Originally opened in 2021, PNOC019, a phase 1 clinical trial, was designed to evaluate the immunotherapy drugs Nivolumab and Ipilimumab in treating children and young adults with recurrent or progressive high-grade glioma (HGG).

This study aims to assess how a tumour’s genetic activity related to cell growth changes after treatment with nivolumab, an immune therapy, in children and young adults with recurrent or worsening high-grade gliomas (HGG).

Researchers will compare these changes to samples from past patients who did not receive this treatment. The study will also evaluate how safe and tolerable nivolumab is when given before and after surgery, and track patient survival and disease progression over six and twelve months to understand the treatment’s potential benefits.

As is common in clinical trials, these drugs were initially supplied by a pharmaceutical company. However, in late 2022, the company withdrew its support, halting the supply of these critical study drugs. For patients already enrolled in the trial, the company agreed to continue providing treatment until completion, but no new patients could be recruited—putting the future of this vital study in jeopardy.

In December 2023, PNOC leadership in the US revised the study’s protocol, allowing recruitment to resume under a new structure. The trial would now continue as a single-arm study focused solely on Nivolumab, requiring the drug to be purchased commercially at an estimated cost of $2,500 per patient per cycle. Without external funding, this cost would have made participation impossible for many families.

Thanks to CBCF’s commitment, patient treatment in Australia—including the cost of Nivolumab, per-patient reimbursements, study monitoring, and coordination—is fully funded. Without this vital funding, the PNOC019 trial would have remained stalled, preventing Australian children from accessing this promising immunotherapy.

With CBCF’s support, recruitment can now continue. The revised protocol (version 7) was approved by the Australian HREC in September 2024. Study sites are preparing to reopen, with the Children’s Hospital at Westmead and Perth Children’s Hospital having completed training in December 2024. Four additional Australian sites—Queensland Children’s Hospital, Monash Children’s Hospital, Royal Children’s Hospital, and Women’s and Children’s Hospital—are also gearing up to enrol patients soon.