Issue 22 | February 2018
A Message from the Lead Principal Investigator
2018 is off to a strong start for PTN. In this issue, we highlight a number of critical studies that are being conducted at PTN sites throughout the world. Although these studies vary widely in terms of therapeutic area and condition, one constant remains: the safety and health of children. At PTN, everything we do focuses on making sure that doctors and families get the information they need to make the best decisions about children's well-being. We invite you to share this newsletter with friends who are interested in staying up-to-date on PTN research, so they can subscribe . As always, thank you—your care and enthusiasm make this essential work possible.
Danny Benjamin, MD, PhD, MPH
PTN to assess safety of drugs passed through breastmilk
PTN is undertaking a groundbreaking study to assess the safety of commonly used off-patent medications when they are given to breastfeeding mothers. The study will track how different drugs are passed through breastmilk to determine dosing levels that are safe for both mom and baby. Although the FDA has implemented a guidance document for conducting lactation studies, off-patent drugs are not included in that rule. The PTN seeks to fill this knowledge gap. Read more .
Fifteen IDeA sites welcomed into POPS study
Fifteen sites in states participating in the NIH Institutional Development Award (IDeA) program are being onboarded into PTN's POPS study, which assesses the safety and efficacy of a variety of commonly used drugs in children and infants. The IDeA program builds research capacities in states that historically have had low levels of NIH funding by supporting research, faculty development, and infrastructure improvements. Read more .
Shakhnovich offers guidelines for treating GERD in obese kids
The common practice of dosing the stomach acid blocker pantoprazole based on children’s weight when treating gastroesophageal reflux disease (GERD) can actually cause more harm than good, said Dr. Valentina Shakhnovich, PTN investigator and Associate Program Director for the Gastroenterology Fellowship Research Program at Children’s Mercy Kansas City. Shakhnovich shared the findings, based on a PTN study, on Children’s Mercy’s Transformational Pediatrics podcast . Read more on the PTN website, or see the article published in The Journal of Pediatrics .
First site activated in study evaluating sildenafil safety
PTN is conducting a study to examine the safety and efficacy of sildenafil in treating bronchopulmonary dysplasia (BPD) in premature infants. The first study site, Golisano Children's Hospital at the University of Rochester Medical Center, was activated Jan. 2 and is currently recruiting participants. BPD is a common chronic lung disease that can lead to life-long medical problems, prolonged hospitalization, and even death. Sildenafil is increasingly being used off-label in premature infants with BPD, but the efficacy and safety for this population is currently unknown. Read more .
PTN captures valuable anthropometric data for infants
A PTN study led by Dr. Susan Abdel-Rahman of Children's Mercy Kansas City in 2015 captured anthropometric data, including leg and arm length and circumference, on more than 2,000 infants up to 90 days old. An article on the study, An Anthropometric Survey of U.S. Preterm and Full-Term Neonates , was published in Annals of Human Biology in December. The d ata were used to develop a weight estimation tool for infants in cases where scales are unavailable or their use is impractical. The related article, A Weight Estimation Strategy for Preterm and Full-term Infants , was recently published in Global Pediatric Health . Read more .
News of Interest
CTTI Charts New Pathways for Pediatric Antibacterial Drug Development
In an article recently published in the Journal of the Pediatric Infectious Diseases Society , the Clinical Trials Transformation Initiative (CTTI) lays out a roadmap for addressing an urgent public health issue: pediatric antibacterial drug development. Growing rates of antibiotic resistance have made the development of new antibacterial therapies a priority for researchers and physicians, especially in the pediatric population. The article, which reflects recommendations from CTTI’s Pediatric Antibacterial Drug Development Project , identifies five key areas for action aimed at overcoming barriers to conducting timely and efficient trials of new antibacterial drugs in children.

Two CTTI Papers Explore Perceptions on Enrolling Children in Clinical Trials
The Clinical Trials Transformation Initiative (CTTI) published two papers in December on barriers to enrolling children in clinical trials in Contemporary Clinical Trials Communications . Even though pediatric trials are required for new drugs, these trials can be especially difficult to design, enroll, and complete. CTTI’s papers explore the challenges from both the provider and parent perspective and suggest ways to overcome them. This research was part of CTTI’s Peds Trials Project .
FDA Draft Guidance Facilitates Pediatric Rare Disease Drug Development
The FDA issued a  draft guidance  on Dec. 6, outlining a potential novel approach for developing new drug therapies for rare pediatric diseases. The guidance proposes the use of modeling and simulation to optimize the design of pediatric studies, extrapolation from studies in adults to reduce the burden on children, and a single control group in a multi-arm, multi-sponsor clinical trial to compare the safety and effectiveness of more than one investigational drug.