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)

NCT ID: NCT06806228

Last Updated: 2025-11-19

Basic Information

• Recruitment Status: SUSPENDED
• Clinical Phase: PHASE1
• Total Enrollment: 10 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2026-03-20
• Study Completion Date: 2027-12-10

Brief Summary

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

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.

Conditions

Glioblastoma Multiforme; Diffuse Midline Glioma, H3 K27M-Mutant; Gliosarcoma; Giant Cell Glioblastoma; GBM

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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.

Group Type: EXPERIMENTAL

S-Gboxin

Intervention Type: BIOLOGICAL

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.

Interventions

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.

Intervention Type: BIOLOGICAL

Other Intervention Names

oxidative phosphorylation inhibitor; OXPHOS inhibitor; suppression of oxidative phosphorylation

Eligibility Criteria

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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 99 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Lytvin,Ruslan

UNKNOWN

Sponsor Role: collaborator

Petrov, Andrey

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Tbilisi Cancer Centre

Tbilisi, , Georgia

Kazakh Institute of Oncology and Radiology

Almaty, , Kazakhstan

National Cancer Institute

Kyiv, , Ukraine

Countries

Georgia; Kazakhstan; Ukraine

Related Links

https://pmc.ncbi.nlm.nih.gov/articles/PMC6655586/

Gboxin is an oxidative phosphorylation inhibitor that targets glioblastoma

https://www.nature.com/articles/s41467-023-40280-3

Cancer cell-mitochondria hybrid membrane coated Gboxin loaded nanomedicines for glioblastoma treatment

https://braintumourresearch.org/blogs/research-campaigning-news/nanomedicine-and-immunotherapy-for-recurrent-glioblastoma?srsltid=AfmBOooU1XGjeuMxSBaBzWn4EGMFvsiNIktJRm4p7Ey8XVnJTor2oYHS

Nanomedicine and Immunotherapy for recurrent glioblastoma

Other Identifiers

STM 0125-2G

Identifier Type: -

Identifier Source: org_study_id