Troriluzole for Recurrent Brain Cancer

Recruiting at 2 trial locations

Overseen By Ugonma Chukwueke, MD

Age: 18+

Sex: Any

Trial Phase: Phase < 1

Sponsor: Eudocia Quant Lee, MD

Must not be taking: CYP1A2 inhibitors, Anti-glutamergic agents

Disqualifiers: Leptomeningeal disease, Hepatitis, HIV, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 13

27 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial investigates whether a drug called troriluzole can help treat individuals with recurrent glioblastoma, an aggressive brain cancer that returns after treatment. Participants will be divided into two groups: one group will take troriluzole before and after surgery, while the other will take it only after surgery. This trial suits those diagnosed with recurrent glioblastoma who have undergone previous treatment but still require surgery. As an Early Phase 1 trial, this research focuses on understanding how troriluzole works in people, offering participants the opportunity to be among the first to receive this potentially groundbreaking treatment.

Do I have to stop taking my current medications for the trial?

You may need to stop taking certain medications before joining the trial, especially if they are strong inhibitors or inducers of specific enzymes or are known to affect the liver. A washout period (time without taking certain medications) of 10 days or 5 half-lives is required for these drugs before starting the study treatment.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research has shown that troriluzole has undergone testing in several studies to assess its safety in people. Early results suggest it is generally well-tolerated. For instance, one study on patients with brain tumors found it helped slow tumor growth without major safety issues.

Troriluzole is a prodrug of riluzole, meaning it converts into riluzole in the body. Riluzole already has FDA approval for another condition, which provides some confidence in its safety.

While early studies suggest troriluzole is safe, this trial remains in its early stages. More research is needed to fully understand its safety in people. Participants should consider this and discuss any concerns with their healthcare provider. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for brain cancer?

Troriluzole is unique because it targets glutamate, a neurotransmitter that can promote tumor growth when present in excess. Unlike standard treatments for recurrent brain cancer, like surgery combined with radiation or chemotherapy, Troriluzole offers a novel mechanism by modulating glutamatergic signaling. Researchers are excited because this approach could slow tumor progression by directly interfering with a pathway that cancer cells exploit, potentially leading to improved outcomes for patients.

What evidence suggests that troriluzole might be an effective treatment for recurrent glioblastoma?

Research has shown that troriluzole, a form of the drug riluzole, might help treat brain cancer by slowing tumor growth. A study with brain tumor patients found that troriluzole was safe and well-tolerated. Other research suggests it can effectively slow the progression of certain tumors. In this trial, participants will join one of two groups: Group A will receive presurgical troriluzole, and Group B will receive troriluzole after surgery. While more information is needed about its use for recurring glioblastoma, these early results are encouraging. This suggests that troriluzole could be a useful treatment option for brain cancer patients. ^{1 2 3 4 6}

Who Is on the Research Team?

Ugonma Chukwueke, MD

Principal Investigator

Dana-Farber Cancer Institute

Are You a Good Fit for This Trial?

This trial is for individuals with recurrent glioblastoma, which is a type of brain tumor that has returned after treatment. Participants should not have any mutations in the IDH gene.

Inclusion Criteria

My tumor does not have IDH1/2 mutations, confirmed by DNA testing.

My MRI shows the tumor has reached the brain's surface layer.

I have had radiotherapy, with or without chemotherapy.

Exclusion Criteria

Laboratory values at the Screening Visit outside specified ranges

I have recently been treated with cancer medications.

I have not had major surgery in the last 28 days.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Pre-surgical Treatment

Participants in Group A receive Troriluzole prior to standard-of-care tumor resection surgery

6 days

Daily visits for drug administration

Surgery

Standard-of-care tumor resection surgery with pre-op and post-op MRI

1 day

1 visit (in-person)

Post-surgical Treatment

Participants receive Troriluzole in 28-day cycles

Ongoing cycles

Daily visits for drug administration, MRIs every 8 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 years

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

What Are the Treatments Tested in This Trial?

Interventions

• Troriluzole

Trial Overview The study is testing Troriluzole, a drug designed to treat recurrent glioblastoma. It's given before surgery to see if it can help control the growth of the tumor.

How Is the Trial Designed?

Group I: Group B: Surgery + Troriluzole Experimental Treatment 1 Intervention

9 participants will be randomly assigned to this group and with complete: * Baseline visit with assessments and MRI * Day 0: pre-op MRI * Day 0: standard of care surgical resection of tumor * Day 0: post-op MRI * Cycle 1 through Cycle 3: --Days 1 through 28 of 28 day cycle: Predetermined dose of Troriluzole 2x daily. * Cycle 3 through End of Treatment: --Days 1 through 28 of 28 day cycle: Predetermined dose of Troriluzole 2x daily. * MRIs every 8 weeks while on treatment. * End of study visit with MRI * Follow up every 3 months for 1 year, and then every 6 months for the next 3 years.

Group II: Group A: Presurgical Troriluzole Experimental Treatment 1 Intervention

18 participants will be randomly assigned to this group and with complete: * Baseline visit with assessments and MRI. * Cycle 0: * Day -6 through Day 0: Predetermined dose of Troriluzole 2x daily. * Day 0: pre-op MRI * Day 0: standard of care surgical resection of tumor * Day 0: post-op MRI * Cycle 1 through Cycle 3: --Days 1 through 28 of 28 day cycle: Predetermined dose of Troriluzole 2x daily. * Cycle 3 through End of Treatment: --Days 1 through 28 of 28 day cycle: Predetermined dose of Troriluzole 2x daily. * MRIs every 8 weeks while on treatment. * End of study visit with MRI * Follow up every 3 months for 1 year, and then every 6 months for the next 3 years.

Who Is Running the Clinical Trial?

Eudocia Quant Lee, MD

Lead Sponsor

Trials

2

Recruited

110+

Ugonma Chukwueke

Lead Sponsor

National Institutes of Health (NIH)

Collaborator

Trials

2,896

Recruited

8,053,000+

Biohaven Pharmaceuticals, Inc.

Industry Sponsor

Trials

49

Recruited

30,100+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06552260

Study Details | NCT06552260 | A Surgical Window of ... This research study is studying troriluzole as a possible treatment for recurrent glioblastoma. ... This is an open-label, randomized window-of-opportunity study ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9250196/

A phase Ib dose-escalation study of troriluzole (BHV-4157), an ... We conducted a phase Ib trial to determine the safety and tolerability of the combination of the riluzole prodrug troriluzole (BHV-4157, trigriluzole) and the ...

3. cancer.gov cancer.gov/research/participate/clinical-trials-search/v?id=NCI-2024-09577

Troriluzole for the Treatment of Recurrent IDH Wild Type ... This early phase I trial tests the safety and tolerability of troriluzole and how well it works to slow tumor growth in patients with IDH wild type ...

4. clinicaltrials.eu clinicaltrials.eu/trial/study-of-troriluzole-and-adi-peg-20-for-patients-with-newly-diagnosed-and-recurrent-glioblastoma/

Study of Troriluzole and ADI-PEG-20 for Patients with ... This clinical trial investigates the effectiveness of Troriluzole and ADI-PEG-20 in treating patients with newly diagnosed or recurrent ...

5. ir.biohaven.com ir.biohaven.com/news-releases/news-release-details/fda-issues-complete-response-letter-biohavens-vyglxia

FDA Issues Complete Response Letter for Biohaven's ... Compelling data from troriluzole's new drug application (NDA) included: a 3-year real-world evidence study (Study 206-RWE) showing slowing ...

6. delta.larvol.com delta.larvol.com/Products/?ProductId=961b8542-fc28-478b-bb67-235666e43789

Dazluma (troriluzole) / Biohaven Troriluzole attenuates opioid intake, reinforcing efficacy, seeking behaviours, physical dependence and antinociceptive tolerance in rats.