48 and 60 months in pediatric population under 18 years.

Enrollment goal: 2-3 subjects per site. Total trial goal enrollment is 10.

Primary investigator: Christopher W. Baird, MD

Contact: Michele J. Borisuk, MSN, CPNP: Michele.borisuk@cardio.chboston.org or 617-355-3515