What side effects are associated with this type of intervention?

Common treatments for brain tumors, particularly those targeting IDH1 and IDH2 mutations like Vorasidenib, often include side effects such as fatigue, nausea, and liver enzyme abnormalities. Other treatments for brain tumors, such as chemotherapy and radiation therapy, can cause additional side effects including hair loss, cognitive changes, and increased risk of infections. Targeted therapies may also lead to specific toxicities like skin rashes and gastrointestinal issues.

What prior FDA approvals exist?

The FDA has approved several treatments for brain tumors, particularly gliomas, which target specific genetic mutations and pathways. One notable example is the approval of drugs like temozolomide, which is used in combination with radiation therapy for glioblastoma. Additionally, the FDA has approved bevacizumab, an anti-angiogenic therapy, for recurrent glioblastoma. While specific approvals for IDH1 and IDH2 inhibitors like Vorasidenib are still under investigation, these treatments represent a growing focus on targeted therapies in the management of brain tumors.

What other types of research exist?

Promising alternatives to Vorasidenib for brain tumor patients include Bevacizumab, an antiangiogenic therapy shown to be effective in neurofibromatosis type 2-related vestibular schwannomas, and Everolimus, an mTORC1 inhibitor studied in patients with growing vestibular schwannomas. Both therapies have demonstrated efficacy in clinical trials and could be considered for glioma treatment.

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Vorasidenib for Glioma

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

Common treatments for brain tumors include chemotherapy, radiation therapy, and targeted molecular therapies. Chemotherapy uses drugs to kill rapidly dividing cancer cells, while radiation therapy uses high-energy rays to destroy tumor cells. Targeted molecular therapies, such as Vorasidenib, inhibit specific genetic mutations like IDH1 and IDH2, reducing the production of the oncometabolite 2-hydroxyglutarate, which is implicated in glioma development and progression. These treatments are crucial as they aim to specifically target tumor cells, potentially improving outcomes and reducing side effects for brain tumor patients.

Phase 3

Waitlist Available

Research Sponsored by Agios Pharmaceuticals, Inc.

Eligibility Criteria Checklist

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

Must have

Have MRI-evaluable, measurable, non-enhancing disease, as confirmed by the BIRC

Have a Karnofsky Performance Scale (KPS) score (for participants ≥16 years of age) or Lansky Play Performance Scale (LPPS) score (for participants <16 years of age) of ≥80%

Must not have

Have had any prior anticancer therapy other than surgery (biopsy, sub-total resection, gross-total resection) for treatment of glioma including systemic chemotherapy, radiotherapy, vaccines, small-molecules, IDH inhibitors, investigational agents, laser ablation, etc

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to approximately 30 months

Awards & highlights

Pivotal Trial

Summary

This trial is testing an oral medication called vorasidenib in patients with a specific type of brain tumor that has a genetic mutation. The drug aims to block proteins that help the tumor grow, potentially slowing or stopping its growth.

Who is the study for?

This trial is for people at least 12 years old with a Grade 2 glioma brain tumor that has returned or remains after surgery. They must weigh over 40 kg, have had no other cancer treatments like chemo or radiation, and not need immediate therapy. Participants require confirmed IDH1/IDH2 mutations and measurable disease on MRI.

What is being tested?

The INDIGO study tests Vorasidenib against a placebo in patients with specific recurrent brain tumors. About 340 participants will be randomly assigned to take either the drug or placebo orally once daily, without knowing which one they're receiving.

What are the potential side effects?

While the trial information does not specify side effects, similar drugs can cause headaches, nausea, fatigue, joint pain, and may affect liver function. Side effects vary from person to person.

Eligibility Criteria

Inclusion Criteria

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

Select...

My MRI shows measurable disease that doesn't enhance with contrast.

Select...

I am mostly active and can carry out my daily activities without help.

Select...

My cancer has a specific IDH1 or IDH2 gene mutation, confirmed by lab tests.

Select...

My brain tumor is classified as Grade 2 oligodendroglioma or astrocytoma.

Select...

I am at least 12 years old and weigh 40 kg or more.

Exclusion Criteria

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

Select...

I have only had surgery for my glioma, no other cancer treatments.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to approximately 30 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to approximately 30 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to approximately 30 months 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)

Awards & Highlights

Pivotal Trial

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

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: VorasidenibExperimental Treatment1 Intervention

Vorasidenib 40 mg, continuous daily dosing.

Group II: Matching PlaceboPlacebo Group1 Intervention

Matching placebo 40 mg, continuous daily dosing.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Vorasidenib

2023

Completed Phase 1

~20

0 / 6Eligibility 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 chemotherapy, radiation therapy, and targeted molecular therapies. Chemotherapy uses drugs to kill rapidly dividing cancer cells, while radiation therapy uses high-energy rays to destroy tumor cells. Targeted molecular therapies, such as Vorasidenib, inhibit specific genetic mutations like IDH1 and IDH2, reducing the production of the oncometabolite 2-hydroxyglutarate, which is implicated in glioma development and progression. These treatments are crucial as they aim to specifically target tumor cells, potentially improving outcomes and reducing side effects for brain tumor patients.

Increased mitochondrial activity in a novel IDH1-R132H mutant human oligodendroglioma xenograft model: in situ detection of 2-HG and α-KG.Bioinformatics analysis reveals potential candidate drugs for different subtypes of glioma.