Skip to main content
An official website of the United States government
Government Funding Lapse
Because of a lapse in government funding, the information on this website may not be up to date, transactions submitted via the website may not be processed, and the agency may not be able to respond to inquiries until appropriations are enacted.

The NIH Clinical Center (the research hospital of NIH) is open. For more details about its operating status, please visit cc.nih.gov.

Updates regarding government operating status and resumption of normal operations can be found at opm.gov.

Email

Transition of Pediatric Brain Tumor Trials to Pediatric Early Phase Clinical Trials Network (PEP-CTN)

NCI is in the process of expanding its Pediatric Early Phase Clinical Trials Network (PEP-CTN) by transitioning early phase pediatric brain tumor trials previously conducted through NCI’s Pediatric Brain Tumor Consortium (PBTC) to PEP-CTN.

The goal of this transition is to improve NCI’s approach to clinical trial development and to expand access to pediatric brain tumor trials to more trial sites. PBTC includes 16 sites, and PEP-CTN has the capacity to expand the availability of these trials to its 42 sites, as well as to any PBTC sites that are not already PEP-CTN members.

This is not a cost-cutting measure; rather, the intent is to better align resources in a way that sustains and strengthens pediatric brain tumor research over the long term. Pediatric brain tumor research remains a high priority for NCI.

NCI understands that researchers, physicians, patients, and families have questions about how this process will work, and whether it will interfere with patients’ access to clinical trials. The Q&A below begins to address these questions, and NCI looks forward to sharing updates as these efforts proceed.

What is the PBTC?

NCI has supported the PBTC for over 25 years. PBTC includes 16 sites that have conducted phase I and II clinical trials of novel treatment approaches in children with brain tumors. PBTC has transitioned promising agents that it studied to the Children's Oncology Group (COG), which represents 220 children’s hospitals, to confirm findings in later-phase trials.

What is PEP-CTN?

The PEP-CTN, established in 2019 to continue the work of the COG phase 1 consortium, is a group of 42 leading children’s hospitals that design and conduct early-phase clinical trials for childhood cancers. Its goal is to test new therapies for safety and effectiveness, and to help move the most promising treatments into later-phase trials through COG.

Why is this transition happening now?

The pediatric cancer research and clinical trial landscape has evolved significantly over the past two decades:

  • The regulatory environment and the ways pharmaceutical companies engage with research institutions have changed considerably.
  • Clinical trial infrastructure has improved through major investments in NCI’s National Clinical Trials Network (NCTN) — which includes COG — along with advancements in trial data management.
  • Patient enrollment in PBTC trials has slowed in recent years, partly due to external factors such as sponsor withdrawal after studies began.

NCI carefully considered how best to support early-phase trials for children with brain tumors going forward. The Institute determined that transitioning these studies to PEP-CTN will expand trial availability while involving pediatric neuro-oncologists that are currently members of PBTC as well as those from the COG CNS Tumor Committee.

This transition is eased by the fact that most PBTC institutions are already part of PEP-CTN, which is already running pediatric brain tumor trials. PEP-CTN has the capacity to expand access to its 42 sites and to complete ongoing PBTC trials where appropriate.

The timing reflects that PBTC was approaching the end of its five-year program cycle. Rather than planning another cycle under the same structure, NCI concluded the most effective approach was to wind down PBTC activities and transition its work to PEP-CTN.

What is the plan for ongoing PBTC studies?

PBTC currently has five active clinical trials. NCI, together with PBTC and PEP-CTN leadership, is reviewing current PBTC studies, as well as continuing to support PEP-CTN brain tumor clinical trials that are already underway. Most PBTC institutions are already a part of PEP-CTN, and those that are not will be able to participate in PEP-CTN trials to complete PBTC studies.

  • Participants currently on PBTC trials that are closed to new enrollment will continue to receive protocol-specified treatment. NCI will work with the PBTC and PEP-CTN to address any issues that arise related to patients’ ability to continue protocol treatment.
  • PBTC trials for which accrual has been too slow to allow successful completion will be closed. Patients already on those studies will continue to receive protocol-specified treatment and follow-up.
  • For PBTC studies with strong scientific value and adequate accrual, NCI will work with PBTC and PEP-CTN to identify ways to complete accrual, as appropriate.
  • Children not yet enrolled on PBTC trials will have the opportunity to enroll on trials through other consortia. Several trials are currently available through the PEP-CTN and NCI’s Pediatric Oncology Branch at the NIH Clinical Center, as well as through the Pediatric Neuro-Oncology Consortia (PNOC) and other consortia that are independent from NCI.
  • Two PEP-CTN trials for which children with brain tumors are eligible are currently open to enrollment: One to test the addition of an anti-cancer drug (AZD1390) during radiation therapy for children with newly diagnosed high grade glioma, diffuse midline glioma, or diffuse intrinsic pontine glioma (NCT06894979); and a second trial for children with relapsed or refractory solid tumors, including CNS tumors and Lymphoma (NCT04870944), which is currently enrolling a cohort of patients with diffuse intrinsic pontine glioma (DIPG).

Is this a cost-cutting measure?

Pediatric brain tumor research remains a high priority for NCI. The decision to transition brain tumor clinical trials from the PBTC to PEP-CTN is not about cost-cutting. The intent is to better align resources in a way that sustains and strengthens pediatric brain tumor research over the long term. A more efficient infrastructure will better support the research itself.

How will future trials be developed? 

Moving forward, pediatric brain tumor physician scientists will design and conduct early-phase trials through PEP-CTN, which will be enhanced to accommodate the increased number of clinical trials for children with brain tumors. Proposals for new clinical trials for children with brain tumors will be able to be submitted for review by the PEP-CTN Agent Prioritization Committee (APC), and those studies that are approved by the APC will move forward for protocol development through the PEP-CTN. Later phase confirmatory trials will continue to be conducted through COG.

NCI will continue to work with both PBTC and PEP-CTN investigators to move childhood brain tumor research forward. NCI will support close collaboration between PEP-CTN and COG Central Nervous System (CNS) Tumor Committee to develop new trials and expand trial opportunities for children with brain tumors.

What happens to the data and protocols developed under PBTC? 

NCI will work with PBTC leadership and the Childhood Cancer Data Initiative to assess which datasets are of significant value and how they can be made broadly accessible for future research.

  • Posted:

If you would like to reproduce some or all of this content, see Reuse of NCI Information for guidance about copyright and permissions. In the case of permitted digital reproduction, please credit the National Cancer Institute as the source and link to the original NCI product using the original product's title; e.g., “Transition of Pediatric Brain Tumor Trials to Pediatric Early Phase Clinical Trials Network (PEP-CTN) was originally published by the National Cancer Institute.”

Email