What is the mechanism of action of this treatment?

Common treatments for brain tumors include surgery, radiation therapy, chemotherapy, and targeted therapies. Safusidenib, an IDH1 mutation inhibitor, works by targeting the mutated IDH1 enzyme, which is involved in the abnormal metabolic processes that drive tumor growth. This targeted approach can potentially halt tumor progression with fewer side effects compared to traditional therapies. Understanding these mechanisms is crucial for brain tumor patients as it allows for more personalized and effective treatment plans, improving outcomes and quality of life.

What side effects are associated with this type of intervention?

Common treatments for brain tumors, such as temozolomide and bevacizumab, have known side effects including fatigue, nausea, and increased risk of infection. Temozolomide can cause hematologic toxicities like thrombocytopenia and neutropenia. Bevacizumab is associated with hypertension, proteinuria, and increased risk of bleeding and thromboembolic events. Targeted therapies like IDH1 mutation inhibitors, similar to Safusidenib, may cause gastrointestinal disturbances, fatigue, and potential liver enzyme abnormalities. It is important to monitor patients closely for these adverse effects during treatment.

What prior FDA approvals exist?

Prior FDA approvals for the treatment of brain tumors include temozolomide, an alkylating agent used for glioblastoma, and bevacizumab, an anti-angiogenic drug approved for recurrent glioblastoma. Targeted therapies such as dabrafenib, a BRAF inhibitor, have also been approved for brain metastases from melanoma. These treatments, like the IDH1 mutation inhibitor Safusidenib currently under study, represent a shift towards targeting specific genetic and molecular abnormalities in brain tumors.

What other types of research exist?

Promising alternatives to Safusidenib for brain tumor patients include ONC201, a selective DRD2 antagonist, which has shown efficacy in treating recurrent glioblastoma and H3 K27M-mutant diffuse midline gliomas. Additionally, GD2-CAR T cell therapy for H3K27M-mutated diffuse midline gliomas and the combination of everolimus and octreotide for aggressive meningiomas are novel treatments showing potential. These therapies are in various stages of clinical trials and have demonstrated promising results in recent studies.

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Small Molecule Inhibitor

Safusidenib for Brain Cancer

Phase 2

Waitlist Available

Research Sponsored by Nuvation Bio Inc.

Eligibility Criteria Checklist

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

Must have

Patient has received no more than 2 prior therapies for disease recurrence/progression.

Patient must be ≥ 18 years of age at the time of signing the informed consent form (ICF).

Must not have

Patients with history or complication of any of the following diseases within 6 months prior to the initial dose of safusidenib: Myocardial infarction, Severe or unstable angina pectoris, Coronary or peripheral endovascular treatment, Heart failure, Cerebrovascular disorder including transient ischemic attack, stroke, central nervous system (CNS) bleeding.

Uncontrolled active systemic fungal, bacterial, or other infection (despite appropriate antibiotics or other treatment).

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from drug treatment to 2 years

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new oral medication called safusidenib for patients with certain types of brain tumors that have not responded to other treatments. The drug works by targeting a specific gene mutation to slow down tumor growth. The study will evaluate the safety and effectiveness of different doses of the medication.

Who is the study for?

Adults over 18 with recurrent or progressive brain tumors (WHO Grade 2/3 glioma) and specific IDH1 mutations can join this trial. They must have had no more than two prior treatments, a measurable lesion, good organ function, and a life expectancy of at least three months. Women must not be able to bear children or use contraception; men should use condoms.

What is being tested?

The study tests different doses of safusidenib, an oral drug for brain tumors with IDH1 mutation. Part 1 assesses safety and initial efficacy in up to 25 patients across five dosage groups. Part 2 evaluates the drug's effectiveness in larger cohorts for each tumor grade.

What are the potential side effects?

Specific side effects are not listed here but may include typical reactions to cancer drugs such as nausea, fatigue, liver issues, blood count changes, and potential allergic reactions based on similar medications.

Eligibility Criteria

Inclusion Criteria

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

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I have had 2 or fewer treatments for my cancer coming back or getting worse.

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I am 18 years or older and can sign the consent form.

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My brain tumor is a Grade 2 or 3 glioma with a specific IDH1 mutation.

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My cancer has an IDH mutation confirmed by a certified lab test.

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My cancer has come back or gotten worse, or I can't handle my current treatment.

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I have a tumor that can be measured by specific medical criteria.

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I can care for myself but may need occasional assistance.

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My kidneys are functioning well.

Exclusion Criteria

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

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I haven't had a heart attack, severe chest pain, heart failure, stroke, or similar issues in the last 6 months.

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I do not have any ongoing infections that aren't responding to treatment.

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I do not currently have an active COVID-19 infection.

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I have a serious irregular heartbeat or my heart's electrical activity is abnormal.

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I am allergic to safusidenib or similar drugs.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from drug treatment to 2 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from drug treatment to 2 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and from drug treatment to 2 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

part1: Incidence of adverse events (AEs) and serious adverse events (SAEs)

part2: ORR by the IRC

Secondary study objectives

Part 1 Stage 1: AUC12h of safusidenib

Part 1 Stage 1: AUC24h [QD only] of safusidenib

Part 1 Stage 1: AUC8h of safusidenib

+21 more

Other study objectives

for part 1 stage 2 surgical participants: 2-hydroxyglutarate (2-HG) concentrations in the tumor tissue

for part 1 stage 2 surgical participants: Safusidenib concentrations in both plasma and tumor tissues

Awards & Highlights

No Placebo-Only Group

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

Trial Design

5Treatment groups

Experimental Treatment

Group I: safusidenib 500mg qdExperimental Treatment1 Intervention

safusidenib 500mg qd administered continuously as a single agent dosed orally on Days 1 to 28 of a 28-day cycle. Subjects may continue treatment with safusidenib until disease progression or development of other unacceptable toxicity.

Group II: safusidenib 500mg bidExperimental Treatment1 Intervention

safusidenib 500mg bid administered continuously as a single agent dosed orally on Days 1 to 28 of a 28-day cycle. Subjects may continue treatment with safusidenib until disease progression or development of other unacceptable toxicity.

Group III: safusidenib 375mg bidExperimental Treatment1 Intervention

safusidenib 375mg bid administered continuously as a single agent dosed orally on Days 1 to 28 of a 28-day cycle. Subjects may continue treatment with safusidenib until disease progression or development of other unacceptable toxicity.

Group IV: safusidenib 250mg bidExperimental Treatment1 Intervention

safusidenib 250mg bid administered continuously as a single agent dosed orally on Days 1 to 28 of a 28-day cycle. Subjects may continue treatment with safusidenib until disease progression or development of other unacceptable toxicity.

Group V: safusidenib 125mg bidExperimental Treatment1 Intervention

safusidenib 125mg bid administered continuously as a single agent dosed orally on Days 1 to 28 of a 28-day cycle. Subjects may continue treatment with safusidenib until disease progression or development of other unacceptable toxicity.

0 / 13Eligibility 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 surgery, radiation therapy, chemotherapy, and targeted therapies. Safusidenib, an IDH1 mutation inhibitor, works by targeting the mutated IDH1 enzyme, which is involved in the abnormal metabolic processes that drive tumor growth. This targeted approach can potentially halt tumor progression with fewer side effects compared to traditional therapies. Understanding these mechanisms is crucial for brain tumor patients as it allows for more personalized and effective treatment plans, improving outcomes and quality of life.

[Pediatric ependymomas: Current diagnosis and therapy].Bioinformatics analysis reveals potential candidate drugs for different subtypes of glioma.