Phase 2 Trial of Single Intratumoral DNX-2401 Treatment for Recurrent or Refractory High Grade Brain Tumors in Children and Young Adults Aged 1 to 25

What this Trial Is About

Researchers are evaluating whether a single injection of DNX-2401, a genetically modified virus, can treat recurrent or treatment-resistant high-grade brain tumors in children and young adults aged 1 to 25 years. These tumors include high-grade gliomas, embryonal CNS tumors, and ependymomas, which generally have poor outcomes and limited treatment options after recurrence. The study also aims to assess the safety and tolerability of DNX-2401 delivered directly into the tumor. Participants will undergo a surgical biopsy of the tumor followed by a single controlled infusion of DNX-2401 into the brain using a specialized cannula with neuronavigation guidance over about one hour. The study uses a two-stage design to evaluate treatment response, with plans to expand if initial responses are promising. The treatment dose is fixed, and no other chemotherapy or immunotherapy is allowed shortly before administration. During the year following treatment, participants will have regular clinic visits for neurological exams, quality of life assessments, and MRI scans to monitor tumor response and side effects. Blood, cerebrospinal fluid, and microbiome samples will be collected to understand immune responses and viral behavior. The main outcome measured is the overall tumor response rate up to 52 weeks after treatment, along with survival rates and safety events monitored up to 12 months.

Intratumoral DNX-2401 for High Grade Pediatric Brain Tumors