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| Diagnosis | Pediatric Low-grade Glioma | Study Status | Open |
| Phase | I/II |
| Age | 2 Years to 25 Years | Randomisation | NO |
| Line of treatment | First line treatment |
| Routes of Treatment Administration | Drug: Mirdametinib (oral)
Drug: Vinblastine (IV) |
| Last Posted Update | 2026-01-14 |
| ClinicalTrials.gov # | NCT06666348 |
International Sponsor
St. Justine's HospitalPrincipal Investigators for Canadian Sites
CHU Sainte-Justine - Dr. Sebastian Perreault
The Hospital for Sick Children - Dr. Anthony Liu
London Children's Hospital - Dr. Shayna Zelcer Centres
Medical contact
Dr. Henrique Bittencourt
Dr. Monia Marzouki
Dr. Sebastien Perreault (neuro-onc)
Social worker/patient navigator contact
Marie-Claude Charrette
Clinical research contact
Marie Saint-Jacques
Medical contact
Dr. Alexandra Zorzi
Dr. Shayna Zelcer
Social worker/patient navigator contact
Cindy Milne Wren
Jessica Mackenzie Harris
Clinical research contact
Mariam Mikhail
Study Description
This is a research study combining an oral drug called mirdametinib with an intravenous chemotherapy drug called vinblastine for the treatment of patients with pediatric low grade glioma with certain mutations.
In this first part, doctors will carefully test different dose levels of the two medicines to find the safest and most appropriate dose to use in the next phase. Patients will take mirdametinib by mouth twice a day at a set dose for 13 treatment cycles (each cycle is 28 days). They will also receive vinblastine through an IV once a week for 17 cycles. If side effects happen, the dose can be lowered up to two times to keep treatment safe. After treatment ends, patients will come back for check-ups every 6 months for 3 years. These visits help doctors track how the tumour responds over time and how patients are doing overall.
Inclusion Criteria
- A parent or substitute decision maker (or the participant if they have capacity) must sign a consent form before joining the study
- Participants need to be able to come to scheduled appointments, take study medicines as instructed, complete lab tests, and have MRIs as part of their care
- Participants must be between 2 and 25 years old when starting mirdametinib.
- Participants must have a type of low-grade brain tumour (PLGG) with one of the following:
- An NF1 gene change (based on medical criteria or genetic testing), or
- A KIAA1549-BRAF fusion, or
- Another MAPK pathway change, except for the BRAF V600E mutation.
- Tumour tissue is needed for testing (usually a stored tissue block, and fresh frozen tissue if available).
- Children with NF1 and low-grade glioma can still join even if they never had surgery or a biopsy.
- A recent MRI before starting
- Must be well enough to take part in daily activities at least 50% of waking hours
- Organ and bone marrow function and body surface area must be within allowable limits
- Teens and adults who could become pregnant must use effective birth control during the study
- Must be able to swallow the medication by mouth, as this drug cannot be given through feeding tubes
Other inclusion and exclusion criteria may apply and will be discussed with you by the study team.
