What side effects are associated with this type of intervention?

Common treatments for craniopharyngioma include surgery, radiation therapy, and investigational agents like IL-6 receptor inhibitors (e.g., Tocilizumab). Surgery can lead to complications such as infection, bleeding, and damage to surrounding brain structures. Radiation therapy may cause long-term toxicities like joint stiffness, skin changes, tissue necrosis, and secondary malignancies. Tocilizumab, an IL-6 receptor inhibitor, is being studied for its potential benefits, but known side effects from similar treatments include increased risk of infections, liver enzyme elevations, and gastrointestinal issues. Patients should discuss these potential risks with their healthcare provider.

What prior FDA approvals exist?

There is no specific mention of prior FDA approvals for the treatment of craniopharyngioma in the provided context. Tocilizumab, an IL-6 receptor inhibitor, is currently being studied for its efficacy in treating craniopharyngioma, but it has not yet been approved for this indication. Generally, treatments for craniopharyngioma have included surgical resection and radiation therapy, with ongoing research into targeted therapies and other novel approaches.

What other types of research exist?

Promising novel alternatives to Tocilizumab for Craniopharyngioma patients include targeted therapies such as BRAF inhibitors (e.g., vemurafenib) and MEK inhibitors (e.g., selumetinib), which have shown efficacy in treating low-grade gliomas and other brain tumors. Additionally, immune checkpoint inhibitors like nivolumab, which target PD-1, may offer potential benefits given their success in other refractory tumors.

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Monoclonal Antibodies

Tocilizumab for Craniopharyngioma

Q: What is the mechanism of action of this treatment?

Tocilizumab, an IL-6 receptor inhibitor, works by blocking the interleukin-6 receptor, thereby reducing inflammation and potentially limiting tumor growth or progression. This mechanism is significant for Craniopharyngioma patients as inflammation can contribute to tumor development and symptoms. By targeting IL-6, Tocilizumab may offer a novel therapeutic approach that could improve patient outcomes.

Phase < 1

Recruiting

Led By Margaret Macy, MD

Research Sponsored by University of Colorado, Denver

Eligibility Criteria Checklist

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

Must have

A serum creatinine based on age/gender as follows: (Age, Male, Female) 3 to < 6 years, 0.8, 0.8; 6 to < 10 years, 1, 1; 10 to < 13 years, 1.2, 1.2; 13 to < 16 years, 1.5, 1.4; 16 years to < 18 years, 1.7, 1.4

Recurrent or progressive* ACP treated with surgery and radiation

Must not have

cardiac arrhythmia

ongoing or active infection (including active tuberculosis)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up start of study to end of study, up to 5 years

Awards & highlights

No Placebo-Only Group

Summary

This trial tests if tocilizumab, an anti-inflammatory drug, can reach and affect brain tumors in patients with craniopharyngioma. If successful, patients will receive the drug regularly over several months to see if it helps control their tumors.

Who is the study for?

This trial is for children and young adults (2 to <21 years old) with a type of brain tumor called craniopharyngioma. It's open to those with new, recurrent, or progressive tumors that haven't been fully removed by surgery or have come back after treatment. Participants need functioning major organs and acceptable blood counts, must not be pregnant or breastfeeding, and should agree to use birth control.

What is being tested?

The study tests Tocilizumab in two phases: Phase 0 checks if the drug reaches the tumor when given before standard surgery. If successful, Phase 1 gives Tocilizumab every two weeks for up to a year to see how well it works as a treatment. Patients can move from Phase 0 to Phase 1 and will be monitored for five years.

What are the potential side effects?

Tocilizumab may cause allergic reactions in some individuals who are sensitive to it. Other potential side effects include increased risk of infection due to immune system suppression, liver function changes, and abnormal blood counts which could lead to bleeding issues or increased susceptibility to infections.

Eligibility Criteria

Inclusion Criteria

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

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My kidney function, based on my age and gender, meets the required levels.

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My ACP has come back or gotten worse after surgery and radiation.

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My child is active and can do most activities without help.

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I am between 2 and 20 years old.

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My previously confirmed ACP has gotten worse or come back.

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My ACP came back or got worse after surgery without radiation.

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I am able to care for myself and perform daily activities.

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My liver tests are within normal limits for my age.

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My organs are functioning well.

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I am over 16 and can care for myself but may not be able to do active work.

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I have a newly diagnosed ACP that can't be fully removed and is measurable.

Exclusion Criteria

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

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I have an irregular heartbeat.

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I do not have any ongoing or active infections.

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My cancer has spread to other parts of my body.

Select...

I have symptoms of heart failure.

Select...

I cannot come back for follow-up visits or get tests to check treatment side effects.

Select...

I have unstable chest pain.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ start of study to end of study, up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~start of study to end of study, up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and start of study to end of study, up to 5 years for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Feasibility Phase: Toxicity Profile

Phase 0: Presence of Tocilizumab and Metabolites

Secondary study objectives

Feasibility Phase: 1-Year Disease Stabilization

Feasibility Phase: Cytokines

Feasibility Phase: Immunity

+4 more

Side effects data

From 2017 Phase 4 trial • 59 Patients • NCT02034474

11%

Low ANC

nausea

agranulocytosis

Inpatient admission

fatigue

100%

80%

60%

40%

20%

Study treatment Arm

Placebo

Tocilizumab

Awards & Highlights

No Placebo-Only Group

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

Trial Design

2Treatment groups

Experimental Treatment

Group I: Tocilizumab Administration: Phase 0Experimental Treatment1 Intervention

In Phase 0, patients will receive one dose of tocilizumab prior to surgery.

Group II: Tocilizumab Administration: Feasibility PhaseExperimental Treatment1 Intervention

During the Feasibility Phase, patients will receive tocilizumab every 2 weeks for up to 13 cycles (approximately 1 year). Patients will be followed for up to 5 years.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Tocilizumab

2012

Completed Phase 4

~1840

0 / 17Eligibility 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

Tocilizumab, an IL-6 receptor inhibitor, works by blocking the interleukin-6 receptor, thereby reducing inflammation and potentially limiting tumor growth or progression. This mechanism is significant for Craniopharyngioma patients as inflammation can contribute to tumor development and symptoms. By targeting IL-6, Tocilizumab may offer a novel therapeutic approach that could improve patient outcomes.

Advances in Immunotherapies for Gliomas.Targeting the medulloblastoma: a molecular-based approach.Immunotherapy for Medulloblastoma: Current Perspectives.