Evaluation of ex vivo drug combination optimization platform in recurrent high grade astrocytic glioma: An interventional, non-randomized, open-label trial protocol

PLoS One. 2024 Jul 26;19(7):e0307818. doi: 10.1371/journal.pone.0307818. eCollection 2024.

Abstract

Introduction: High grade astrocytic glioma (HGG) is a lethal solid malignancy with high recurrence rates and limited survival. While several cytotoxic agents have demonstrated efficacy against HGG, drug sensitivity testing platforms to aid in therapy selection are lacking. Patient-derived organoids (PDOs) have been shown to faithfully preserve the biological characteristics of several cancer types including HGG, and coupled with the experimental-analytical hybrid platform Quadratic Phenotypic Optimization Platform (QPOP) which evaluates therapeutic sensitivity at a patient-specific level, may aid as a tool for personalized medical decisions to improve treatment outcomes for HGG patients.

Methods: This is an interventional, non-randomized, open-label study, which aims to enroll 10 patients who will receive QPOP-guided chemotherapy at the time of first HGG recurrence following progression on standard first-line therapy. At the initial presentation of HGG, tumor will be harvested for primary PDO generation during the first biopsy/surgery. At the point of tumor recurrence, patients will be enrolled onto the main study to receive systemic therapy as second-line treatment. Subjects who undergo surgery at the time of recurrence will have a second harvest of tissue for PDO generation. Established PDOs will be subject to QPOP analyses to determine their therapeutic sensitivities to specific panels of drugs. A QPOP-guided treatment selection algorithm will then be used to select the most appropriate drug combination. The primary endpoint of the study is six-month progression-free survival. The secondary endpoints include twelve-month overall survival, RANO criteria and toxicities. In our radiological biomarker sub-study, we plan to evaluate novel radiopharmaceutical-based neuroimaging in determining blood-brain barrier permeability and to assess in vivo drug effects on tumor vasculature over time.

Trial registration: This trial was registered on 8th September 2022 with ClinicalTrials.gov Identifier: NCT05532397.

Publication types

  • Clinical Trial Protocol

MeSH terms

  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use
  • Astrocytoma / diagnostic imaging
  • Astrocytoma / drug therapy
  • Astrocytoma / pathology
  • Brain Neoplasms* / diagnostic imaging
  • Brain Neoplasms* / drug therapy
  • Brain Neoplasms* / pathology
  • Clinical Trials as Topic
  • Humans
  • Neoplasm Grading
  • Neoplasm Recurrence, Local* / drug therapy
  • Neoplasm Recurrence, Local* / pathology
  • Organoids / diagnostic imaging
  • Organoids / drug effects
  • Organoids / pathology

Associated data

  • ClinicalTrials.gov/NCT05532397

Grants and funding

ALAW received funding from the National University Health System (NUHS) Seed Fund (NUHSRO/2021/052/RO5+6/Seed-Mar/01) and TTB obtained funding from the National University of Singapore Institute for Digital Medicine (WisDM) Program Seed Fund (WisDM/Seed/002/2021). The funders had no role in study design, data collection and analysis, decision to publish, or preparation of the manuscript.