Trametinib and Everolimus for Treatment of Pediatric and Young Adult Patients With Recurrent Glio… (NCT04485559) | Clinical Trial Compass
RecruitingPhase 1
Trametinib and Everolimus for Treatment of Pediatric and Young Adult Patients With Recurrent Gliomas (PNOC021)
United States50 participantsStarted 2020-12-09
Plain-language summary
This phase I trial studies the side effects and best dose of trametinib and everolimus in treating pediatric and young adult patients with gliomas that have come back (recurrent). Trametinib acts by targeting a protein in cells called MEK and disrupting tumor growth. Everolimus is a drug that may block another pathway in tumor cells that can help tumors grow. Giving trametinib and everolimus may work better to treat low and high grade gliomas compared to trametinib or everolimus alone.
Who can participate
Age range
1 Year – 25 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.
Inclusion Criteria:
* Participants must have histologically confirmed diagnosis of an LGG (WHO grade I-II) that is recurrent or progressive after prior treatment (biologic, chemotherapy or radiation therapy) or must have a histologically confirmed diagnosis of a high grade glioma (HGG) (WHO grade III-VI)
* Participants with LGG who have had surgery alone are not eligible.
* Participants with neurofibromatosis type 1 (NF-1) are eligible but must have available tissue per study requirements neurofibromatosis (NF) status will be collected
* Participants with spinal cord primaries or disseminated disease are eligible
* For enrollment, snap frozen tissue (150 mg) or 10 unstained 10 um formalin-fixed, paraffin-embedded (FFPE) slides for comprehensive genomic testing or results of prior testing is required
* If clinical comprehensive testing has already been performed, the requirement for submission of tissue may be waived after discussion and review of results with study chairs
* Participants must have evaluable disease
* Prior therapy: Participants must have received prior therapy other than surgery and must have fully recovered from the acute toxic effects of all prior chemotherapy, biologics, immunotherapy, or radiotherapy prior to entering this study
* Myelosuppressive chemotherapy: Participants must have received their last dose of known myelosuppressive anticancer chemotherapy at least three weeks prior to study registration or at least six weeks if they had rec…
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 is a Phase 1 trial focused on finding the right dose and measuring side effects of trametinib plus everolimus, what does that mean for what's currently known about whether this combination actually helps recurrent gliomas in kids — and how does that uncertainty weigh against other options for us?
2The trial is testing two different dosing schedules — continuous and intermittent — and one of the main goals is figuring out how much of each drug is too much; given that, how would you describe the level of safety risk involved, and what kinds of side effects have been seen with trametinib or everolimus individually in children?
3Since this trial is specifically for recurrent gliomas that have already come back, is there a standard treatment path we should consider first, or has my child's situation already reached the point where a trial like this might be the most appropriate next step?
4The trial involves detailed pharmacokinetic measurements — blood samples to track how the drug moves through the body — how often would my child need to come in for visits or tests, and is that level of commitment realistic for our family's situation?
5If my child enrolls and experiences a dose-limiting toxicity, what happens next — would they be taken off the trial entirely, switched to a different dose schedule, or managed some other way?
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
1
Maximum tolerated dose (MTD) of trametinib in combination with everolimus for both continuous and intermittent dosing schedules
Timeframe: Up to 28 days
2
Incidence of adverse events for both continuous and intermittent dosing schedules
Timeframe: Up to 30 days after the last day of treatment
3
Dose limiting toxicities (DLTs) of the combination for both continuous and intermittent dosing schedules
Timeframe: Up to 28 days
4
Recommended phase 2 dose (RP2D)
Timeframe: Up to 28 days
5
Maximum Concentration (Cmax) of trametinib and everolimus
Timeframe: Up to 5 years
6
Area Under the Curve (AUC) of trametinib and everolimus