Actively Recruiting

Early Phase 1
Age: 18Years +
All Genders
ID06552260

A Surgical Window of Opportunity Clinical Trial of Troriluzole in Recurrent IDH Wild-Type Glioblastoma

Led by Ugonma Chukwueke · Updated on 2025-09-11

27

Participants Needed

3

Research Sites

52 weeks

Total Duration

On this page

Sponsors

U

Ugonma Chukwueke

Lead Sponsor

N

National Institutes of Health (NIH)

Collaborating Sponsor

AI-Summary

What this Trial Is About

Researchers are studying troriluzole as a potential treatment for people with recurrent isocitrate dehydrogenase wild-type (IDH WT) glioblastoma, a type of brain tumor. This is an open-label, randomized early phase clinical trial designed as a surgical window-of-opportunity study to see how active troriluzole is on tumors. The study is funded by Biohaven Pharmaceuticals and involves about 27 participants who have surgically accessible tumors. Participants are randomly assigned in a 2:1 ratio to one of two groups. Group A receives troriluzole twice daily for six days before surgery, followed by surgery and continued troriluzole treatment in 28-day cycles. Group B undergoes surgery first without pre-surgery troriluzole, then starts the same post-surgery troriluzole treatment. Both groups have MRI scans before and after surgery and every eight weeks during treatment, with follow-up visits every three months for one year and then every six months for three more years. Participants will have screening evaluations, blood tests, tumor biopsies, MRI scans, and electrocardiograms (ECGs) throughout the study. Researchers will measure the effect of troriluzole on brain activity during surgery using electrocorticography and analyze tumor tissue for various biological markers. Safety will be monitored closely, and the total participation may last several years due to the extended follow-up schedule.

CONDITIONS

Brief Title

A Surgical Window of Opportunity Clinical Trial of Troriluzole in Recurrent IDH Wild-Type Glioblastoma

Who Can Participate

Age: 18Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Age 18 years or older
  • Histopathologically confirmed IDH-wildtype glioblastoma, WHO Grade 4, including gliosarcoma variants
  • Prior treatment with radiotherapy with or without chemotherapy
  • Recurrent or progressive disease with no more than 2 prior relapses
  • Confirmed measurable disease per RANO 2.0 for GBM
  • Tumor documented as IDH1/2 wildtype by DNA sequencing in certified lab
  • Availability of archival tumor tissue for mutation testing
  • Karnofsky Performance Status of 60 or higher
  • Candidate for surgical tumor removal
  • Tumor extends to cortical gray matter based on MRI
  • Participants with prior or concurrent malignancy that does not interfere with study
  • Cardiac function assessed as NYHA class 2B or better if history of heart disease
  • Women of child-bearing potential must use highly effective contraception during and 1 month after treatment
  • Male participants must use highly effective contraception from first dose through 3 months after last dose
  • Ability to understand and willing to sign informed consent
Not Eligible

You will not qualify if you...

  • Low blood counts or abnormal liver/kidney function as defined by specific lab values
  • Positive pregnancy test in individuals of childbearing potential
  • Presence of diffuse leptomeningeal or extracranial disease
  • Prior treatment with troriluzole or riluzole
  • Recent treatments with certain chemotherapy or anti-VEGF therapies within specified time frames
  • Use of investigational agents within 28 days or 5 half-lives prior to study
  • Recent radiotherapy within 12 weeks unless new tumor outside prior radiation field
  • Significant allergy or toxicity from prior therapy not resolved to mild levels
  • Major surgery within 28 days prior to study drug
  • Active or unstable infections requiring systemic therapy
  • Contraindications to MRI
  • Use of strong CYP1A2 inhibitors or inducers, anti-glutamatergic agents, or hepatotoxic drugs without required washout
  • Pregnant or breastfeeding females
  • History of interstitial lung disease
  • Known hepatitis B, HIV, or active hepatitis C needing treatment
  • Any severe medical or psychiatric condition increasing risk or interfering with study
  • Difficulty swallowing, malabsorption, or chronic gastrointestinal disease affecting drug absorption

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person)

Run-in Period

Duration - 7 days (Day -6 through Day 0)

Participants in Group A receive Troriluzole twice daily prior to tumor resection surgery.

1 baseline visit and pre-op MRI on Day 0

Surgery and Immediate Post-operative Care

Duration - 1 day (Day 0)

Participants undergo standard-of-care tumor resection surgery with MRI scans before and after surgery.

1 surgery day with pre-op and post-op MRIs

Treatment

Duration - Repeated 28-day cycles until disease progression or discontinuation

Participants receive Troriluzole orally twice daily in repeated 28-day cycles after surgery.

MRIs every 8 weeks while on treatment

Follow-up

Duration - Up to 4 years

Participants are monitored with MRI scans every 3 months for 1 year, then every 6 months for the next 3 years.

Visits every 3 months for 1 year, then every 6 months for 3 years

Trial Site Locations

Total: 3 locations

1

Brigham and Women's Hospital

Boston, Massachusetts, United States, 02215

Actively Recruiting

2

Dana-Farber Cancer Institute

Boston, Massachusetts, United States, 02215

Actively Recruiting

3

Massachusetts General Hospital Cancer Center

Boston, Massachusetts, United States, 02215

Actively Recruiting

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Research Team

U

Ugonma Chukwueke, MD

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

2

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