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Updated: Jun 17, 2025
A Protocol for Explant Cultures of IDH1-mutant Diffuse Low-grade Gliomas
Published on: May 9, 2025
A perioperative study of Safusidenib in patients with IDH1-mutated glioma
Sarah A Cain1, Monique Topp2, Mark Rosenthal2
1Department of Neurosurgery, Royal Melbourne Hospital, Parkville, 3052, Australia.
Abstract:
This is a single arm, open label perioperative trial to assess the feasibility, pharmacokinetics and pharmacodynamics of treatment with safusidenib following biopsy, and prior to surgical resection in patients with IDH1 mutated glioma who have not received radiation therapy or chemotherapy. Fifteen participants will receive treatment in two parts. First, biopsy followed by one cycle (28 days) of safusidenib, an orally available, small molecular inhibitor of mutated IDH1, then maximal safe resection of the tumor (Part A). Second, after recovery from surgery, safusidenib until disease progression or unacceptable toxicity (Part B). This research will enable objective measurement of biological activity of safusidenib in patients with IDH1 mutated glioma. Anti-tumor activity will be assessed by progression free survival and time to next intervention.Clinical Trial Registration: NCT05577416 (ClinicalTrials.gov).
Insights
This study evaluates safusidenib for IDH1 mutated glioma patients. It assesses the drug
Area of Science:
- Neuro-oncology
- Pharmacology
- Clinical Trials
Background:
- Gliomas with IDH1 mutations are a distinct molecular subtype.
- Targeted therapies for IDH1-mutated gliomas are under investigation.
- Safusidenib is an orally available small molecule inhibitor of mutated IDH1.
Purpose of the Study:
- To assess the feasibility, pharmacokinetics, and pharmacodynamics of safusidenib.
- To evaluate the anti-tumor activity of safusidenib in patients with IDH1-mutated glioma.
- To measure the biological activity of safusidenib in a perioperative setting.
Main Methods:
- Single-arm, open-label, perioperative clinical trial.
- Treatment involves biopsy, safusidenib administration, surgical resection, and further safusidenib treatment.
- Pharmacokinetic and pharmacodynamic analyses will be performed.
Main Results:
- Feasibility, pharmacokinetics, and pharmacodynamics of safusidenib are being assessed.
- Anti-tumor activity will be evaluated by progression-free survival and time to next intervention.
- Objective measurement of biological activity is enabled by this research.
Conclusions:
- Safusidenib is being investigated as a targeted therapy for IDH1-mutated glioma.
- The study aims to provide objective measures of safusidenib's biological activity.
- Results will inform future clinical development of safusidenib.
