Clinical Research To improve care and clinical outcomes for infants, children, and adolescents with acute and chronic medical conditions, the Connecticut Children’s Research Institute supports groundbreaking clinical trials. Identification and testing of novel therapies requires a rigorous clinical trials program and infrastructure to support a variety of therapeutic studies. Our clinical trials team undertakes interventional studies ranging from Phase I assessment of toxicity of a new agent, to Phase II efficacy evaluations, to Phase III trials aimed at determining whether a new therapy will improve care for patients when compared to a previously determined standard of care. The collaboration between the clinical trials program and our scientific leaders provides for investigative growth in both programs, with fresh discoveries leading to new clinical trials and innovative treatments for the children and families we serve. Connecticut Children’s investigator-initiated trials partners with various sponsors to facilitate multi-center trials many of which are focused on rare diseases in neurology, nephrology and endocrinology, gastrointestinal disorders, hematological and oncological disorders, neonatology, and infectious disease. Gastrointestinal Disorders Dr. Jeffrey Hyams is leading an NIDDK-funded multi-site project called CAMEO: Clinical, Imaging, and Endoscopic Outcomes of Children Newly Diagnosed with Crohn’s Disease. This study will be conducted across 27 sites, with Connecticut Children’s participating as the lead site. CAMEO will examine the association between pre-treatment clinical, radiologic, genomic, and microbial factors and a patient’s ability to achieve complete healing after one year of optimized anti-TNF therapy. This will include personalized anti-TNF biologic therapy guided by therapeutic drug monitoring and a novel dosing algorithm developed by the study team. Hematological/Oncological Disorders Dr. Mike Isakoff is the site PI for Children’s Oncology Group, an international consortium of pediatric oncology centers aimed at conducting Phase I, II, and III clinical trials, along with biology studies for children, adolescents and young adults with cancer. Dr. Isakoff, in partnership with his scientific and medical collaborators world-wide, pursue vital investigative research to identify cancer pathologies and treatment models for children and families. Rare Neurologic Disorders Dr. Gyula Ascadi, Division Head of Neurology conducts clinical research in the areas of muscular dystrophies, including Duchenne muscular dystrophy and Charcot-Marie-Tooth disease, as well as spinal muscular atrophy (SMA). He currently has six ongoing clinical trials to better understand longitudinal outcomes of therapy that will be useful in guiding subsequent clinical trials. Endocrine Disorders Connecticut Children’s has a robust Glycogen Storage Disease Program and Disorders of Hypoglycemia. This is a joint program between Connecticut Children’s and the University of Connecticut, the mission of which is to pursue research while providing evidence-based care for children with these diseases. Rebecca Riba-Wolman, MD, Clinical Director of Endocrinology and Diabetes, is the lead of the largest collaboration site for the world’s first glycogen storage disease human gene therapy clinical trial. This work was started by David Weinstein, MD, MSc and was launched at University of Connecticut Health Center in July 2018—an advance 20 years in the making, and thus has yielded promising results. Her research extends to other forms of hypoglycemic disorders, both acquired and congenital. Pain & Palliative Medicine The Division of Pain & Palliative Medicine at Connecticut Children’s Medical Center is at the forefront of pediatric pain research. Our top physicians and researchers are dedicated to easing children’s pain with clinical advances, supported by commercial, private and federal grants from the National Institutes of Health (NIH). Emily Wakefield, PsyD, is dedicated to understanding the role and impact of psychosocial factors for children and adolescents with chronic pain on health outcomes and is focused on developing clinical interventions that will improve quality of life in this population. William Zempsky, MD, is the Division Head of Pain & Palliative Medicine. His research over the past 20 years has focused on assessment and treatment of pain in youth and young adults, with a major goal to bring cutting-edge pain research techniques to advance evaluation and management of pain in this population. Clinical Trials Unit The Clinical Trials Unit, housed within the Connecticut Children’s Research Institute, provides study coordination, lab services, regulatory and IRB submission management, quality assurance, education and consultation to clinical research investigators from study start-up to study closure for externally funded research studies. They provide guidance and study coordination for investigator-initiated investigational drugs and devices submissions and Expanded Access Treatment Programs (compassionate use) for patients/families who have immediate life-threatening illnesses. The unit partners with investigators to ensure study compliance to all local and external agencies. The team coordinates and manages funded and consortia phase II-IV clinical trials and non-interventional studies. For our most ill patients/families, many of our clinical trials provide opportunities to access investigational medications not otherwise available. The Clinical Trials Unit has dedicated office space and private, research patient rooms equipped with infusion chairs and equipment to ensure safety and monitoring of all clinical trial activity. There is also additional clinical lab space that allows for the processing, packaging, shipping and storing research specimens.