NCT06357377

A Study of the Safety, Dosing, and Delivery of NEO100 in Patients With Pediatric Brain Tumors

An Open Label, Phase 1b Safety, Dose-finding, Brain Tumor Delivery, and Pharmacokinetics Study of Intranasal NEO100 in Patients With Pediatric-type Select Brain Tumors

• Status: Not Yet Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 12 (estimated)
• Sponsor: Neonc Technologies, Inc. · Industry
• Sex: All
• Age: 5 Years – 18 Years
• Healthy volunteers: Not accepted

Summary

This is an open label, Phase 1b safety, dose-finding, brain tumor delivery, and pharmacokinetics study of intranasal NEO100 in patients with pediatric-type diffuse high grade gliomas. Patients will receive IN NEO100 that will follow a dose titration design, followed by a standard dose escalation design to establish safety. Brain tumor delivery of NEO100 will be confirmed in each disease sub-type by surgical resection/needle biopsy only if clinically indicated and scheduled for clinical purposes and testing with residual tissue for NEO100 and the major metabolite of NEO100 (Perillic Acid).

Detailed description

NEO100 drug product is a non-sterile solution for intranasal administration. A feature of the clinical trials of NEO100 is to investigate the efficacy of this drug when it is delivered intranasally. Based on the general body of data on intranasal drug delivery, as well as the promising results from clinical studies in which perillyl alcohol was delivered intranasally, NeOnc expects that intranasal administration of NEO100 will allow rapid and more selective delivery of therapeutic levels of the drug to the brain, while minimizing systemic toxicity and first pass metabolism. The study is a dose titration 1+1 design with a modified Fibonacci dose escalation, followed by a expansion design, used to determine the maximum tolerated dose and to select a recommended Phase 2 dose (RP2D). The starting dose of 192 mg mg/dose administered QID, on a 28-day cycle, is one dose below the safe dose administered to adults in a Phase 1/2a study. Dosing will increase using a modified Fibonacci dose escalation. The daily dose will start at 768 mg/day, and rise to 1,152 mg/day, which is also the safe dose administered to adults in a Phase 1/2a study. The study is open to patient with Pediatric-type high grade glioma: Newly diagnosed and recurrent Diffuse Midline Glioma, H3 K27-altered; Diffuse hemispheric glioma, H3 G34-mutant, grade III and IV diffuse pediatric-type HGG, H3-wildtype and IDH-wildtype (including spinal cord tumors); Recurrent malignant tumors involving the brainstem or posterior fossa (choroid plexus carcinoma, CNS embryonal tumors, and pineoblastoma).

Conditions

• Pediatric Tumor of CNS
• Pediatric Tumor of Brain
• Diffuse Midline Glioma, H3 K27M-Mutant
• Pediatric Tumor of Brain Stem
• Pineocytoma
• Choroid Plexus Carcinoma, Childhood
• Spinal Cord Tumor
• High Grade Glioma

Interventions

Type	Name	Description
DRUG	NEO100	NEO100 is a purified form (\>98.5%) of the naturally occurring monoterpene perillyl alcohol. NEO100 drug product is a non-sterile solution for intranasal administration. It is compounded as a 10% solution in a 50:50 mixture of ethanol:glycerol.

Timeline

• Start date: 2026-01-01
• Primary completion: 2026-10-01
• Completion: 2026-10-01
• First posted: 2024-04-10
• Last updated: 2026-01-14

Regulatory

• FDA-regulated drug study