What side effects are associated with this type of intervention?

MEK inhibitors like binimetinib, used in the treatment of Adamantinomatous Craniopharyngioma, commonly cause side effects such as rash, diarrhea, fatigue, peripheral edema, and nausea. Serious but less common side effects include cardiomyopathy, retinal vein occlusion, and interstitial lung disease.

What prior FDA approvals exist?

There is no specific mention of prior FDA approvals for the treatment of Adamantinous Craniopharyngioma using MEK1 and MEK2 inhibitors like MEKTOVI (binimetinib). However, MEKTOVI is currently being studied in a Phase II trial for pediatric patients with recurrent Adamantinous Craniopharyngioma. This suggests that MEK inhibitors are being explored as a potential treatment option, although no prior FDA approvals for this specific indication are noted.

What other types of research exist?

Promising alternatives to MEKTOVI (binimetinib) for Adamantinous Craniopharyngioma patients include bevacizumab, which has shown efficacy in various neurofibromatosis-related tumors, and everolimus, an mTORC1 inhibitor that has been studied in neurofibromatosis type 2 patients with growing vestibular schwannomas. Additionally, lapatinib, an EGFR and HER2 inhibitor, has been evaluated in clinical trials for neurofibromatosis type 2 and may offer potential benefits.

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MEK Inhibitor

MEKTOVI for Pediatric Craniopharyngioma

Phase 2

Recruiting

Led By Maryam Fouladi

Research Sponsored by Nationwide Children's Hospital

Eligibility Criteria Checklist

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

Must have

Patients must be ≥ 12 months and ≤ 25 years of age at the time of study enrollment

Karnofsky ≥ 50% for patients > 16 years of age and Lansky ≥ 50 for patients ≤ 16 years of age

Must not have

Patients with a history of serious gastrointestinal disease or inability to swallow, retain, or absorb oral medications

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 24 months

Awards & highlights

No Placebo-Only Group

Summary

This trial tests MEKTOVI, a pill that targets cancer growth proteins, in children and young adults with a recurring brain tumor called Adamantinomatous Craniopharyngioma. The drug works by blocking signals that make cancer cells grow.

Who is the study for?

This trial is for kids and young adults aged 1 to 25 with recurrent Adamantinomatous Craniopharyngioma, a type of brain tumor. They must have measurable disease, stable health conditions, and meet specific organ function criteria. Those who've had recent surgery or certain treatments aren't eligible.

What is being tested?

The trial tests MEKTOVI (binimetinib), an oral drug that blocks specific enzymes in cancer cells. It's given to patients who've had previous surgeries or radiation therapy for their brain tumors. The study aims to see how well it works in these young patients.

What are the potential side effects?

Possible side effects include digestive issues, liver problems, heart complications like changes in heartbeat rhythm, blood disorders such as low counts of white cells or platelets which can increase infection risk and bleeding tendency respectively.

Eligibility Criteria

Inclusion Criteria

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

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I am between 12 months and 25 years old.

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I am mostly able to care for myself, regardless of my age.

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I have been diagnosed with adamantinomatous craniopharyngioma.

Exclusion Criteria

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

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I have severe stomach issues or can't take pills properly.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 24 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~24 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 24 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

Sustained objective response rate of patients with measurable ACP who have undergone surgery but have not been previously treated with radiation to treatment with oral binimetinib

Sustained objective response rate of patients with recurrent/progressive previously irradiated ACP to treatment with oral binimetinib

Secondary study objectives

Biological effects of binimetinib on ACP tumor tissue and cyst fluid.

PFS of ACP patients treated with binimetinib after radiation

PFS of ACP patients treated with binimetinib who have not received radiation

+1 more

Awards & Highlights

No Placebo-Only Group

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

Trial Design

1Treatment groups

Experimental Treatment

Group I: Stratum 1 and Stratum 2Experimental Treatment1 Intervention

Stratum 1: Patients with progressive or recurrent adamantinomatous craniopharyngiomas following radiation therapy. Stratum 2: Patients with measurable adamantinomatous craniopharyngioma who have undergone surgery but have not previously received radiation therapy. Progressive disease is allowed but not required

0 / 4Eligibility 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

MEK inhibitors, such as binimetinib, target the MEK1 and MEK2 enzymes in the MAPK/ERK signaling pathway, which is frequently dysregulated in Adamantinous Craniopharyngioma. By inhibiting these enzymes, MEK inhibitors can reduce tumor cell proliferation and induce apoptosis. This targeted approach is crucial for Adamantinous Craniopharyngioma patients, as these tumors are often resistant to conventional therapies, making MEK inhibitors a promising option for more effective disease management.

Casein Kinase 1D Encodes a Novel Drug Target in Hedgehog-GLI-Driven Cancers and Tumor-Initiating Cells Resistant to SMO Inhibition.Phase I study using crenolanib to target PDGFR kinase in children and young adults with newly diagnosed DIPG or recurrent high-grade glioma, including DIPG.IGF1R Is a Potential New Therapeutic Target for HGNET-BCOR Brain Tumor Patients.