# Children's Oncology Group Pediatric Early Phase Clinical Trial Network

> **NIH NIH UM1** · PUBLIC HEALTH INSTITUTE · 2024 · $600,000

## Abstract

PEP-CTN Project Summary/Abstract:
The mission of the Pediatric Early Phase Clinical Trials Network (PEP-CTN) is to identify and develop
effective new agents for children and adolescents with cancer, through rational and efficient clinical and
laboratory research. PEP-CTN clinical trials will incorporate correlative genomics, biology, pharmacology, and
imaging studies to further the understanding of the disposition and action of new agents introduced into the
treatment of children with cancer. Annually, the PEP-CTN will enroll approximately 120 children and
adolescents with cancer onto clinical trials of novel anticancer agents at the 21 core-member sites and 21
non-core member sites throughout the US, Canada, and Australia. The network institutions are selected
through a peer review process, and serve as a national and international model for new agent development in
pediatric oncology. The PEP-CTN leverages the database infrastructure and resources of the parent
Children’s Oncology Group (COG) while maintaining its own administrative and operational infrastructure to
ensure rapid development, implementation, and reporting of specialized and complex early phase clinical
trials. The PEP-CTN has expertise and resources for the conduct of translational biology, pharmacokinetic,
and pharmacogenetic studies, and utilizes state-of-the-art informatics systems to facilitate the transfer of
response and correlative imaging studies for central review and analyses.
The PEP-CTN's primary specific aims are: 1) To safely and efficiently introduce novel anticancer agents into
the pediatric setting through the conduct of early phase clinical trials; 2) To expeditiously obtain preliminary
efficacy signals through use of phase 2 expansion cohorts and pilot studies in order to inform tumor specific
trials that will be conducted across COG sites; 3) To perform genomic analyses, including single gene studies
or gene panels, to identify appropriate patients for early phase studies of targeted agents; 4) To identify
associations of tumor characteristics with response to new agents using genomic analyses such as whole
exome sequencing and RNA sequencing; 5) To incorporate pharmacologic and biologic endpoints, including
circulating tumor DNA, other translational laboratory studies, and imaging modalities, into early phase studies,
in order to enhance our understanding of the new agents and their effect on tumors Development of targeted
therapy for childhood cancer is a high priority as it offers the prospect of more efficacious and less toxic
therapeutics.
Project Summary/Abstract Page 21

## Key facts

- **NIH application ID:** 11083363
- **Project number:** 3UM1CA228823-05S1
- **Recipient organization:** PUBLIC HEALTH INSTITUTE
- **Principal Investigator:** Brenda J. Weigel
- **Activity code:** UM1 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $600,000
- **Award type:** 3
- **Project period:** 2024-05-03 → 2024-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/11083363

## Citation

> US National Institutes of Health, RePORTER application 11083363, Children's Oncology Group Pediatric Early Phase Clinical Trial Network (3UM1CA228823-05S1). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/11083363. Licensed CC0.

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