Issue 43 | March 2024

Hello from the PTN,

We hope your 2024 has been off to a good start! As we exit winter and head toward spring, the Pediatric Trials Network (PTN) is excited to share what we’ve been up to in the first quarter of 2024.

Early this year, the PTN reached a huge milestone in informing its 20th U.S. Food and Drug Administration (FDA) label change. Data collected from several PTN studies and more than 15 years of research were used to update the label for the antifungal medicine fluconazole. The new label includes information for providers to consider when prescribing fluconazole to infants.

A new site for the study Pharmacokinetic and Safety of Antiretroviral and Related Drugs in Lactating Women and Breastmilk Fed Infants (BMS02) opened in Botswana, and it has already enrolled several participants. This study site is learning how medicines used for the treatment of HIV/AIDS may transfer from mother to baby through breastfeeding. Information learned from this study will help advise mothers and providers across the world.

Another study milestone was reached by Pharmacokinetics, Pharmacodynamics, and Safety of a Single Dose Intravenous Methadone in Healthy Adult Volunteers (MTH02). This study has completed enrollment, and its findings will lay the groundwork for future studies of methadone in children.

The PTN will be hosting an ancillary event at this year’s annual Pediatric Academic Societies Meeting in Toronto in May. Join us to learn more about our work and generate ideas about pediatric and maternal therapeutics. The PTN is teaming up with the International Children’s Advisory Network (iCAN) to help raise funds to send advocates to their Annual Summit in July. Please consider donating to help reach our goal!

See a summary of the PTN’s activities in 2023 in the 2023 Annual Review, which is now available online. Stay connected with PTN news throughout the year by connecting on our new LinkedIn page, and checking for monthly results-at-a-glance summaries.



Thank you for your continued work and support in making medicines and devices safer for pediatric patients. We’re looking forward to an exciting year!

Sincerely,

PTN research informs fluconazole label change

Data from multiple PTN studies contributed to an FDA label change for fluconazole. Previous labels for fluconazole contained almost no information related to dosages for infants, even though it is commonly used to treat Candida (yeast) infections, particularly in the neonatal intensive care unit (NICU) and pediatric intensive care unit (PICU). The new label contains information on pharmacokinetics and dosing suggestions, as well as safety and efficacy of fluconazole for both treatment and prevention of Candida infections in full-term and premature infants.

This is the 20th FDA label change informed by PTN research. This achievement represents the culmination of over 15 years of work on fluconazole from faculty and staff at the PTN, in addition to collaborating sites and thought leaders across the U.S.

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In January, the PTN said “Dumela!” (“hello” in Setswana) to Botswana as it activated a study site in the country. The site has enrolled many participants in the Pharmacokinetic and Safety of Antiretroviral and Related Drugs in Lactating Women and Breastmilk Fed Infants (BMS02) study. This study will help answer questions about breastfeeding for women living with HIV/AIDS around the world. A better understanding of the safety of common antiretroviral medications for breastfeeding infants will provide data to help clinical professionals everywhere advise breastfeeding mothers who live with HIV/AIDs.

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The PTN study Pharmacokinetics, Pharmacodynamics, and Safety of a Single Dose Intravenous Methadone in Healthy Adult Volunteers (MTH02) completed enrollment early this year. This study is evaluating how healthy adults process methadone administered through IV. The results will lay the groundwork for studying methadone in pediatric populations.

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PTN at PAS 2024

The PTN will be sponsoring an ancillary event in collaboration with MPRINT at this year’s Pediatric Academic Societies (PAS) Annual Meeting in Toronto. The event will be a Shark Tank-style presentation on pediatric and maternal therapeutics. A panel will act as the “shark tank” and react to ideas. The event will take place at the conference on Saturday, May 4, at 12:30pm – 2pm.

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The PTN has a long-standing relationship with the International Children’s Advisory Network (iCAN), a worldwide consortium of children’s advisory groups. iCAN's dedicated youth member groups work in unison around the world to provide a voice for children and families in medicine, research, and innovation. PTN partners with iCAN to ensure the voices of children and families are considered and incorporated into as much work of the PTN as possible.

Each year, iCAN hosts a Summit, providing iCAN members with the opportunity to gather and learn in a collaborative environment. This year, the Summit will be held in Bari, Italy, and our goal is to raise funds to send children from the KIDS Uganda Chapter (some pictured and recorded below) to the event.

If you're interested in participating, you can scan the QR code below or visit the fundraising page here: https://www.zeffy.com/en-US/team/50e56f16-7480-467f-9536-042609f6ee38

OUR GOAL: $5,000 (Which will cover the cost of sending two kids to the Summit)

TIMELINE: The fundraising will take place from March 22 - April 5

VIDEO: https://www.youtube.com/watch?v=Eo6jXejZ9bA

2023 Annual Review

The PTN Annual Review for 2023 was sent via email on March 1 and is now available on the PTN website. This document captures PTN’s research and activities in the last year. Find it and past Annual Reviews on the new PTN Annual Review webpage.

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Ticarcillin-clavulanate results-at-a-glance

The PTN 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 results-at-a-glance summaries. The most recently completed summary was on a PTN study to learn the pharmacokinetics of ticarcillin-clavulanate, an antibiotic. This information is necessary for health care providers to prescribe the safest, most effective dose of ticarcillin-clavulanate when treating infections in premature infants.

READ

Keep an eye on the Results-at-a-Glance webpage for more study summaries as they are completed.

Connect with the PTN

The PTN has expanded its online network to include LinkedIn! Connect with us on LinkedIn to build a community of pediatric clinical research investigators, collaborators, and advocates, and stay up-to-date with the PTN.

CONNECT

PTN label changes to date

Under the Best Pharmaceuticals for Children Act (BPCA), the PTN provides the FDA with information to inform drug label changes with the information needed to prescribe the most appropriate doses of the medications to children. As a result of research conducted through the BPCA program the following label changes have been made:

• Acyclovir
• Ampicillin
• Caffeine
• Clindamycin (x2)
• Diazepam
• Doxycycline
• Fluconazole
• Levetiracetam
• Lisinopril
• Lithium
• Lorazepam
• Mercy babyTAPE
• 2D Mercy TAPE
• 3D Mercy TAPE
• Meropenem
• Propylthiouracil
• Pralidoxime
• Rifampin
• Sodium nitroprusside

READ MORE ABOUT BPCA

PTN studies currently enrolling:

The PTN has six ongoing studies, all are currently enrolling. Click the links below to learn more about each study.

• Anesthetics and Analgesics in Children (ANA)
• A Prospective, Blinded, Cross-Over Trial of the Exposure-Response Relationship of Terbutaline Sulfate in Adults with Asthma (TBS02)
• Commonly used drugs during lactation and infant exposure (CUDDLE)
• Pharmacokinetics, Pharmacodynamics, and Safety Profile of Understudied Drugs Administered to Children per Standard of Care (POP02)
• Pharmacokinetic and Safety of Antiretroviral and Related Drugs in Lactating Women and Breastmilk Fed Infants (BMS02)
• Safety of Sildenafil in Premature Infants (Sildenafil II)

PTN studies recruiting soon:

• Guanfacine for Hyperactivity in Children with Down Syndrome (HYP01)

Useful PTN links:

• The PTN Publication Policy lays out the process of PTN publication writing and review, required information, and the role of the PTN Publication Committee.
• In an effort to share data with other researchers, PTN provides access to its analytical methods for evaluating drugs in several matrices including plasma, dried blood spots, urine, cerebral spinal fluid, and breastmilk. View the methods and request access to data.