PTN Post | Issue 39

Issue 39 | March 2023

Hello from the PTN,

2023 is off to an exciting start! At the beginning of the year, a rifampin label change, informed by PTN research, was approved by the FDA. The label will now include dosage recommendations for infants. This marks the 18^th label change by PTN to date — a big accomplishment.

Earlier this month, PTN shared the 2021-2022 Annual Review. This review is a summary of PTN efforts and accomplishments over the past year. You are encouraged to read and share it if you haven’t yet. Also, check out the PTN video that highlights the importance of diversity in pediatric clinical research. This video features research participants associated with the International Children’s Advisory Network (iCAN) as well as PTN investigators.

Looking ahead to the rest of 2023, PTN will be spotlighting investigators. The first Investigator Spotlight of the year, featuring Dr. Chi Hornik, is available on the website. PTN will also continue producing Results-at-a-Glance summaries for completed studies. Continue reading this newsletter to learn more about ongoing and upcoming PTN studies.

We hope your 2023 had a good start, and look forward to what the rest of the year will hold. Thank you for your continued work on making medications and devices safe and effective for all children.

Danny Benjamin, MD, PhD, MPH

Pediatric Trials Network, Principal Investigator

Rachel Greenberg, MD, MB, MHS

Pediatric Trials Network, Steering Committee Chair

PTN Annual Review 2021 - 2022

The PTN Annual Review for 2021-2022 was sent via email on March 2, and is now available on the PTN website. This document captures PTN’s research, participant engagement, and science shared in the last year. Find it and past Annual Reviews on the new PTN Annual Review webpage.

Rifampin Label Change

A PTN pharmacokinetic (PK) study recently contributed to a medication label change for the drug rifampin. This research effort, Pharmacokinetics (PK) of Antistaphylococcal Antibiotics in Infants, contributed PK and dosing data for rifampin. The data were submitted to the FDA and used to update the rifampin label to include dosing recommendations for infants. Thanks to this collaborative effort among PTN and FDA researchers, authorized medical providers now have appropriate guidance on how to prescribe and administer rifampin to infants.

PTN Diversity Video

In March of last year, the PTN and iCAN collaborated to produce a video that discusses the importance of diversity and representation in pediatric clinical research. The video was shared publicly and on the PTN website this year. It features kids involved with iCAN who have personal experience with clinical trials as well as PTN researchers. They shared why they think diversity is important and their reflections on increasing diversity and lowering barriers to participation in pediatric clinical research. The video also summarizes an analysis of participant representation across 33 Best Pharmaceuticals for Children Act (BPCA) studies and highlights opportunities for improvement.

Investigator Spotlight: Chi Hornik

In her PTN Investigator Spotlight, Dr. Chi Hornik shared her experiences with the PTN and pediatric clinical research as well as two encounters with research participants that remind her why this work is so important.

“I believe in PTN’s mission. I am a clinician, educator, and researcher for this very reason,” said Dr. Hornik. “I hope to help children beyond the walls of Duke Hospital, and I hope to mentor clinician scientists to join in the mission of ensuring that the research we conduct has representation of children from all races/ethnic backgrounds and social-economic status, so we can continue to move the needle forward to improve health for all children.”

PTN at the Annual PAS Meeting

The Pediatric Academic Society (PAS) meeting is scheduled to take place April 27 – May 1 in Washington, D.C. Investigators from the PTN will sponsor an ancillary event, featuring a rapid-fire debate focused on controversies in pediatric clinical trials. The PTN event will take place Saturday, April 29, from 12:30 p.m. – 2:00 p.m. at the Marriott Marquis, in the Monument room.

Results At-a-Glance

The Pediatric Trials Network works to make medications and medical devices safer and more effective for all children. As part of that work, we share what we learn with research participants and the general public through Result-At-A-Glance (formerly known as Lay Summaries). We appreciate every family who participates in research and makes this work possible.

Keep an eye on the Results At-a-Glance webpage for more study summaries throughout this year.

PTN Studies Currently enrolling:

PTN has five ongoing studies, four of which are enrolling and one that is in the follow-up phase. Pediatric participants of these studies range in age from infants to 21 years old, as well as breastfeeding mothers. Click the links below to learn more about each study.

PTN Studies Recruiting soon:

Pharmacokinetics, Pharmacodynamics, and Safety of a Single Dose Intravenous Methadone in Healthy Adult Volunteers (MTH02)
A Prospective, Blinded, Cross-Over Trial of the Exposure-Response Relationship of Terbutaline Sulfate in Adults with Asthma (TBS02)
Guanfacine for Hyperactivity in Children with Down Syndrome (HYP01)
Pharmacokinetics and Safety of Commonly Used Drugs in Lactating Women and Breastfed Infants (BMS02)

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PTN is sponsored by the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) and provides pediatricians, researchers, and regulators with new information on how children respond to medications.