Trials / Recruiting

RecruitingNCT05839379

Targeted Pediatric High-Grade Glioma Therapy

Molecularly-Guided Phase II Umbrella Trial for Children, Adolescents, and Young Adults Newly Diagnosed With High-Grade Glioma, Including Diffuse Intrinsic Pontine Glioma

Status: Recruiting
Phase: —
Study type: Observational
Enrollment: 350 (estimated)

Sponsor: Nationwide Children's Hospital · Academic / Other
Sex: All
Age: 12 Months – 39 Years
Healthy volunteers: Not accepted

Summary

The goal of this study is to perform genetic sequencing on brain tumors from children, adolescents, and young adult patients who have been newly diagnosed with a high-grade glioma. This molecular profiling will decide if patients are eligible to participate in a subsequent treatment-based clinical trial based on the genetic alterations identified in their tumor.

Detailed description

A novel, molecularly-guided, multi-arm phase umbrella II trial is proposed in children, adolescents, and young adults with newly diagnosed HGG, including DIPG, in which we will (1) conduct comprehensive molecular screening of tumor tissue using a multi-omic approach (WES/WGS, gene fusion panels/RNASeq, DNA methylation microarray) across international CONNECT genomics cores with rapid return of clinical results, (2) stratify patients to biologically-targeted treatment arms, based on the tumor molecular profile and histopathology, and (3) perform longitudinal evaluation of peripheral blood, cerebrospinal fluid (CSF), and/or tumor tissue as well as advanced neuro-imaging to determine genomic, immune, and radiologic biomarkers predictive of response, recurrence, resistance, and toxicity. Based on results of the above tumor molecular profiling and pathology-based confirmation of HGG diagnosis, eligible patients will be assigned to one of several biologically guided treatment arms on a phase II trial. Approximately 300-350 patients will be enrolled on the screening protocol through which biospecimens (paired tumor DNA/RNA and normal comparator samples) will undergo extensive molecular profiling and/or there will be comprehensive central molecular and pathology review of previously obtained molecular results to assess eligibility to any of the therapeutic subprotocols of the phase II study.

Conditions

Timeline

Start date: 2024-08-02
Primary completion: 2029-08-28
Completion: 2034-08-28
First posted: 2023-05-03
Last updated: 2026-03-31

Locations

21 sites across 7 countries: United States, Australia, Canada, Germany, Netherlands, New Zealand, United Kingdom

Source: ClinicalTrials.gov record NCT05839379. Inclusion in this directory is not an endorsement.