Trials & Studies

Clinical Trials and Prospective Studies

A rundown of the latest clinical trials and prospective studies now enrolling patients at the Heart Center.


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

Primary investigator: Mary Mullen MD, PhD

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


Title: Levosimendan Expanded Access Compassionate Use in Pediatric Patients with Advanced Decompensated Heart Failure who are Refractory to Standard Therapy

Description: To obtain clinical access to levosimendan, a calcium-sensitizing inodilator, for pediatric patients under FDA Expanded Access to infants and children in severe decompensated heart failure who are not responding to standard care.

Enrollment goal: Not more than 80 patients

Primary investigator: Peta Alexander, MBBS

Contact: Peta Alexander: Peta.alexander@cardio.chboston.org or 617-355-7866


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 patients

Primary investigator: Lisa Bergersen, MD MPH

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


Title: New Enrollment SAPIEN XT Post-Approval Study (COMPASSION XT PAS)

Description: The goal of this study is to evaluate the safety of the FDA approved SAPIEN XT THV in the pulmonic position.

Enrollment goal: 28 patients

Primary investigator: Diego Porras, MD

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 surgical or transcatheter pulmonary valve replacement.

Enrollment goal: 20 patients

Primary investigator: Lisa Bergersen, MD MPH

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


Title: Anatomic and Hemo-dynamic results of the Cone operation for Ebstein's malformation of the tricuspid valve

Description: The Cone operation is a procedure to repair the tricuspid valve in patients with Ebstein's anomaly using the patient’s own tissue. Short term results have shown that the Cone repair has improved valvar function as compared to conventional Tricuspid valve type repair. The purpose of this study is to determine long terms results of tricuspid valve function, right ventricular remodeling and quality of life at our institution with noninvasive imaging, exercise testing and a quality of life questionnaire.

Enrollment goal: 130 patients

Primary investigator: Gerald Marx, MD

Contact: Mariana Chávez, MD: Mariana.Chavez@childrens.harvard.edu or 617-355-3515


Title:  The National Pediatric Cardiology Quality Improvement Collaborative- A Collaborative Initiative to Improve the Care of Children with Complex Congenital Heart Disease- Phase II

Description:  The National Pediatric Cardiology Quality Improvement Collaborative is a research and quality improvement network whose mission is to improve the outcomes of care for children with single ventricle congenital heart disease.  Parents consent to enrollment in a Registry Database and statistical analysis informs Quality Improvement Efforts and Outcome Assessment.

Enrollment Goal: Open Enrollment for the next 3-5 years

Primary investigator: David Brown, MD

Primary Contact: Terry Saia PNP: terry.saia@cardio.chboston.org or 617-355-8437


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

Description: 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:physician pairs including participating NECCA sites.

Primary Investigators: Drs. Laura Mansfield and David Brown

Contact: Laura Mansfield, Laura.Mansfield@cardio.chboston.org or 978-501-6176


Title: Apixaban vs. VKA or LMWH in Pediatric Subjects with Congenital or Acquired Heart Disease on Chronic Anticoagulation for Thromboembolism Prevention

Description: This is a multi-center, randomized trial designed to assess the safety and pharmacokinetics of the anticoagulation agent Apixaban compared to conventional anticoagulation drugs in children with acquired and congenital heart disease. At Boston Children’s Hospital, up to 40 children between 2 to < 18 years of age will be enrolled and receive therapy for up to 12 months.

Enrollment goal: 40

Primary investigator: Christina VanderPluym, MD

Contact: Najveen Alvi: Najveen.Alvi@cardio.chboston.org or 617-355-7245


Title: Pharmacodynamics, Pharmacokinetics, Safety and Efficacy of Rivaroxaban for Thromboprophylaxis in Pediatric Subjects 2 to 8 Years of Age after the Fontan

Description: This prospective, open label active controlled multi-center study is tasked with the goal to evaluate the safety and optimal dosing of rivaroxaban (an oral anticoagulation agent) compared to acetylsalicylic acid for thrombus prevention in children aged 2 to 8 years with single ventricle physiology who have completed the Fontan procedure. Participants will receive therapy for up to 12 months.

Enrollment goal: 25

Primary investigator: Christina VanderPluym, MD

Contact: Priscilla Cevallos: Priscila.Cevallos@cardio.chboston.org


Title: PumpKIN (Pump for Kids, Infants and Neonates) Safety and Efficacy of Jarvik VAD for Children Sized 8 to 20 kg

Description: This multi-center two-arm clinical trial will evaluate the safety and potential benefit of the Jarvik 2015 versus the EXCOR Pediatric as a bridge to transplant in smaller children with severe heart failure.

Enrollment goal: 15

Primary investigators: Christina VanderPluym, MD and Francis Fynn-Thompson, MD

Contact: Christina VanderPluym, MD: Christina.vanderpluym@cardio.chboston.org


Title: Evaluation of the safety and tolerability of oral ENTRESTO for the treatment of pediatric heart failure patients

Description: Multicenter, 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 patients

Primary Investigator: Kevin P. Daly, MD

Contact: Kevin Daly, MD: kevin.daly@cardio.chboston.org or Jane Messere, BSN RN at: 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

Primary Investigator: Kevin P. Daly, MD

Contact: Kevin Daly, MD: kevin.daly@cardio.chboston.org or Jane Messere, BSN RN at: 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: Validation of a novel oxygen consumption measurement technique in neonates

Description: In collaboration with a company, we have created a device to measure the number of calories a baby consumes while on the ventilator, something that cannot be currently measured accurately.  We will be studying this device in newborn infants in the CICU and NICUs at BCH for 1 hour each, comparing measured values to a gold standard.  

Enrollment goal: 50

Primary investigator: John N. Kheir, MD

Contact: John N. Kheir, MD: John.Kheir@cardio.chboston.org or 857-636-8890


Title: Correlation of CMR and in vitro measures of aortic stiffness in connective tissue disease patients undergoing aortic root replacement

Description: Prior to planned aortic root replacement, patients will be enrolled and a clinical CMR study will be performed. After explant of the aorta, tissue samples will be tested for biaxial elasticity. These measures will be compared with CMR-based measures of aortic stiffness to develop novel biomarkers of aortic structural health.

Enrollment Goal: 25 patients (at CHB and BWH)

Primary Investigator: Ashwin Prakash

Contact: Aaron Prosnitz: aaron.prosnitz@cardio.chboston.org, or 401-440-3094


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

Description: The Edwards Pericardial Bioprosthesis, Model 11000A, is indicated for patients five 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, Multicenter study. Up to one hundred (100) pulmonary valve replacement (PVR) subjects at up to ten (10) clinical sites will be enrolled. Clinical data will be collected from patients enrolled for 5 years after the 100th patient is enrolled.  Data will be collected from at least 3 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, bio-absorbable, 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 homograft’s 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 of age of below 18 years old.

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


Title: HALO Clinical Study:  A single arm, prospective, non-randomized, multi-center clinical investigation of the SJMTM Masters HP 15mm Rotatable Mechanical Heart Valve 

Description:  With the current commercially available mechanical mitral valves size ranges of 16mm to 37mm and the mitral annulus in young pediatric patients ranging from 12mm to 30mm with a mean of 18mm and evidence existing that suggests oversized valves are associated with increased mortality and morbidity rates particularly in the very young patient population, a 15mm investigational mechanical heart valve (MHV) is being studied.  A mitral valve annulus will typically require a sewing diameter of approximately 2mm larger than the valve size. As a 16mm valve is currently the smallest diameter valve on the market there is a portion of the pediatric population that would benefit from the availability of a smaller diameter valve.  This is made possible by removing the sewing cuff from the annulus.  The investigational device is a 15mm, rotatable, bi-leaflet MHV designed for implantation in the mitral position. This valve is part of the SJMTM Masters HP Series product line. The study is a single arm, prospective, non-randomized, multi-center clinical investigation. Each enrolled subject will be assessed at baseline, procedure, post- procedure, 30 days, six months, 12 months, and annually thereafter for as long as the valve remains implanted, but not to exceed five years. 

Enrollment goal: 2-3 Subjects per site.  Total trial goal enrollment is 20.

Primary investigator: Christopher W. Baird, MD

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


Title: Data Fusion: A Sustainable, Scalable, Open Source Registry Advancing PVD Research

Description: We are recruiting patients diagnosed with pediatric pulmonary hypertension (PH) before the age of 18. No additional visits or tests will be conducted and all information will be de-identified. Data will be compared to others to learn more about treatment and improve care for patients like you or your child.

Enrollment Goal: 1200+

Primary investigator: Mary Mullen MD, PhD

Contact: Jessica Mecklosky, Jessica.Mecklosky@cardio.chboston.org or 617-919-4456


Title: Placenta Biopsies in Hypoplastic Left Heart Syndrome Patients

Description: Placental biopsies will be collected from women with fetuses diagnosed with hypoplastic left heart syndrome (HLHS) at the time of delivery. The goal is to compare the growth of stem cells from aplacenta of a child with HLHS to cells from neonates with no known medical diagnosis.

Enrollment goal: 10

Primary investigator: Sitaram Emani, MD

Contact: Bre Piekarski, RN, BSN: Breanna.Piekarski@cardio.chboston.org or 617-919-4457


Title: Evaluation of Coagulation Testing in Patients Undergoing Cardiac Surgery

Description: Discarded blood from routine analysis will be used to evaluate the input of newer technologies for diagnosis of coagulopathy. The goal is to design an algorithm using point-of-care monitoring that could be used to diagnose coagulopathies and to guide hemostatic therapies to alleviate morbidity and mortality in children undergoing cardiac surgery.

Enrollment goal: 250

Primary investigator: Juan Ibla, MD

Contact: Bre Piekarski, RN, BSN: Breanna.Piekarski@cardio.chboston.org or 617-919-4457


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

Description: It is hypothesized that pre-op surgical planning via inspection of a 3-D printed cardiac model will reduce time spent inspecting the intracardiac anatomy, and thus also reduce OR time, reduce the incidence of residual lesions, and improve comfort and confidence levels of the surgeon.

Enrollment goal: 30

Primary investigator: Sitaram Emani, MD

Contact: Bre Piekarski, RN, BSN: Breanna.Piekarski@cardio.chboston.org or 617-919-4457


Title: Transplantation of Autologously Derived Mitochondria for Protection against Ischemia-Reperfusion Injury following Ischemia Due to ECMO

Description: A robust therapeutic intervention is proposed to ameliorate myocardial ischemia/ reperfusion injury and significantly decrease morbidity and mortality in patients requiring extracorporeal membrane  oxygenation (ECMO), by direct injection of autogeneic mitochondria into the ischemic myocardium.

Enrollment goal: 10

Primary investigator: Sitaram Emani, MD

Contact: Bre Piekarski, RN, BSN: Breanna.Piekarski@cardio.chboston.org or 617-919-4457


Title: Mesoblast Stem Cell Therapy for Patients with Single Ventricle and Borderline Left Ventricle

Description: This is a randomized trial for those patients with hypoplastic left heart syndrome (HLHS), unbalanced atrioventricular canal (uAVC), or borderline left heart undergoing LV recruitment or future plans  for recruitment. Subjects will receive mesenchymal precursor cells (MPCs) injected directly into the LV endocardium during their planned surgical procedure.

Enrollment goal: 24

Primary investigator: Sitaram Emani, MD

Contact: Bre Piekarski, RN, BSN: Breanna.Piekarski@cardio.chboston.orgor 617-919-4457


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: A risk stratification model will be created utilizing a large, multi-institutional registry under the auspices of the Congenital Heart Surgeon Society (CHSS). The purpose of the study is to determine the outcome of surgical intervention versus observation in children and young adults with AAOCA.

Enrollment goal: 200

Primary investigator: Meena Nathan, MD

Contact: Bre Piekarski, RN, BSN: Breanna.Piekarski@cardio.chboston.org or 617-919-4457


Title: Preserved Umbilical Vein Use as Autologous Shunt Conduits in Neonatal Cardiac Surgery

Description: This is a safety and feasibility trial to evaluate the use of autologous umbilical vein as shunts/ conduits, as well as patches in neonatal cardiac surgery. At time of delivery, umbilical cord will be  harvested with the umbilical vein dissected free and preserved until first clinically indicated Stage I palliative procedure.

Enrollment goal: 16

Primary investigator: Sitaram Emani, MD

Contact: Bre Piekarski, RN, BSN: Breanna.Piekarski@cardio.chboston.org or 617-919-4457