CLINICAL RESEARCH Clinical Research’s mission is to conduct and catalyze high-impact, internationally recognized research and policy work that improves the health, health care, well-being, and population health of infants, children, adolescents, and communities. This mission is accomplished through several mechanisms: 1) conducting research on high impact topics such as health services research, cutting-edge treatment modalities, and translational research; 2) providing a comprehensive research infrastructure to support all Connecticut Children’s investigators, which includes research support staff, a statistical core, and expert input on research operations and management; 3) mentoring early investigators, including high-school students, undergraduates, medical students, residents, fellows, and junior faculty; and 4) disseminating and publicizing the research findings of Connecticut Children’s investigators. Clinical Research is staffed by more than 35 highly skilled professionals, including research managers, associates, and assistants, biostatisticians, and certified clinical trial study coordinators and nurses. Connecticut Children’s research portfolio is supported by grants from a variety of federal agencies (the National Institutes of Health, Health Resources and Services Administration, Canada Institutes of Health Research, the Centers for Disease Control and Prevention, and the National Science Foundation), state agencies (the Connecticut Department of Public Health, and the Connecticut Department of Children and Families), foundations (Alvord Foundation, National Pediatric Cancer Foundation, Crohn’s and Colitis Foundation of America, and the GSD Foundation), philanthropy, and the pharmaceutical industry. Connecticut Children’s has robust research collaborations with UConn Health, UConn Storrs, and the Jackson Laboratory for Genomic Medicine. There were several noteworthy accomplishments for the Department of Research in 2020. These include the successful recruitment of a director of Research
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ANNUAL ACADEMIC REPORT 2020
Operations, implementation of a Covid-19 repository study, national and international presentations, groundbreaking clinical trials, recruitments and promotions, research training programs, advances in translational research, and the publication of 18 articles. We are excited to announce the successful recruitment of Garry Lapidus, PA-C, MPH, as director of Research Operations and Development. Mr. Lapidus is former director and co-founder of the Injury Prevention Center at Connecticut Children’s. He is a national leader in injury prevention research, education and training, community-based programs, and public policy. In recognition of his contributions, he has received the Charles Huntington Award from the Connecticut Public Health Association (2013), and the Distinguished Career Award from the American Public Health Association (2020). In addition to his work as director, Mr. Lapidus fills a clinical role in providing care as a physician assistant to sick and injured children in the Department of Emergency Medicine. In 2020, he was also named co-director of the Office of Advanced Practice, and he provides institutional leadership for Connecticut Children’s physician assistants and advanced practice registered nurses. Mr. Lapidus holds a senior faculty appointment as an associate professor in Pediatrics and Public Health at the University of Connecticut School of Medicine. At the beginning of the Covid-19 pandemic, Physicianin-Chief Juan Salazar, MD, MPH, supported by Katherine Herbst, MS, and Mabeline Velez, MS, collaborated with the Jackson Laboratories to develop a research resource for scientists studying Covid-19 and Covid-related conditions in children. The Patients Under Investigation for Covid-19 Repository captures data and biological samples from children hospitalized at Connecticut Children’s for Covid-19 and related conditions including multisystem inflammatory syndrome in children (MIS-C). The team was able to rapidly develop and initiate the repository in early April, resulting in the successful recruitment of over 50 severely ill children.
The repository is an invaluable body of information that is available to scientists in Connecticut and across the country. Ms. Herbst continued in her role as a member of the European Society for Paediatric Urology’s Research Committee. She presented an invited educational session entitled “Basic Statistics: Find Out If You Are Average or If You Are Mean” at the Society’s ESPU Virtual 2020 meeting. The meeting included 35 colleagues presenting from 17 different countries and three continents. Our Clinical Trials team supported several leadingedge studies that hold promise for new treatment options for our patients in including the successful implantation of a new device. With Frederic Bernstein, DO, as principal investigator, and coordinated by James Santanelli, MS, MPH, we are pleased to announce the successful implantation of a Lifetech CeraFlex FASD closure device for a patient with a congenital diagnosis of secundum atrial septal defect through the FDA’s compassionate use program. This is the first in-human implantation performed in the United States for this device. Throughout the pandemic, Clinical Trials team members, including Hendriana Nielsen, RD, BSN, Robin Arens, BS, CCRP, and Mr. Santanelli, successfully ensured uninterrupted access to critical treatment trials for patients in Hematology/Oncology, Neurology, and Gastroenterology. In addition, Clinical Trials team members collaborated with the Office for Sponsored Programs and other ancillary departments to quickly open a trial for two patients with Duchenne’s muscular dystrophy who needed access to a critical investigational medication and had been previously traveling out-of-state to obtain access. Clinical Trials team members, including Bunly Kuoch and James Ransom, MA, quickly pivoted to support the Research Department for Dr. Salazar’s Patients Under Investigation for Covid-19 Repository, in the successful collection and delivery of bio specimens to the Jackson Laboratories.
