MT-125 for Glioblastoma

(NOVA-GBM Trial)

Not yet recruiting at 2 trial locations
Age: 18+
Sex: Any
Trial Phase: Phase 1 & 2
Sponsor: Myosin Therapeutics Inc.
Must be taking: Steroids
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to test a new treatment, MT-125, for glioblastoma, a type of brain cancer. Participants will receive MT-125 alongside standard treatments to assess its safety and effectiveness. The trial will explore different doses to determine the optimal one. Individuals recently diagnosed with a specific type of glioblastoma and stable on their medication might be suitable candidates. As a Phase 1 trial, this research focuses on understanding how MT-125 works in people, offering participants the opportunity to be among the first to receive this new treatment.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. However, you must be on a stable dose of steroids for at least 2 weeks before enrolling. It's best to discuss your specific medications with the trial team.

Is there any evidence suggesting that MT-125 is likely to be safe for humans?

Research has shown that MT-125 is generally well-tolerated in early studies, with a strong safety record indicating few serious side effects in initial tests. MT-125 is designed to effectively reach the brain, which is crucial for treating brain conditions like glioblastoma.

Other studies have focused on determining the optimal dose that works without causing excessive side effects. So far, results are promising, suggesting that MT-125 can be safely administered to patients with careful monitoring. However, further research is necessary to fully understand its safety in humans.12345

Why are researchers excited about this trial's treatment?

Researchers are excited about MT-125 for treating glioblastoma because it offers a potentially new way to enhance the effects of radiation therapy (RT) and temozolomide (TMZ), the current standard treatments. Unlike traditional options, MT-125 works by possibly targeting specific cancer cell pathways that make tumor cells more vulnerable to RT and TMZ. This could lead to better outcomes for patients by making cancer cells easier to destroy and potentially improving survival rates. Additionally, the flexible dosing of MT-125, tested at various levels, might help personalize treatment plans to maximize effectiveness while minimizing side effects.

What evidence suggests that MT-125 might be an effective treatment for glioblastoma?

Research has shown that MT-125 could be promising for treating glioblastoma, a type of brain cancer. Studies have found that MT-125 can stop glioblastoma cells from spreading and help animals live longer. This treatment effectively reaches the brain and is well-tolerated in these studies. The FDA granted MT-125 "fast track" status, indicating its potential as an important new treatment for this aggressive cancer. Overall, early evidence suggests that MT-125 might help extend the lives of people with glioblastoma. Participants in this trial will receive different dosages of MT-125 to evaluate its effectiveness and safety.13567

Are You a Good Fit for This Trial?

This trial is for adults (18+) with newly diagnosed glioblastoma that is IDH wildtype and MGMT methylated. Participants must be in reasonably good physical condition, have adequate blood counts, liver and kidney function, and stable clotting tests.

Inclusion Criteria

7. Serum eGFR ≥60 ml/min
I am 18 years old or older.
2. Absolute neutrophil count (ANC) ≥1500/mm3
See 7 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive MT-125 with chemoradiation for 6 weeks, administered 5 consecutive days a week with 2 days off

6 weeks
Weekly visits for treatment administration

Dose Escalation

Evaluation of up to four dose levels using a Bayesian Optimal Interval (BOIN) design to determine the maximum tolerated dose (MTD)

6 weeks

Randomized Expansion

Participants are enrolled into a randomized, parallel dose expansion cohort to receive either MTD or one dose lower than MTD

6 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment, including overall response rate (ORR), progression-free survival (PFS6), and overall survival (OS)

Up to 1 year

What Are the Treatments Tested in This Trial?

Interventions

  • MT-125

Trial Overview

The study is testing different doses of a new drug called MT-125 given along with standard chemoradiation (radiotherapy plus temozolomide) to see if it’s safe and tolerable. The trial has two phases: first finding the best dose, then comparing selected doses.

How Is the Trial Designed?

5

Treatment groups

Experimental Treatment

Group I: MT-125 at MTD and one dose lower than MTDExperimental Treatment1 Intervention
Group II: MT-125 at 83.5 mgExperimental Treatment1 Intervention
Group III: MT-125 at 50 mgExperimental Treatment1 Intervention
Group IV: MT-125 at 25 mgExperimental Treatment1 Intervention
Group V: MT-125 at 100 mgExperimental Treatment1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

Myosin Therapeutics Inc.

Lead Sponsor

Mayo Clinic

Collaborator

Trials
3,427
Recruited
3,221,000+

Citations

MT-125 inhibits non-muscle myosin IIA and IIB and ...

MT-125 has high brain penetrance and an excellent safety profile, blocks GBM invasion and cytokinesis, and prolongs survival in murine GBM ...

A Phase 1/2 Study of the Safety and Tolerability of MT-125 ...

MT-125 inhibits non-muscle myosin IIA and IIB and prolongs survival in glioblastoma. Cell. 2025 Aug 21;188(17):4622-4639.e19. doi: 10.1016/j.cell.2025.05 ...

MT-125: A glimpse of hope for glioblastoma patients

MT-125 with its remarkable brain retention and in vivo tolerance impedes GBM invasion and extends the lifespan of murine GBM models through ...

FDA Awards Fast Track Designation to MT-125 for ...

MT-125 has shown strong preclinical activity and tolerability, supporting its potential to introduce a novel therapeutic approach in aggressive ...

FDA Grants Fast Track Designation to MT-125 in ...

The evaluation of MT-125's safety and preliminary activity in glioblastoma is underway in a phase 1/2 trial.

A Phase 1/2 Study of the Safety and Tolerability of MT-125 ...

The purpose of the study is to determine the recommended dose and further understand the safety of MT-125 in participants who have been diagnosed with ...

A Phase 1/2 Study of the Safety and Tolerability of MT-125 ...

The purpose of the study is to determine the recommended dose and further understand the safety of MT-125 in participants who have been ...