NCT06666348 Phase 1/2 Study of Mirdametinib + Vinblastine for Newly Diagnosed/Previously Untreated PLGG + Activation of MAPK
| NCT ID | NCT06666348 |
| Status | Recruiting |
| Phase | Phase 1, Phase 2 |
| Sponsor | St. Justine's Hospital |
| Condition | Pediatric Low-grade Glioma |
| Study Type | INTERVENTIONAL |
| Enrollment | 50 participants |
| Start Date | 2026-01-12 |
| Primary Completion | 2030-04 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 50 participants in total. It began in 2026-01-12 with a primary completion date of 2030-04.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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.
Eligibility Criteria
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 investigational agents. * Cardiac: QTcB ≥ 480 msec or an absolute resting left ventricular ejection fraction (LVEF) of ≤ 49%. * Patients who have any other malignancy, except if the other primary malignancy is neither currently clinically significant nor requiring active intervention. * Tumor with BRAF V600E mutation. * Patients who received previous systemic or radiotherapy treatment. * Other severe and uncontrollable medical disease * Blood pressure higher than 95th percentile for patient's age, height and gender. * Increased risk of serious retinopathy and retinal vein occlusion. * Known diagnosis of human immunodeficiency virus infection, hepatitis B or C. * Previous major surgery within 2 weeks. * History of allergic reactions to compounds of similar chemical or biological composition to mirdametinib. * Pregnant or breastfeeding.
Contact & Investigator
Sébastien Perreault, M.D, FRCPC
STUDY CHAIR
CHU Sainte Justine, URCHOI Department
Frequently Asked Questions
Who can join the NCT06666348 clinical trial?
This trial is open to participants of all sexes, aged 2 Years or older, up to 25 Years, studying Pediatric Low-grade Glioma. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT06666348 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT06666348 currently recruiting?
Yes, NCT06666348 is actively recruiting participants. Contact the research team at s.perreault@umontreal.ca for enrollment information.
Where is the NCT06666348 trial being conducted?
This trial is being conducted at Calgary, Canada, Edmonton, Canada, Vancouver, Canada, Winnipeg, Canada and 6 additional locations.
Who is sponsoring the NCT06666348 clinical trial?
NCT06666348 is sponsored by St. Justine's Hospital. The principal investigator is Sébastien Perreault, M.D, FRCPC at CHU Sainte Justine, URCHOI Department. The trial plans to enroll 50 participants.