Methimazole for Brain Tumor

Overseen By David Peereboom, MD

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Case Comprehensive Cancer Center

Must not be taking: Investigational agents

Disqualifiers: Uncontrolled illness, Other malignancy, Hyperthyroidism, others

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

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 4 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 1

19 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a drug called Methimazole to determine its potential in treating glioblastoma, a type of brain tumor. Although Methimazole typically treats thyroid issues, researchers aim to assess its safety and effectiveness for brain tumors. Participants will receive varying doses to identify the optimal balance between benefits and side effects. This trial suits individuals requiring tumor surgery and diagnosed with grade 4 glioma, a highly aggressive brain cancer. As a Phase 2 trial, the research focuses on evaluating the treatment's effectiveness in an initial, smaller group of participants.

Will I have to stop taking my current medications?

The trial protocol does not specify if you need to stop taking your current medications. However, there are specific time intervals required from prior treatments, and certain conditions must be met for patients on anticoagulants. It's best to discuss your current medications with the study team.

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

Research shows that Methimazole is generally safe for people, as it is already used to treat thyroid problems. Although not yet approved for treating brain tumors, individuals using Methimazole for thyroid issues have tolerated it well. In this study, participants with glioblastoma will receive different doses. Researchers will start with a low dose and increase it only if no serious side effects occur. This careful, step-by-step method helps ensure participant safety. If any side effects arise, researchers will adjust the dose as needed. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Most treatments for recurrent glioblastoma, like chemotherapy and radiation, aim to directly destroy cancer cells. But methimazole offers a different approach by potentially lowering thyroid hormone levels, which might affect tumor growth indirectly. Researchers are excited about methimazole because it could complement existing therapies by targeting the tumor environment, possibly enhancing the effectiveness of surgical resection and other treatments. This unique mechanism could provide new hope for patients who have limited options with current standards of care.

What evidence suggests that Methimazole might be an effective treatment for brain tumors?

Research suggests that Methimazole, which participants in this trial will receive, might help treat glioblastoma, a type of brain tumor. Although typically used for thyroid issues, studies indicate it could also benefit brain tumors by affecting certain hormones and chemicals in the body. Some early results show that Methimazole might enhance other cancer treatments by boosting the body's natural ability to fight cancer with hydrogen sulfide. While more data is needed to confirm its success in treating glioblastoma, Methimazole's mechanism gives researchers hope that it could be a valuable tool against this aggressive cancer. ^{1 3 4 5 6}

Who Is on the Research Team?

David Peereboom, MD

Principal Investigator

Cleveland Clinic Taussig Cancer Institute, Case Comprehensive Cancer Center

Are You a Good Fit for This Trial?

Adults with a specific brain tumor called glioblastoma, who are planning to have surgery for it, can join this trial. They need normal organ function and blood levels, no serious recent treatments or infections, and must be able to swallow pills. Pregnant women or those with certain medical conditions like heart failure or uncontrolled thyroid disease cannot participate.

Inclusion Criteria

I am able to care for myself but may not be able to do active work.

I can swallow whole tablets.

I had hepatitis C but am cured, or I'm being treated with no detectable virus.

Exclusion Criteria

Subjects receiving any other investigational agents

I do not have any uncontrolled illnesses that could affect my participation.

Pregnant or breastfeeding

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Pre-operative Methimazole Administration

Participants receive oral methimazole for at least 5 days pre-operatively or until lower circulating thyroid hormone levels are achieved

1 week

1 visit (in-person)

Surgical Resection and Post-operative Methimazole

Participants undergo surgical resection, followed by methimazole administration starting no sooner than 10 days post-op for 4 weeks

4 weeks

Multiple visits for pharmacodynamic assays and MRI

Secondary Chemotherapy and Monitoring

Secondary chemotherapy may be added at the treating physician's discretion, with continued monitoring through PBPD assays and MRI

4 weeks

Multiple visits for pharmacodynamic assays and MRI

Follow-up

Participants are monitored for safety and effectiveness after treatment

Up to 6 months

What Are the Treatments Tested in This Trial?

Interventions

• Methimazole

Trial Overview The trial is testing Methimazole's safety and effectiveness in treating glioblastoma. Participants will receive varying doses of the drug before their surgery to see how well they tolerate it and if it helps control the tumor growth. The study includes regular health checks, MRI scans, and blood tests.

How Is the Trial Designed?

Group I: Surgical Resection, Pharmacodynamic Assays, and Methimazole Experimental Treatment 3 Interventions

Participants with recurrent glioblastoma for whom surgical resection is indicated will have baseline peripheral blood pharmacodynamic assays (PBPD) followed by oral mehimazole at least 5 days pre-operatively or until lower circulating theyroid hormone levels are achieved. PBPD assays will then be repeated. After surgical resection, PBPD will be repeated a day later. When the participant is deemed able to begin methimazole (no sooner than 10 days post-op) PBPD assays will be repeated and the participant will begin methimazole for 4 weeks. At the end of the first 4 week cycle, an MRI will be performed. PBPD assays will be repeated after which secondary chemotherapy will be added at the treating physician's discretion. After 4 weeks, PBPD will be repeated and the participant will undergo another MRI at 8 weeks.

Methimazole is already approved in United States, European Union, Canada for the following indications:

Approved in United States as Tapazole for:

• Hyperthyroidism

Approved in European Union as Thiamazole for:

• Hyperthyroidism
• Graves' disease
• Toxic multinodular goiter
• Thyrotoxic crisis

Approved in Canada as Thiamazole for:

• Hyperthyroidism

Who Is Running the Clinical Trial?

Case Comprehensive Cancer Center

Lead Sponsor

Trials

472

Recruited

33,400+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05607407

Methimazole in Patients With Progressive Glioblastoma The purpose of this study is to test the effectiveness, safety, and tolerability of a drug called Methimazole. The investigational drug, Methimazole is not ...

2. trialx.com trialx.com/clinical-trials/listings/242424/methimazole-in-patients-with-progressive-glioblastoma/

Methimazole in Patients With Progressive Glioblastoma Overview. The purpose of this study is to test the effectiveness, safety, and tolerability of a drug called Methimazole.

3. academic.oup.com academic.oup.com/neuro-oncology/article/26/Supplement_8/viii293/7889451

A WINDOW OF OPPORTUNITY TRIAL OF METHIMAZOLE IN ... This window of opportunity trial evaluates safety, HPC in plasma and tumor, and efficacy of methimazole + chemotherapy in patients with ...

4. consultqd.clevelandclinic.org consultqd.clevelandclinic.org/can-boosting-hydrogen-sulfide-bolster-standard-of-care-glioblastoma-therapy-to-extend-survival

Can Boosting Hydrogen Sulfide Bolster Standard-of-Care ... New Cleveland Clinic research efforts are exploring the cancer-suppressing effects of endogenous hydrogen sulfide (H2S) in glioblastoma.

5. withpower.com withpower.com/trial/phase-2-glioblastoma-2024-51167

Methimazole for Brain Tumor The purpose of this study is to test the effectiveness, safety, and tolerability of a drug called Methimazole. The investigational drug, Methimazole is not ...

6. centerwatch.com centerwatch.com/clinical-trials/listings/NCT05607407/methimazole-in-patients-with-progressive-glioblastoma

Methimazole in Patients With Progressive Glioblastoma The investigational drug, Methimazole is not FDA approved for brain tumors, but it is used to treat thyroid illnesses. Different doses of ...