The Pediatric Trials Network (PTN) said “Dumela” (hello in Setswana) to Botswana as it activated a Botswana-based site in January. The site has already enrolled several 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 important questions about breastfeeding for women living with HIV/AIDS around the world.

Botswana, like many other countries in Africa, was hit hard by the HIV/AIDs pandemic more than a decade ago. However, with its government’s leadership and commitment to providing free antiretroviral medications, the once devastating disease has become a chronic condition for many. Today, over 20% of the adult population lives with HIV and 82% of those people take antiretroviral medications every day. But many women taking these medications who are also breastfeeding their infants have unanswered questions about safety. While studies indicate that the transmission of HIV from mother to child is reduced if the mother is on antiretroviral medications, there are not many studies that describe the extent of antiretroviral drug transfer into breastmilk. Because of the prevalence of HIV in Botswana and its government’s commitment to treating the disease, many women are likely to be interested in helping to find answers to these important questions. The BMS02 study will specifically study the following drugs:

• Dolutegravir
• Emtricitabine
• Tenofovir disoproxil fumarate
• Lamivudine

A better understanding of the safety of common antiretroviral medications in breastfeeding infants will provide data to help clinical professionals everywhere to advise breastfeeding mothers who live with HIV/AIDs. Confirmed safety might promote breastfeeding and benefit both mothers and their children.

This partnership will build on collaborations that Duke University Associate Professor of Pediatrics and Global Health, Matthew Kelly, MD, MPH has already established with Botswana.
“We are so excited to partner with PTN on this important study that has implications both nationally and internationally for women and their infants,” said Dr. Kelly. “This is an important question for women living with HIV everywhere.”

“We are happy to build on UPenn’s existing collaboration,” said Angelique Boutzoukas, Principal Investigator for the Project. “Expanding PTN research into low- and middle-income countries, such as Botswana, means that PTN can expand its mission to impact even more children’s lives through better evidence for medications.”

Members of the Pediatric Trials Network (PTN) team recently had the opportunity to attend, participate, and present at the 2023 International Children’s Advisory Network (iCAN) Summit in La Jolla, California.

Global Trials Associate Director, Phyllis Kennel, and Research Communications and Engagement Specialist, Meagan Daly, presented to a group of young people and industry representatives. The presentation familiarized the group with the important work of DCRI Pediatrics and PTN. The presentation also provided updates on the ways in which iCAN participation has helped inform the work of PTN.

One of the most recent collaborations between iCAN and PTN has centered around the development of a pediatric anthology. This body of work includes contributions from pediatric patients and are reflections on their experience with pediatric clinical care. The ultimate goal is to produce a hard copy of this content that will be made available as an engagement tool for DCRI pediatric trial participants.

iCAN is a worldwide consortium of children’s advisory groups, known as Kids Impacting Disease Through Science (KIDS) and Young Persons Advisory Groups (YPAGS). These dedicated youth member groups work in unison around the world to provide a voice for children and families in medicine, research, and innovation.

PTN served as a sponsor of the 2023 Summit along with Jumo Health, Pfizer, Bio Cryst, Fortera, Lilly, and others. Presence at the Summit afforded a variety of networking opportunities and fruitful discussions with industry sponsors to ensure we all working together to advance clinical research and care for pediatric populations.

PTN looks forward to a continued partnership with iCAN and all the opportunities it provides in helping advancing the care of our youngest patients.

In late April, the Pediatric Trials Network (PTN) sponsored a session at the Pediatric Academic Societies (PAS) Annual Meeting in Washington, D.C. The event involved quick debates between PTN investigators and collaborators on topics related to pediatric clinical research. A patient advocate and student with experience participating in pediatric clinical research shared her thoughts on each topic. The event was attended by more than 100 people. Participation from the audience, as well as the perspectives offered by the patient advocate, made for engaging debates.

This session was co-led with the International Children’s Advisory Network (iCAN). Rhiannon Perry, the patient advocate participating in the event, is a member of iCAN Young Adult Professionals, and ambassador for the Hope for Henry Patient Advisory Board and the Be the Match Network. She is studying Sociology, Human Services, and Psychology at Mount St. Mary’s University. After each debate, Rhiannon shared her thoughts on the investigators’ stances. Her participation brought the valuable perspective of someone who is personally involved with pediatric clinical research.

On participating in clinical trials, Rhiannon said “Having a disease is a trial whether you’re enrolled in one or not. You take what medication you can and hope that works…We sign on in hopes we can get something beneficial out of it.”

The debates were on two topics, with two debaters for each topic. The speakers had four minutes to make their arguments, followed by one minute for rebuttal. Debaters were assigned the side they argued for. Before each debate, the audience was polled on their opinion. After listening to the investigators and Rhiannon, they were surveyed again to see if their minds changed. Audience members also had the chance to ask questions of the debaters.

The first debate was between Gregory L. Kearns, PharmD, PhD, FAAP and Roger Soll, MD, FAAP on the question “What are the most important types of studies that should be done to move the field of pediatric therapeutics forward?” Dr. Kearns argued for smaller, dose ranging studies. Dr. Soll argued for large, randomized controlled trials. The second debate was between Danny Benjamin, MD, PhD, MPH and Chris Feudtner, MD, PhD, MPH, HCEC-C on the question “Should pediatric participants be incentivized to participate in drug and device studies?” Dr. Benjamin argued yes, while Dr. Feudtner argued that there are ethical concerns.

The PTN thanks everyone who attended and participated in this event at the PAS Annual Meeting and hopes that these debates inspired new ideas and ways of thinking about pediatric clinical research.

View a video from the meeting and photos below:

 

 

The Pediatric Academic Society (PAS) meeting is scheduled to take place April 27 – May 1  in Washington, D.C. Investigators from The Pediatric Trials Network (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.

PTN investigators plan to discuss the most important types of studies that should be done to move the field of pediatric therapeutics forward and whether/how pediatric participants should be incentivized to participate in drug and device studies.

Attendees of the session will listen to arguments and have the opportunity to vote for which perspective they agree with most. Additionally, attendees will have the opportunity to learn more about PTN and engage in discussions with the investigators leading the network.

Presenters include:

• Danny Benjamin, MD, PhD, MPH
  Distinguished Professor of Pediatrics
  Duke Clinical Research Institute
• Chris Feudtner, MD PhD MPH HCEC-C
  Chief, Division of General Pediatrics
  The Children’s Hospital of Philadelphia
• Rachel Greenberg, MD, MB, MHS
  Associate Professor of Pediatrics
  Duke Clinical Research Institute
• Gregory L. Kearns, PharmD, PhD, FAAP
  Professor of Medicine and Pediatrics
  Texas Christian University (TCU)
• Roger Soll, MD, FAAP
  Professor of Neonatology
  University of Vermont College of Medicine

For more information on the annual PAS meeting and registration, visit the PAS website.

The 2021/2022 PTN Annual Report is now available. Click here to access.

A message from PTN leadership: 

In 2022, the Pediatric Trials Network (PTN) carried out its mission to improve health for all children by performing research, engaging participants, and sharing science. To date, the PTN has informed 17 label changes for drugs and devices to include dosing/usage guidance for children. Clinical study reports on several drugs were submitted to the FDA this year and will be used to inform more label changes. The PTN had five ongoing studies in 2022, which address topics like COVID-19 treatment, anesthetics, and drug dosages for participants ranging from infants, to 21-year-olds, to breastfeeding mothers. The Sildenafil study in particular has enrolled over 80 infants across two cohorts, and is expecting to open another cohort soon.

In addition to the upcoming Sildenafil cohort, the PTN is expecting to open three new studies in 2023. Two of these studies will be a bit different than what we’re used to, because they will involve adult participants. One will study intravenous methadone in healthy adults (MTH02), and the other will study terbutaline sulfate in adults with asthma (TBS02). The answers learned from these clinical trials will inform the development of trials for pediatric participants. The third study enrolling in 2023 will evaluate guanfacine for children with Down Syndrome (HYP01).

Since last year’s report, the PTN has released nine new publications to share these important data with the scientific community. We also developed a Results-at-a-Glance webpage to be the home of quick, easy-to-read study summaries that can be shared with the public and study participants.

Diversity and participant engagement were also a big focus of the PTN in 2022. We engaged pediatric participants by asking them what diversity in pediatric clinical research means to them, as well as directly involving them with the development of clinical trials by allowing them to provide feedback on clinical trial assent forms. We developed an anthology of creative works by pediatric trial participants to spotlight their voices and experiences. Additionally, we showed gratitude to participants and their families by developing and sending personalized thank-you notes.

We hope you will read this Annual Review for more details on these accomplishments. We look forward to an exciting 2023.

Danny Benjamin, MD, PhD, MPH
Pediatric Trials Network, Principal Investigator

Rachel Greenberg, MD, MB, MHS
Pediatric Trials Network, Steering Committee Chair