What is the mechanism of action of this treatment?

Glioblastoma treatments primarily include surgery, radiation therapy, and chemotherapy. Surgery aims to remove as much of the tumor as possible, reducing the tumor burden. Radiation therapy uses high-energy rays to kill cancer cells and shrink tumors by damaging their DNA. Chemotherapy, such as temozolomide, works by interfering with the DNA replication of cancer cells, leading to cell death. Janus kinase inhibitors like Tofacitinib target specific signaling pathways involved in cell growth and immune response, potentially reducing tumor growth and modulating the immune environment. These mechanisms are crucial for Glioblastoma patients as they aim to control tumor progression, improve survival rates, and maintain quality of life.

What side effects are associated with this type of intervention?

Common treatments for Glioblastoma include chemotherapy, radiation therapy, and investigational therapies. Chemotherapy, particularly with temozolomide, often causes side effects such as nausea, vomiting, fatigue, and myelosuppression (reduced blood cell counts). Radiation therapy can lead to fatigue, hair loss at the treatment site, and skin changes. Investigational therapies like ONC201 may have side effects that are still being studied, but can include gastrointestinal disturbances and fatigue. It is important to discuss potential side effects with a healthcare provider to manage and mitigate them effectively.

What prior FDA approvals exist?

The FDA has approved several treatments for Glioblastoma, including temozolomide, an oral alkylating agent, and bevacizumab, a monoclonal antibody targeting VEGF. These treatments aim to inhibit tumor growth and angiogenesis. While Tofacitinib, a Janus kinase inhibitor, is being studied for recurrent Glioblastoma, there are no current FDA approvals for Janus kinase inhibitors specifically for this condition. The focus remains on therapies that target tumor proliferation and vascular support.

What other types of research exist?

Promising alternatives to Tofacitinib for glioblastoma patients include: 1) Ivosidenib, a mutant-IDH1 inhibitor, which has shown tolerability and some efficacy in IDH1-mutant gliomas. 2) Vorasidenib, a dual mutant-IDH1/2 inhibitor, currently in phase III trials for IDH1/2-mutant gliomas. 3) BRAF/MEK inhibitors like dabrafenib and trametinib, which have demonstrated efficacy in BRAF V600E-mutant gliomas. 4) Immunotherapies such as PD-1 blockade with nivolumab, which has shown activity in recurrent high-grade meningiomas and may have potential in glioblastoma.

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Janus Kinase (JAK) Inhibitor

Tofacitinib for Glioblastoma

Phase 3

Recruiting

Led By Michael Youssef, MD

Research Sponsored by University of Texas Southwestern Medical Center

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Karnofsky Performance Status (KPS) ≥ 60%.

GBMs of the study patients must have EGFR gene amplification.

Must not have

History of allergic reactions attributed to compounds of similar composition to Tofacitinib.

Prior treatment with an EGFR or JAK inhibitor.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 2 years after study treatment

Awards & highlights

Pivotal Trial

No Placebo-Only Group

Summary

This trial tests Tofacitinib, an oral medication, on patients with recurrent Glioblastoma. The drug works by reducing immune system activity, potentially slowing or stopping cancer growth. Tofacitinib is an oral medication initially used for the treatment of rheumatoid arthritis.

Who is the study for?

Adults with recurrent Glioblastoma (GBM) who've had prior treatment, can swallow pills, and have a Karnofsky Performance Status of at least 60%. They must use contraception if of childbearing potential. Excluded are those with uncontrolled hypertension, other recent cancers, certain allergies or conditions that affect drug absorption or compliance.

What is being tested?

The trial is testing the effects of Tofacitinib 10mg on patients with recurrent GBM. It aims to see how well this medication works for those whose tumors have EGFR gene amplification and who meet specific health criteria.

What are the potential side effects?

While not explicitly listed in the provided information, common side effects of Tofacitinib may include infections, headaches, diarrhea, high blood pressure and cholesterol levels. More serious risks could involve immune system issues and increased risk of certain cancers.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I can care for myself but may need occasional help.

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My brain tumor shows increased EGFR gene activity.

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My brain tumor has recurred and is confirmed to be a specific type of aggressive cancer.

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I can swallow pills.

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I agree to use barrier contraception during and for 6 months after treatment.

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I am 18 years old or older.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am allergic to medications similar to Tofacitinib.

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I have been treated with an EGFR or JAK inhibitor before.

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I do not have any uncontrolled illnesses.

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I have had a severe spike in blood pressure or my high blood pressure is not well-controlled.

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I am currently being treated with Bevacizumab.

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I have severe, ongoing nausea or vomiting, trouble swallowing, or have had major gut surgery.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 2 years after study treatment

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 2 years after study treatment

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 2 years after study treatment for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Progression-free survival (PFS) of the study cohort as defined by RANO criteria.

Secondary study objectives

Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability].

Overall survival (OS) of the study cohort.

Tumor response by RANO criteria.

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Tofacitinib 10 mgExperimental Treatment1 Intervention

Participants will take the 10mg Tofacitinib twice daily until evidence of progression, intolerance of treatment, withdrawal of consent, or death.

0 / 12Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Glioblastoma treatments primarily include surgery, radiation therapy, and chemotherapy. Surgery aims to remove as much of the tumor as possible, reducing the tumor burden. Radiation therapy uses high-energy rays to kill cancer cells and shrink tumors by damaging their DNA. Chemotherapy, such as temozolomide, works by interfering with the DNA replication of cancer cells, leading to cell death. Janus kinase inhibitors like Tofacitinib target specific signaling pathways involved in cell growth and immune response, potentially reducing tumor growth and modulating the immune environment. These mechanisms are crucial for Glioblastoma patients as they aim to control tumor progression, improve survival rates, and maintain quality of life.