Trial design

Sponsor- vs. FDA-Initiated Changes to Pediatric Clinical Trial Protocols: A Comparison of Associated Participant Burden

Posted on by Kayla Korzekwinski

Therapeutic Innovation & Regulatory Science • February 2025

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Abdel-Rahman S, Sund Z, Alderman C, Abella K, Kennel P, on behalf of the Best Pharmaceuticals for Children Act – Pediatric Trials Network Steering Committee.

Risks associated with clinical trial participation are a foremost consideration during protocol development whereas trial-associated burdens receive less focus despite their measurable impact on enrollment and retention. Of late, the U.S. Food and Drug Administration (FDA) has elevated discussions on barriers to research participation resulting from overly burdensome trials. Given the agency’s role in shaping clinical protocol design, this study examined the perceived burden associated with FDA-proposed study changes in the context of pediatric, off-patent, labeling studies.

Mentorship

Posted on by Kayla Korzekwinski

Journal of the Pediatric Infectious Diseases Society • December 2023

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Zimmerman KO

A unique feature of the Duke Summer Training in Academic Research (STAR) program is its focus on mentorship. STAR is an opportunity for junior faculty members and fellows to learn how to be mentors, and for undergraduate and high school students to learn how to be mentored. These are important and fundamental points; mentorship, from the perspective of mentor or mentee, should be a deliberate and skilled activity that can result from adequate training.

Duke STAR Program

Posted on by Kayla Korzekwinski

Journal of the Pediatric Infectious Diseases Society • December 2023

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Benjamin DK Jr

The DCRI Pediatrics group designed a summer internship program that had a wide reach and reflects its purpose and mission to conduct research to help children everywhere. This led to the creation of the Duke STAR (Summer Training in Academic Research) Program, a program that provides ~20 high school or college students per summer the opportunity to be exposed to clinical research and to work closely with researchers and practicing clinicians. Recruiting efforts prioritized participants from historically marginalized communities in North Carolina, with a goal to include at least several students each year who were “hidden talent.”

Improving Outcomes for Infants After Cardiopulmonary Bypass Surgery for Congenital Heart Disease: A Commentary on Recent Randomized Controlled Trials

Posted on by Kayla Korzekwinski

Pediatric Critical Care Medicine • November 2023

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Schlapbach LJ, Gibbons KS, Butt W, Kannankeril PJ, Li JS, Hill KD, NITRIC Study Group, STRESS Network Investigators

The recent NITRIC and STRESS trials demonstrate opportunities to perform pragmatic large randomized trials in congenital heart disease. We discuss lessons learnt from these trials which can inform future trial design and conduct in the field of pediatric heart surgery.

Assessing clinical site readiness for electronic health record (EHR)-to-electronic data capture (EDC) automated data collection

Posted on by Kayla Korzekwinski

Contemporary Clinical Trials • May 2023

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Eisenstein EL, Zozus MN, Garza MY, Lanham HJ, Adagarla B, Walden A, Zimmerman KO, Kumar KR

eSource software is used to automatically copy a patient’s electronic health record data into a clinical study’s electronic case report form. However, there is little evidence to assist sponsors in identifying the best sites for multi-center eSource studies. This site developed an eSource site readiness survey. The survey was administered to principal investigators, clinical research coordinators, and chief research information officers at Pediatric Trial Network sites.

Developing lay summaries and thank you notes in paediatric pragmatic clinical trials

Posted on by Kayla Korzekwinski

Health Expectations • June 2022

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Zimmerman KO, Perry B, Nsonwu A, Hanlen-Rosado E, Lane MD, Benjamin DK Jr., Becker M, Corneli A; on behalf of the Best Pharmaceuticals for Children Act — Pediatric Trials Network Steering Committee
This study conducted formative research to assess the acceptability of lay summaries and thank you notes, as well as to refine and expand guidance on participant and family engagement in Pediatric Trials Network’s (PTN) pragmatic paediatric clinical research. Researchers conducted in-depth qualitative interviews with adolescent clinical trial participants and caregivers of paediatric participants in four trials conducted by PTN across eight sites. This is the first study to describe stakeholder preferences for thank you note content and layout. Using these findings, researchers finalized PTN’s trial communication guidance for use in future PTN trials.

Capacity Building in a New Clinical Trials Network through Inter-Network Collaboration

Posted on by Kayla Korzekwinski

The Journal of Pediatrics • January 2022

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Knight L, Pahud BA, Scheffler M, Euteneuer JC, Allen C, Ross J, Ali W, Meyer M, Purohit PJ, Zimmerman KO, Sullivan JE; on behalf of the ECHO IDeA States Pediatric Clinical Trials Network
The Institutional Development Award (IDeA) is a congressionally mandated program helping to build research capacity by supporting research, faculty development, and infrastructure improvements in states with low National Institutes of Health (NIH) funding levels. Children, racial, and ethnic minorities, rural populations, and patients of low socioeconomic status are underrepresented in clinical research, which limits the generalizability of results. To address this gap, 17 institutions were awarded funding in 2016 to establish the IDeA States Pediatric Clinical Trials Network (ISPCTN) by the NIH’s Environmental Influences on Child Health Outcomes Program.

Pediatric Trials Network: Stakeholder views on thanking families and providing study findings on pragmatic pediatric clinical research

Posted on by Kayla Korzekwinski

Contemporary Clinical Trials Communications • May 2021

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Corneli A, Perry B, Benjamin DK Jr, Zimmerman KO
This study conducted formative research using in-depth interviews to identify preferences for and anticipated responses to receiving thank you notes and lay summaries of aggregate results among caregivers and adolescent participants of pragmatic pediatric studies conducted by the National Institute of Health-sponsored Pediatric Trials Network. Nearly all participants said receiving a thank you note would make them feel valued, appreciated, and proud because they contributed to science. Similarly, nearly all participants said that receiving a summary of research results would make them aware of their role in improving the lives of children, feel like they are an active partner in research, and believe that researchers want to keep them informed.

Evaluating Site-Level Implementations of the HL7 FHIR Standard to Support eSource Data Exchange in Clinical Research

Posted on by Kayla Korzekwinski

Studies in Health Technology and Informatics • May 2021

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Garza MY, Eisenstein E, Kumar KR, Zimmerman KO, Zozus M
Direct extraction and use of electronic health record (EHR) data is a long-term and multifaceted endeavor. A systematic mapping of study data elements was used to measure the coverage of the Health Level Seven (HL7®) Fast Healthcare Interoperability Resources (FHIR®) standard for a federally sponsored, pragmatic cardiovascular randomized controlled trial (RCT) targeting adults. Researchers evaluated site-level implementations of the HL7® FHIR® standard to investigate study- and site-level differences that could affect coverage and offer insight into the feasibility of a FHIR-based eSource solution for multicenter clinical research.

Racial and Ethnic Diversity in Studies Funded Under the Best Pharmaceuticals for Children Act

Posted on by Kayla Korzekwinski

Pediatrics • May 2021

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Abdel-Rahman SM, Paul IM, Hornik C, Sullivan JE, Wade K, Delmore P, Sharma G, Benjamin DK, Zimmerman KO

The Best Pharmaceuticals for Children Act (BPCA) incentivizes the study of on-patent medicines in children and mandates that the NIH sponsor research on off-patent drugs important to pediatric therapeutics. Failing to enroll cohorts that reflect the pediatric population at large restricts the generalizability of such studies. This investigation evaluates racial and ethnic minority representation among participants enrolled in BPCA-sponsored studies. This study revealed no evidence of racial and ethnic bias in enrollment for pediatric studies conducted with funding from BPCA, fulfilling the legislation’s expectation to ensure adequate representation of all children.