CompletedPhase 1/2

Study of MEK162 for Children With Low-Grade Gliomas

United States105 participantsStarted 2016-05-04

Plain-language summary

The goal of this clinical trial is to study the drug MEK162 in children with a brain tumor call low-grade glioma, as well as in children with other tumors in which a specific growth signal is abnormally turned on. The main questions it aims to answer are: What is the correct dose of MEK162 in children? What are the side effects of MEK162 in children? Is MEK162 effective in children with low-grade glioma? Participants on the study receive MEK162 by mouth twice daily for up to 2 years.

Who can participate

Age range

1 Year – 18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Patients with recurrent or progressive disease, as defined in the following three strata below, will be eligible. For eligibility determination, tumor imaging from at least two time-points must be available to document radiographic progression or recurrence. Patients with non-progressive refractory tumors will not be eligible. * Stratum 1: patients with LGG with a BRAF truncated fusion that is measurable in at least two dimensions on imaging. * Stratum 2: patients with NF1 and LGG that is measurable in at least two dimensions on imaging. * Stratum 3: Pediatric patients with a recurrent or progressive tumor thought to involve the Ras/Raf/ERK pathway but not included in strata 1 or 2 that is measurable in at least two dimensions on imaging. This includes any LGG not included in strata 1 or 2 (i.e., any LGG without a BRAF truncated fusion in a patient without NF1), any tumor other than LGG in a patient with NF1, and any other tumor with a documented activating BRAF, NRAS, or KRAS mutation. * Stratum 4 (surgical arm, target validation): Patients who meet criteria for stratum 1, 2, or 3 for whom tumor biopsy and/or resection is clinically indicated. * Tumor tissue for correlative studies must be available for all patients except those with NF1 and LGG (stratum 2) or any patient with optic pathway glioma (stratum 2 or 3), for whom tumor tissue is optional. * Patients must have received at least one prior chemotherapy or radiation regimen prior to progression. * At the time of enro…

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Since this MEK162 trial in children with low-grade gliomas has already completed, has the data been published, and what did it show about how well the drug controlled tumor growth over 12 treatment cycles?
2Because this was a Phase 1/2 study, what do the results tell us about the safety profile of MEK162 in children specifically, and are there side effects we should watch for if this drug becomes part of a treatment plan?
3The trial tracked 'best overall response rate' as a key outcome — does that data suggest MEK162 worked better for certain subtypes of low-grade glioma, and would my child's specific tumor type be one that responded?
4Are there now newer MEK inhibitors or other targeted therapies that have moved further along in testing since this trial completed, and would those be a better option to consider instead?
5How does MEK162 compare to the standard treatments currently available for low-grade gliomas in children, and would it make sense to try standard therapy first before considering something based on this trial's findings?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Best Overall Response Rate (Strata 1 and 2)

Timeframe: 48 weeks

12 Cycle Progression-free and Overall Survival (Strata 1 and 2)

Timeframe: 48 weeks

Trial details

NCT IDNCT02285439

SponsorChildren's Hospital Los Angeles

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2022-11-02

Results submitted2025-07-21

View on ClinicalTrials.gov More Low-Grade Gliomas trials