Phase I Pilot Study to Evaluate the Anti-glioblastoma Effect of S-Gboxin in Standard Treatment of Glioblastoma/Diffuse Midline Glioma and Response to Treatment (Regardless of Mutation Status)

The objective of this pilot study is to evaluate the efficacy of adding S-Gboxin to standard RT/TMZ treatment protocols in patients with glioblastoma multiforme (GBM) or midline glioma (DMG), regardless of their mutation status

Study Overview

• Status: Suspended
• Conditions: Glioblastoma Multiforme; Gliosarcoma; GBM; Diffuse Midline Glioma, H3 K27M-Mutant; Giant Cell Glioblastoma
• Intervention / Treatment: Biological: S-Gboxin

Detailed Description

This is a pilot study to evaluate the efficacy of adding the mitochondrial oxidative phosphorylation inhibitor S-Gboxin (OXPHOS) to standard treatment for glioblastoma multiforme or glioma.

Subjects will start oral S-gboxin orally twice daily and will evaluate the patients and vital status if they tolerate this dose at 3, 7and 15 days after initiation.

Patients will undergo MRI scans before treatment and then at multiple time points during their participation in this Clinical Trial to monitor early tumor response to treatment ( MRI dates will be scheduled by the clinical trial coordinator)

Peripheral blood samples will be collected at baseline, days 1-7-15-28-36-72 to compare biological indicators of treatment response. In some cases, circulating tumor cells (CTCs) will be monitored by real-time qRT-PCR to correlate with clinical data.

Patients will also be assessed for the side effects they experience. Disease-free and overall survival outcomes will be recorded.

Patients will also have their depressive symptoms, quality of life, and neurocognitive function assessed at multiple time points during and after treatment.

After completion of the study, patients will be followed up at 3 months.

• Study Type: Interventional
• Enrollment (Estimated): 10
• Phase: Phase 1

Contacts and Locations

Study Locations

• Georgia
  • Tbilisi, Georgia, 0198
    • Tbilisi Cancer Centre
• Kazakhstan
  • Almaty, Kazakhstan, 490 078
    • Kazakh Institute of Oncology and Radiology
• Ukraine
  • Kyiv, Ukraine, 33/43
    • National Cancer Institute

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• confirmed diagnosis of Glioblastoma according to RANO criteria by contrast-enhanced MRI or PET-CT (including diffuse midline glioma, gliosarcoma or giant cell glioblastoma), relapse or disease progression
• patient is able to understand and give consent to participate in the study
• Karnofsky performance score ≥ 60
• women of childbearing potential must have a negative pregnancy test result no later than 7 days before registration

Exclusion Criteria:

• pregnant
• known allergies
• active treatment on annotner clinical trial
• inability to complete with protocol or studio procedures.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: Patients will be offered 4 courses of Gboxin during (or without) standard treatment.; Patients will be offered 4 courses of S-Gboxin during standard treatment. Each course (18 days) consists of twice daily intake of S-Gboxin for 14 days and four days of rest. Each dose of S-Gboxin is packed in a gelatin capsule with a bandage and applied markings.

Biological: S-Gboxin; Gboxin specifically inhibits the growth of human glioblastoma cells but not normal cells. Gboxin rapidly and irreversibly impairs oxygen consumption in glioblastoma cells. Its positive charge for binding to mitochondrial oxidative phosphorylation complexes is dependent on the proton gradient of the inner mitochondrial membrane, and it inhibits F 0 F 1 ATP synthase activity in tumor cells. S-Gboxin crosses the blood-brain barrier at therapeutically effective concentrations.; Other Names: oxidative phosphorylation inhibitor; OXPHOS inhibitor; suppression of oxidative phosphorylation

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Brain tumor metabolism as measured by PET and MRI	The standardized uptake value (SUV) of HGG will be measured. SUV is a standard PET measurement. MRI with contrast will evaluate the change in tumor size before and after therapy S-Gboxin	Up to 6 month

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall survival (OS)	Kaplan-Meier survival estimates for OS will be presented with using Greenwood's formula at 12 months.	Up to 12 month
Incidence of adverse events graded according to the Common Toxicity Criteria for Adverse Events (CTCAE) version (v)4.03	The data will be organized by major organ system and MedDRA severity after completion of the Clinical Study.	Up to 1 month after last dose of S-Gboxin

Collaborators and Investigators

• Sponsor: Petrov, Andrey
• Collaborators: Lytvin,Ruslan

Publications and helpful links

Helpful Links

• Gboxin is an oxidative phosphorylation inhibitor that targets glioblastoma
• Cancer cell-mitochondria hybrid membrane coated Gboxin loaded nanomedicines for glioblastoma treatment
• Nanomedicine and Immunotherapy for recurrent glioblastoma

Study record dates

Study Major Dates

• Study Start (Actual): May 20, 2026
• Primary Completion (Estimated): October 10, 2027
• Study Completion (Estimated): December 10, 2027

Study Registration Dates

• First Submitted: January 26, 2025
• First Submitted That Met QC Criteria: January 31, 2025
• First Posted (Actual): February 4, 2025

Study Record Updates

• Last Update Posted (Actual): June 3, 2026
• Last Update Submitted That Met QC Criteria: May 30, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms; Neoplasms by Histologic Type; Neoplasms, Glandular and Epithelial; Astrocytoma; Glioma; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms, Nerve Tissue; Glioblastoma; Gliosarcoma
• Other Study ID Numbers: STM 0125-2G

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Sharing Time Frame: documents will be available 3 months after completion of the research
• IPD Sharing Access Criteria: free access
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; ICF

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No