What side effects are associated with this type of intervention?

Common treatments for brain tumors, such as temozolomide, often result in side effects including nausea, vomiting, fatigue, and myelosuppression (reduced bone marrow activity leading to fewer blood cells). Other treatments, like bevacizumab, can cause hypertension, proteinuria, and increased risk of bleeding or thromboembolic events. Scalp cooling devices used to prevent alopecia during chemotherapy can lead to headaches, scalp pain, and a feeling of cold. Investigational drugs like ONC206, while still under study, may share similar side effects, including gastrointestinal disturbances and hematologic toxicities.

What prior FDA approvals exist?

The FDA has approved several drugs for the treatment of brain tumors, particularly glioblastomas and other high-grade gliomas. Temozolomide, an oral alkylating agent, is commonly used in combination with radiation therapy for newly diagnosed glioblastoma. Bevacizumab, an anti-angiogenic drug, has been approved for recurrent glioblastoma. Additionally, investigational drugs like ONC201, which targets H3 K27M-mutant gliomas, and other emerging therapies such as CAR T-cell therapy and oncolytic viruses, are under active investigation. These treatments aim to improve outcomes for patients with aggressive CNS neoplasms.

What other types of research exist?

Promising alternatives to ONC206 for brain tumor patients include: 1) Ivosidenib, a mutant-IDH1 inhibitor, which has shown tolerability and efficacy in IDH1-mutant gliomas. 2) Vorasidenib, a dual mutant-IDH1/2 inhibitor, currently in phase III trials for IDH1/2-mutant gliomas. 3) Bevacizumab, an anti-angiogenic therapy, which has been used in combination with other treatments for recurrent gliomas. 4) CDK4/6 inhibitors like abemaciclib, which have shown complete response in leptomeningeal carcinomatosis secondary to breast cancer and may have potential in CNS neoplasms.

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Small Molecule

ONC206 for Brain Cancer

Phase 1

Recruiting

Led By Mark Gilbert, MD

Research Sponsored by Chimerix

Eligibility Criteria Checklist

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

Must have

Patients aged ≥18 years with a recurrent, primary CNS neoplasm with histologically confirmed primary CNS neoplasm including various subtypes

Patients must have normal organ and marrow function as defined

Must not have

Patients with active cardiac disease

Patients with a known HIV-positive test on combination anti-retroviral therapy

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 28 days

Awards & highlights

Summary

This trial tests ONC206, a new oral drug, in patients whose brain or spinal cord tumors have returned. The goal is to find the highest safe dose by increasing the amount given over time and monitoring for side effects.

Who is the study for?

Adults over 18 with recurrent brain tumors, who are in good physical condition (KPS ≥70), have normal organ/marrow function, and haven't had recent chemotherapy or major surgery. They must be able to swallow pills or liquids, consent to the study, test negative for COVID-19, provide a tumor sample, undergo MRI with contrast, and have measurable disease. Pregnant women and those on certain drugs or with specific medical conditions can't participate.

What is being tested?

The trial is testing ONC206's safety at different doses for treating various types of recurring brain tumors. It's an early-stage study where patients take oral ONC206 either once weekly or multiple times a week to find the highest dose they can tolerate without severe side effects.

What are the potential side effects?

While not explicitly listed in the provided information since this is a Phase I trial primarily focused on determining safe dosage levels, potential side effects may include typical reactions such as nausea, fatigue, digestive issues but will be closely monitored throughout the trial.

Eligibility Criteria

Inclusion Criteria

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

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I am over 18 and have a confirmed brain tumor that has come back.

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My organ and bone marrow functions are normal.

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My cancer has returned and can be measured, with no further standard treatments available.

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I am mostly able to care for myself.

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I haven't had experimental or standard chemotherapy recently.

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All my side effects from previous treatments have resolved.

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I can swallow pills or liquids without difficulty.

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I can provide a sample of my tumor from a previous surgery or biopsy.

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I can undergo an MRI with contrast.

Exclusion Criteria

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

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I have a heart condition that is currently causing symptoms.

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I am HIV-positive and on combination anti-retroviral therapy.

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I have a stomach or intestine condition that affects how my body absorbs medicine.

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I have a history of heart problems.

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I have not taken strong medication affecting liver enzymes in the last 14 days.

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I am not pregnant or breastfeeding.

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I have had a stroke in the past 3 months.

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I have epilepsy that doesn't respond to treatment or I've had recent seizures.

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I am currently on blood thinners like warfarin.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 28 days

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~28 days

This trial's timeline: 3 weeks for screening, Varies for treatment, and 28 days for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Maximum Tolerated Dose (MTD) of single-agent, oral ONC206

Number of Participants who Experienced Dose-Limiting Toxicities (DLTs)

Trial Design

1Treatment groups

Experimental Treatment

Group I: ONC206Experimental Treatment1 Intervention

0 / 18Eligibility 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

Common treatments for brain tumors include temozolomide, bevacizumab, and investigational drugs like ONC201 and ONC206. Temozolomide is an alkylating agent that damages the DNA of cancer cells, leading to cell death. Bevacizumab is an anti-angiogenic drug that inhibits the growth of blood vessels that supply the tumor, thereby starving it of nutrients. ONC201 and ONC206 are investigational drugs that target specific molecular pathways in cancer cells, such as the dopamine receptor D2 (DRD2) and mitochondrial protease ClpP, leading to cancer cell death. These treatments are crucial for brain tumor patients as they offer targeted approaches to disrupt tumor growth and improve survival outcomes.

Bioinformatics analysis reveals potential candidate drugs for different subtypes of glioma.