What side effects are associated with this type of intervention?

Common treatments for ependymoma include surgery, radiation therapy, and chemotherapy. Side effects of these treatments can vary but often include neurocognitive impairment, endocrine abnormalities, and an increased risk of secondary cancers. Specifically, for treatments similar to 5-Azacytidine, which is a DNA methyltransferase inhibitor, side effects may include myelosuppression (leading to anemia, neutropenia, and thrombocytopenia), gastrointestinal symptoms (such as nausea and vomiting), and potential neurotoxicity. These side effects can significantly impact the patient's quality of life and require careful management.

What prior FDA approvals exist?

As of now, there are no specific FDA-approved treatments for Ependymoma that are directly similar to 5-Azacytidine, a DNA Methyltransferase Inhibitor. Treatment for Ependymoma typically involves surgical resection followed by radiation therapy. Chemotherapy is less commonly used and is generally considered for recurrent cases or when surgery and radiation are not fully effective. The study of 5-Azacytidine in the context of Ependymoma is part of ongoing research to explore new therapeutic options.

What other types of research exist?

Promising novel alternatives to 5-Azacytidine for Ependymoma patients include pharmacological inhibitors of DNMTs and histone deacetylases, which have shown significant inhibition of tumor growth in C19MC-associated brain tumors. Additionally, targeted therapies such as bevacizumab, everolimus, and selumetinib have demonstrated efficacy in treating various brain tumors and may offer potential benefits for Ependymoma patients.

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Anti-metabolites

5-Azacytidine Infusion for Brain Cancer (5-AZA Trial)

Phase 1

Recruiting

Led By David I Sandberg, MD

Research Sponsored by The University of Texas Health Science Center, Houston

Eligibility Criteria Checklist

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

Must have

Patients with histologically verified ependymoma, with recurrence or progression anywhere in the brain and/or spine

Patients with refractory disease, defined as residual tumor which has not been completely cleared despite prior treatments, originating in the posterior fossa of the brain

Must not have

Received another investigational or chemotherapy agent or radiation therapy within 7 days prior to 5-AZA infusion into the fourth ventricle

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 1 day after surgery to remove tumor (which is about 1 week before the first infusion)

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing the safety and effectiveness of a drug called 5-Azacytidine for patients with recurring brain tumors. The goal is to find the best dose and see if the drug can help reduce the tumors.

Who is the study for?

This trial is for patients with a specific brain cancer called recurrent posterior fossa ependymoma. They must have an implanted catheter or agree to one, a life expectancy of at least 12 weeks, stable neurological conditions, and adequate bone marrow function. Pregnant women, those with untreated infections or recent treatments from other trials are excluded.

What is being tested?

The study tests different dosing frequencies of the drug 5-Azacytidine (5-AZA) infused directly into the fourth ventricle of the brain to find the safest maximum dose and assess its effectiveness against tumor activity through imaging studies.

What are the potential side effects?

While not explicitly listed in your summary, side effects for this type of treatment could include headaches, nausea, infection risk at infusion site, potential blood disorders due to bone marrow involvement and possible worsening of existing neurological deficits.

Eligibility Criteria

Inclusion Criteria

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

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My ependymoma cancer has come back or gotten worse in my brain or spine.

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My brain tumor in the back of my head hasn't gone away despite treatment.

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I have or agree to get a catheter in my brain for treatment.

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I have recovered from the side effects of my previous cancer treatments.

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My blood tests show enough neutrophils, platelets, and hemoglobin.

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I am mostly active and can do things for myself.

Exclusion Criteria

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

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I haven't had any experimental drugs, chemotherapy, or radiation within the last week.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 1 day after surgery to remove tumor (which is about 1 week before the first infusion)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~1 day after surgery to remove tumor (which is about 1 week before the first infusion)

This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 day after surgery to remove tumor (which is about 1 week before the first infusion) for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Number of participants who experienced dose-limiting toxicity (DLT)

Secondary study objectives

Number of participants with disease progression as assessed by magnetic resonance imaging (MRI)

Awards & Highlights

No Placebo-Only Group

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

Trial Design

3Treatment groups

Experimental Treatment

Group I: group 3Experimental Treatment1 Intervention

5-Azacytidine (5-AZA) group 3: Enrolled patients will undergo surgical placement of a ventricular catheter into the fourth ventricle that will be attached to a subcutaneously placed reservoir. Patients will be divided into 3 dose groups and receive 8 weeks of intraventricular 5-AZA (12 mg) into the fourth ventricle. Patients in Group 3 will receive four 5-AZA infusions every week.

Group II: group 2Experimental Treatment1 Intervention

5-Azacytidine (5-AZA) group 2: Enrolled patients will undergo surgical placement of a ventricular catheter into the fourth ventricle that will be attached to a subcutaneously placed reservoir. Patients will be divided into 3 dose groups and receive 8 weeks of intraventricular 5-AZA (12 mg) into the fourth ventricle. Patients in Group 2 will receive three 5-AZA infusions every week.

Group III: group 1Experimental Treatment1 Intervention

5-Azacytidine (5-AZA) group 1: Enrolled patients will undergo surgical placement of a ventricular catheter into the fourth ventricle that will be attached to a subcutaneously placed reservoir. Patients will be divided into 3 dose groups and receive 8 weeks of intraventricular 5-AZA (12 mg) into the fourth ventricle. Patients in Group 1 will receive two 5-AZA infusions every week.

0 / 7Eligibility 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

5-Azacytidine, a DNA methyltransferase inhibitor, works by incorporating into DNA and RNA, leading to the inhibition of DNA methylation. This demethylation can reactivate tumor suppressor genes that were previously silenced by hypermethylation, thereby inhibiting tumor growth. For Ependymoma patients, this mechanism is significant as it offers a targeted approach to potentially control tumor progression and improve outcomes. Other common treatments for Ependymoma include surgery, radiation therapy, and chemotherapy, which aim to remove or destroy tumor cells but may not specifically target the molecular pathways involved in tumor growth.

Treatment of adult brainstem glioma with combined antiangiogenic therapy: a case report and literature review.