Clinical Trials

At leading children’s hospitals, clinical trials are a key research tool for advancing medical education and clinical care. Most commonly, clinical trials are used to test the safety and effectiveness of drugs and devices. Clinical trials are led by research teams that can include doctors and other medical professionals.

An extensive series of clinical trials is currently underway at Connecticut Children’s, including:

Endocrinology

Primary Investigator: Elizabeth Estrada, MD
Study Title: Studies to Treat or Prevent Pediatric Type 2 Diabetes

Primary Investigator: Elizabeth Estrada, MD
Study Title: Colesevelam Oral Suspension as Monotherapy or Add-on to Metformin Therapy in Pediatric Subjects with Type 2 Diabetes Mellitus

Primary Investigator: Karen Rubin, MD
Study Title: Effect of Early Growth Hormone Treatment on Long-term Growth and Skeletal Maturation in Girls with Turner Syndrome (“Turner Tweens and Teens” Study)

Primary Investigator: Karen Rubin, MD
Study Title: The Genetics and Neuroendocrinology of Short Stature International Study

Gastroenterology

Primary Investigator: Karan Emerick, MD
Study Title: A Comparative Study of the Antiviral Efficacy and Safety of Entecavir (ETV) versus Placebo in Pediatric Subjects with Chronic Hepatitis B Virus (HBV) Infection who are HBeAg-Positive+

Primary Investigator: Karan Emerick, MD
Study Title: Evaluation of the Pharmacokinetics, Safety, Tolerability and Efficacy of Entecavir (ETV) in Pediatric Subjects with Chronic Hepatitis B Virus (HBV) Infection who are HBeAg-Positive

Primary Investigator: Jeffrey Hyams, MD
Study Title: A Multicenter International Observational Study of the Long-term Safety of Infliximab (REMICADE®)

Primary Investigator: Jeffrey Hyams, MD
Study Title: A Multicenter, Open-label Study of the Human Anti−TNF Monoclonal Antibody Adalimumab to Evaluate the Efficacy and the Long-term Safety and Tolerability of Repeated Administration of Adalimumab in Pediatric Subjects with Crohn's Disease Who Have Demonstrated a Clinical Response in the M06-806 Study

Primary Investigator: Jeffrey Hyams, MD
Study Title: A Phase 2, open-label, multicenter study to assess the safety and efficacy of
certolizumab pegol in children and adolescents with active Crohn’s disease (NURTURE Study)Multidisciplinary Human Study on the Genetic, Environmental and Microbial Interactions that Cause Inflammatory Bowel Disease (GEM)

Primary Investigator: Jeffrey Hyams, MD
Study Title: Evaluation of Exagen Diagnostic Tests in Children with Inflammatory Bowel Disease and Irritable Bowel Syndrome: A Pilot Study

Primary Investigator: Jeffrey Hyams, MD
Study Title: Predicting response to intravenous corticosteroid therapy in pediatric patients

Primary Investigator: Jeffrey Hyams, MD
Study Title: Risk Stratification and Identification of Immunogenetic and Microbial Markers of Rapid Disease Progression in Children with Crohn’s Disease 

Hematology & Oncology

Primary Investigator: Nathan Hagstrom, MD
Study Title: Fibrogammin P: Human Plasma Derived Factor XIII Concentrate in Patients with Congenital Factor XIII Deficiency

Primary Investigator: Nehal Parikh, MD
Study Title: A multicenter, prospective, open-label, clinical trial to assess the safety and the efficacy of a new intravenous immune globulin (IGIV3I Grifols 10%) in patients with idiopathic (immune) thrombocytopenic purpura

Primary Investigator: Nehal Parikh, MD
Study Title: A Phase 1 Trial of TPI 287 as a Single Agent and in Combination with Temozolomide in Patients with Refractory or Recurrent Neuroblastoma or Medulloblastoma

Infectious Diseases

Primary Investigator: Melissa Held, MD
Study Title: Rotarix Surveillance Activity for Gastroenteritis among Infancy <19 months of age

Primary Investigator: Juan Salazar, MD
Study Title: A Phase 2, Open-Label, Multicenter Study of the Safety of Ritonavir-Boosted GS-9137 (GS-9137/r) Administered in Combination with Other Antiretroviral Agents for the Treatment of HIV-1 Infected Subjects

Neonatal Intensive Care Unit (NICU)

Primary Investigator: James Hagadorn, MD
Study Title: Inhaled Nitric Oxide for the Prevention of Bronchopulmonary Dysplasia (BPD) in Preterm Infants Requiring Mechanical Ventilation or Positive Pressure Support on Days 5-14 after Birth

Primary Care

Primary Investigator: Catherine Wiley, MD
Study Title: A Phase 1/2a, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, Immunogenicity, and Viral Shedding of MEDI-559, a Live Attenuated Intranasal Vaccine Against Respiratory Syncytial Virus in Healthy 1 to <24-Month-Old Children

Pulmonary Medicine

Primary Investigator: Craig Schramm, MD
Study Title: A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to
Evaluate the Safety and Efficacy of VX-770 in Subjects Aged 12 Years and Older with Cystic Fibrosis who are Homozygous for the F508del-CFTR Mutation

Primary Investigator: Craig Lapin, MD
Study Title: A Phase 3, Multicenter, Multinational, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of MP-376 (Levofloxacin Inhalation Solution; Aeroquin™) In Stable Cystic Fibrosis Patients

Primary Investigator: Craig Lapin, MD
Study Title: A Phase 3, Open-Label, Randomized Trial to Evaluate the Safety and Efficacy of MP-376 Inhalation Solution (AEROQUIN™) Versus Tobramycin Inhalation Solution in Stable Cystic Fibrosis Patients

Primary Investigator: Craig Lapin, MD
Study Title: iCARE: I Change Adherence & Raise Expectation

Rheumatology

Primary Investigator: Barbara Edelheit, MD
Study Title: Improved Understanding of the Biology and Use of the TNF Inhibition in JIA

Primary Investigator: Lawrence Zemel, MD
Study Title: A 12-Week Randomized, Double-Blind, Placebo Controlled, Parallel Group, 2-Arm Study to Evaluate the Efficacy and Safety of Tocilizumab in Patients with Active Systemic Juvenile Idiopathic Arthritis (SJIA); with a 92-Week Single Arm Open-Label Extension to Examine the Longterm use of Toxilizumab

Primary Investigator: Lawrence Zemel, MD
Study Title: A 15-Week, Randomized, Double-Blind, Parallel-Group, Placebo-Controlled, Flexible-Dose, Safety and Efficacy Study of Pregabalin in Adolescents (12-16 years old) with Fibromyalgia

Primary Investigator: Lawrence Zemel, MD
Study Title: A Phase 4, 6-Week, Randomized Double-Blind, Multicenter, Active-Controlled Trial to Evaluate the Effects of Celecoxib (Celebrex) or Naproxen on Blood Pressure in Pediatric Subjects with Juvenile Idiopathic Arthritis

Primary Investigator: Lawrence Zemel, MD
Study Title: Randomized Placebo Phase study of Rilonacept in the Treatment of systemic juvenile idiopathic arthritis (RAPPORT)