What is MT-125 studying for glioblastoma?

MT-125 is being studied to understand its safety, dosing, and how it works when combined with radiotherapy in adults newly diagnosed with glioblastoma. The study aims to find the best dose and assess side effects because this is the first time MT-125 is tested in humans. This research is sponsored by Myosin Therapeutics Inc. and focuses on patients unlikely to respond to standard chemotherapy.

Who may be eligible for this glioblastoma clinical trial?

Adults 18 years and older with a new diagnosis of IDH wild type and MGMT unmethylated glioblastoma may be eligible. Participants should have a good performance status and meet certain blood and organ function requirements. Both men and women can join, but people of childbearing potential must use contraception. Please review eligibility criteria for more details.

Is there any cost to participate in this glioblastoma clinical trial?

Study-related care, including the study drug MT-125, radiotherapy, and all trial visits, is provided at no cost. However, personal travel and other expenses may vary and are not guaranteed to be covered. Participants should plan for these potential costs independently.

Actively Recruiting

Phase 1

Phase 2

Age: 18Years +

All Genders

ID07185880

Phase 12 Study of MT-125 with Radiation Therapy in Adults Newly Diagnosed with Glioblastoma

Led by Myosin Therapeutics Inc. · Updated on 2026-04-14

Participants Needed

Research Sites

274 weeks

Total Duration

AI-Summary

What this Trial Is About

Researchers are evaluating the safety, tolerability, and dosing of a new investigational drug called MT-125 in adults newly diagnosed with glioblastoma, a primary brain tumor. MT-125 targets specific proteins involved in tumor cell growth and is being studied in combination with standard radiotherapy. The study focuses on patients with a particular tumor type IDH wild type, MGMT unmethylated who typically do not respond well to current therapies, addressing an urgent need for new treatment options. Participants will receive MT-125 alongside their radiotherapy. The study begins with a dose escalation phase where small groups receive increasing doses of MT-125 for 5 days a week with 2 days off over 6 weeks, to find the highest dose that can be safely tolerated. After establishing this dose, additional participants will be randomly assigned to receive either the highest dose or one dose level lower, also combined with radiotherapy, to further assess safety and preliminary responses. During the study, participants will be monitored closely for any side effects and dose-limiting toxicities over the 6-week treatment period. Researchers will measure drug levels in the blood at several time points and assess overall safety, tolerability, and preliminary efficacy. Participants will complete questionnaires and have regular laboratory tests, heart function assessments, and pregnancy tests as applicable. The total participation time includes the 6 weeks of treatment with follow-up monitoring to ensure safety and collect data on response and survival outcomes.

CONDITIONS

Brief Title

A Phase 1/2 Study of the Safety and Tolerability of MT-125 in GBM Patients

How This Trial Compares With Other Options

This table compares participation in this trial with other options available for this condition. The information is drawn from published research and the trial’s study details and is provided for educational purposes only.

Factors	MT-125 (Dose-Escalation Study)	Current Standard: Radiotherapy + Temozolomide
What it is	An investigational drug regimen (MT-125) administered to adults with newly diagnosed IDH wild type and MGMT unmethylated glioblastoma in a sequential dose-escalation format to determine the safety, tolerability, and optimal dose.	The established current-care regimen for this diagnosis is radiotherapy combined with concomitant and adjuvant temozolomide, which is widely used and supported by strong evidence for adults with IDH wild type and MGMT unmethylated glioblastoma.
Role in the study / clinical care	MT-125 is being tested to assess safety (dose-limiting toxicity), tolerability, pharmacokinetics, and determine the highest tolerable dose. Its effect on clinical outcomes is exploratory.	Radiotherapy plus temozolomide serves as the therapeutic backbone for newly diagnosed adult glioblastoma and is considered routine clinical practice. No investigational procedure is involved in current standard care.
Primary use	Exploratory evaluation of MT-125 as a possible therapeutic agent for newly diagnosed IDH wild type, MGMT unmethylated glioblastoma to find the maximum tolerated dose and observe initial safety profile.	Curative-intent, evidence-based standard treatment that combines radiotherapy with temozolomide to prolong survival in adult glioblastoma.
How it is given or performed	MT-125 is given in different dose groups, starting from 25 mg and escalating up to 100 mg, with an expansion cohort at maximum tolerated dose and one dose lower, as defined by the study protocol.	Current standard treatment involves external beam radiation therapy with daily oral temozolomide during radiotherapy, followed by adjuvant cycles of temozolomide after radiotherapy completion.
Level of evidence/supporting data	Evidence is limited to ongoing early-phase clinical investigation. No patient outcome or survival data from this regimen is yet available.	Longstanding, high-quality evidence from randomized clinical trials and pooled analyses demonstrates a survival benefit for radiotherapy plus temozolomide in newly diagnosed glioblastoma, even though patients with MGMT unmethylated tumors have a smaller benefit than those with MGMT methylated tumors.
Important limitations	Safety and tolerability at all tested doses and any clinical benefit remain unknown. This is an early-stage trial with small sample sizes and no control group. Long-term efficacy, impact on survival, and comparison to established therapies are not yet established.	Patients with MGMT unmethylated GBM have only a modest benefit from temozolomide, and overall prognosis remains poor. Most patients ultimately progress despite current standard care.

Disclaimer: This comparison is created with the help of artificial intelligence(AI) and may contain errors or missing information. It is not medical advice and does not mean one option is better or safer than another. Clinical trials are still being studied, and joining a trial is your choice. Please consult your healthcare professional before making any decisions.

Who Can Participate

Age: 18Years +

All Genders

Eligibility Criteria

You may qualify if you...

Age 18 years or older at the time of consent
Newly diagnosed with histologically or molecularly confirmed IDH wild type and MGMT unmethylated glioblastoma
Eastern Cooperative Oncology Group (ECOG) Performance Status of 0, 1, or 2
Laboratory values within specified normal ranges: hemoglobin ≥9.0 g/dL, absolute neutrophil count ≥1500/mm3, platelet count ≥100,000/mm3, total bilirubin ≤1.5 times upper limit of normal, ALT and AST ≤3 times upper limit of normal (or ≤5 times if liver involvement), PT/INR/aPTT ≤1.5 times upper limit of normal or within therapeutic range if on anticoagulants, serum eGFR ≥60 ml/min
QTc interval ≤470 ms on triplicate 12-lead ECG
Left Ventricular Ejection Fraction (LVEF) ≥55% by echocardiography
Negative serum pregnancy test within 7 days before first dose for persons of childbearing potential
Ability to provide written informed consent
Ability to complete questionnaires independently or with assistance
Willingness to return for follow-up visits during active monitoring
Stable steroid dose for at least 2 weeks prior to enrollment

You will not qualify if you...

Pregnant or nursing persons
Persons of childbearing potential and persons able to father a child unwilling to use adequate contraception
Presence of co-morbid systemic illnesses or severe concurrent diseases that may interfere with safety assessment
Receiving any other investigational agents
Any disease, condition, or treatment that poses unacceptable risk or interferes with study conduct or data interpretation

Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person)

Treatment

Duration - 6 weeks

Participants receive MT-125 monotherapy combined with radiation therapy for their newly diagnosed glioblastoma. MT-125 is administered 5 consecutive days per week with 2 days off for a total of 6 weeks of outpatient treatment.

Weekly visits for up to 6 weeks