Clinical Trials and Prospective Studies | Heart Murmurs Winter 2016-2017

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


Title: Placental 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 a
placenta 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.org or 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


Title: Assessing the Use of a Preventive Cardiology Lifestyle Screening (PCLS) Tool in a Pediatric Subspecialty Lipid Clinic

Description: In order to facilitate care, the Preventive Cardiology providers have developed a Pediatric Preventive Cardiology Lifestyle (PCLS) questionnaire that we believe will enable our clinical practice to identify lipid-specific high-risk lifestyle behaviors in a way that: 1) identifies lifestyle behaviors as well as longer and more intensive research questionnaires; 2) either does not delay, or even speeds up data collection on relevant lifestyle behaviors; 3) results in high patient and provider satisfaction; and 4) results in no worsening in lipid outcomes compared to usual care. Harnessing the existing infrastructure of the ongoing Subspecialty Lipid SCAMP, which collects family history, laboratory data, anthropometrics and other characteristics from the patient/ family, we will assess our present practice in Preventive Cardiology clinics with regard to time spent and satisfaction of the patient/ family and the provider. We will then deploy our newly developed questionnaire while continuing to monitor these factors for an additional 3-6 months. In this fashion, we plan to test time spent in lifestyle counseling, satisfaction, test-retest reliability, correlation with previously validated tools, and relationship to clinical outcomes.

Enrollment goal: 25

Primary investigators: S. Skylar Griggs MS, RD, LDN and Dr. Sarah de Ferranti MD, MPH

Contact: S. Skylar Griggs MS, RD, LDN: Suzanne.Griggs@childrens.harvard.edu or 617-355-4329


Title: Oral Treprostinil as Transition or Add-On Therapy for Pediatric Patients with PAH

Description: This multi-center, open-label, multiple dose study investigates the safety, tolerability and pharmacokinetics of oral treprostinil in pediatric patients (ages 7-17) with PAH. The study looks at three different cohorts: Cohorts 1 and 2 include patients transitioning from current prostacyclin use to oral treprostinil. Cohort 3 patients are de novo prostacyclin subjects.

Enrollment goal: 5

Primary investigator: Mary Mullen MD, PhD

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


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

Description: This multi-center, long-term study leverages and extends the existing Pediatric Pulmonary Hypertension Network (PPHNet) for large-scale registry and EHR-based research of patients with pulmonary
hypertension. This virtual PH research repository aims to complement traditional data sources for epidemiological research, enabling superior collection of phenotype, outcomes, and therapeutic efficacy data.

Enrollment goal: 300

Primary investigators: Mary Mullen MD, PhD and Thomas Kulik MD

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


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 on neurocognitive function for patients with repaired complex congenital heart disease who are scheduled for right ventricular outflow tract re-intervention for patients undergoing surgical or transcatheter pulmonary valve replacement.

Enrollment goal: 20

Primary investigator: Lisa Bergersen, MD MPH

Contact: Priscila Cevallos and Andi Goodman: CardioOutcomes@childrens.harvard.edu


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 approved pulmonic position.

Enrollment goal: 28

Primary investigator: Lisa Bergersen, MD MPH

Contact: Lisa Bergersen, MD, MPH: Lisa.Bergersen@cardio.chboston.org or 617-355-8228


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

Primary investigator: Lisa Bergersen, MD, MPH

Contact: Priscila Cevallos and Andi Goodman: CardioOutcomes@childrens.harvard.edu


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

Description: 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 12 hours after surgery in the CICU.

Enrollment goal: 10

Primary investigator: Juan Ibla, MD

Contact: Carolyn Dunbar-Masterson, RN: Carolyn.Dunbar-Masterson@cardio.chboston.org or 617-355-4201


Title: Fontan Udenafil Exercise Longitudinal (FUEL) Assessment Trial

Description: 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: 40

Primary investigator: Jonathan Rhodes, MD and Rahul Rathod, MD

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


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

Description: 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

Primary investigator: Meena Nathan, MD, MPH

Contact: Carolyn Dunbar-Masterson, RN: Carolyn.Dunbar-Masterson@cardio.chboston.org or 617-355-4201


Title: 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

Description: The purpose of this study is 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

Primary investigator: Jane W. Newburger, MD, MPH

Contact: Carolyn Dunbar-Masterson, RN: Carolyn.Dunbar-Masterson@cardio.chboston.org or 617-355-4201


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

Description: 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

Primary investigator: Jane W. Newburger, MD, MPH

Contact: Catherine Hartigan: Catherine.Hartigan@cardio.chboston.org or 617-919-4463


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

Description: 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

Primary investigator: Jane W. Newburger, MD, MPH

Contact: Alison Lord: Alison.Lord@cardio.chboston.org or 617-355-4201

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