Phase 1/2 Study of Mirdametinib + Vinblastine for Newly Diagnosed/Previously Untreated PLGG + Act… (NCT06666348) | Clinical Trial Compass
RecruitingPhase 1/2
Phase 1/2 Study of Mirdametinib + Vinblastine for Newly Diagnosed/Previously Untreated PLGG + Activation of MAPK
Canada50 participantsStarted 2026-01-12
Plain-language summary
This is a 3-part open-label study (feasibility phase, treatment phase and follow-up phase) of orally administered mirdametinib in combination with intravenous vinblastine chemotherapy in patients with PLGG with activation of MAPK pathway.
Feasibility Phase:
The maximum tolerated/recommended phase 2 dose (MTD/RP2D) of the mirdametinib plus vinblastine combination will be assessed using a modified Rolling-6 design.
Treatment Phase:
Patients will receive mirdametinib twice daily (continuously) at a fixed dose (2mg/m2 po BID up to 4 mg BID) for a total of 13 cycles (28 days cycle). Weekly intravenous vinblastine at MTD will be given for a total of 17 cycles. If adverse events occur, two dose reductions are allowed.
Follow-up Phase:
Following the end of treatment, patients will be scheduled for a follow-up visit every 6 months for 36 months to evaluate PFS, TTP and OS.
Who can participate
Age range
2 Years – 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:
* Signed written informed consent prior to study participation.
* Study activities compliance: must be willing and able to comply with scheduled visits, treatment schedule, laboratory testing, and other requirements of the study, including disease assessment by contrast enhanced MRI.
* Aged ≥ 2 years to ≤ 25 years when starting mirdametinib.
* BSA ≥ 0,40m2
* Diagnosis:
Participants must have PLGG with NF1 gene mutation (based on clinical NIH criteria, germline NF1 mutation or molecular analysis of the tumor) or PLGG with KIAA1549-BRAF fusion (based on molecular analysis of the tumor) or PLGG with evidence of MAPK pathway alteration with the exception of patients with BRAF V600E mutation (based on molecular analysis of the tumor).
* Tumor tissue is required (at minimum, paraffin-embedded tissue block and additionally fresh frozen tissue \[if available\]). Patients with NF1 and Low Grade Glioma (LGG) can still be enrolled without tissue if no surgery or biopsy was conducted.
* Baseline MRI.
* Life expectancy greater than 6 months.
* Lansky/Karnofsky score ≥ 50.
* Normal organ and marrow function (see study protocol for specifics).
* Female and male patients of fertile age must agree to use highly effective contraceptive measures.
* Must be able to ingest by mouth and retain entirely the administered medication. Mirdametinib can not be administered via nasogastric tube or gastrostomy tube.
Exclusion Criteria:
* Patients who are receiving other investigat…
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 1/2 trial and part of the goal is still finding the right dose of mirdametinib combined with vinblastine, what does that mean for what's currently known about the safety of this combination in children?
2My child's tumor would need to show MAPK pathway activation to be eligible — has their tumor already been tested for this, and if not, what does that testing involve?
3How does combining mirdametinib with vinblastine compare to the standard chemotherapy approaches typically used first for pediatric low-grade glioma, and would starting with a standard treatment be a better path before considering a trial like this?
4Since this trial is specifically for newly diagnosed, previously untreated patients, does enrolling now affect which treatments would still be available if the tumor came back or didn't respond?
5What does the treatment schedule for mirdametinib plus vinblastine actually look like week to week, and how might the demands of participating in this trial — like clinic visits, monitoring, or travel — fit into our family's daily life?
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
Determine maximum tolerated dose of Mirdametinib plus Vinblastine.
Timeframe: 3 years
2
Determine the objective response rate of mirdametinib plus vinblastine for PLGG with Mitogen-activated Protein Kinase (MAPK) pathway activation in treatment naïve patients.